254. Porphyria Clinical trials / Disease details
Clinical trials : 70 / Drugs : 54 - (DrugBank : 19) / Drug target genes : 19 - Drug target pathways : 35
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2021-001831-17-ES (EUCTR) | 01/03/2022 | 16/12/2021 | An open label, long-term extension study to determine how safe and tolerable oral Dersimelagon is in subjects with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) | A Phase 3, Multicenter, Open-label, Long-term, Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) | Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 24.0;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Symptoms and general pathology [C23] | Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive name: MT-7117 | Mitsubishi Tanabe Pharma Development America (MTDA), Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 184 | Phase 3 | United States;Canada;Spain;Germany;United Kingdom;Japan | ||
| 2 | NCT05020184 (ClinicalTrials.gov) | December 2021 | 26/7/2021 | Effect of Oral Cimetidine in the Protoporphyrias | Effect of Oral Cimetidine in the Protoporphyrias | Erythropoietic Protoporphyria;X-linked Protoporphyria | Drug: Cimetidine;Drug: Placebo | Amy K. Dickey, M.D. | University of Texas | Not yet recruiting | 15 Years | N/A | All | 20 | Phase 2 | United States |
| 3 | NCT05005975 (ClinicalTrials.gov) | August 10, 2021 | 28/7/2021 | Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) | A Phase 3, Multicenter, Open-label, Long-term, Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) | EPP;XLP | Drug: MT-7117 | Mitsubishi Tanabe Pharma Development America, Inc. | NULL | Recruiting | 12 Years | 75 Years | All | 175 | Phase 3 | United States;Australia;Canada;Germany;Italy;Japan;Norway;Spain;Sweden;United Kingdom |
| 4 | NCT04923516 (ClinicalTrials.gov) | June 2021 | 8/6/2021 | Prevalence of Acute Hepatic Porphyria | Prevalence of Acute Hepatic Porphyria in Population With Suggestive Clinical Picture | Acute Hepatic Porphyria | Other: Dosage of Delta-aminolevulinic acid and Porphobilinogen | Association pour la Recherche en Medecine Interne | NULL | Not yet recruiting | 18 Years | 60 Years | All | 500 | NULL | |
| 5 | EUCTR2019-004226-16-FI (EUCTR) | 29/04/2021 | 05/10/2020 | A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents with Erythropoietic Protoporphyria or x-linked Protoporphyria | A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria - RESPITE | Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 21.1;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Symptoms and general pathology [C23] | Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive name: MT-7117 | Mitsubishi Tanabe Pharma Development America (MTDA), Inc. | NULL | Not Recruiting | Female: yes Male: yes | 159 | Phase 3 | United States;Canada;Finland;Spain;Australia;Norway;Germany;United Kingdom;Japan;Italy;Sweden | ||
| 6 | EUCTR2019-004226-16-DE (EUCTR) | 20/04/2021 | 15/09/2020 | A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents with Erythropoietic Protoporphyria or x-linked Protoporphyria | A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria - RESPITE | Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 24.0;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Symptoms and general pathology [C23] | Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive name: MT-7117 | Mitsubishi Tanabe Pharma Development America (MTDA), Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 184 | Phase 3 | United States;Canada;Finland;Spain;Australia;Norway;Germany;United Kingdom;Italy;Japan;Sweden | ||
| 7 | JPRN-jRCT2071200074 | 02/02/2021 | 24/12/2020 | Expanded Access Protocol of Givosiran for Patients with Acute Hepatic Porphyria | Expanded Access Protocol of Givosiran for Patients with Acute Hepatic Porphyria (AHP) | Acute hepatic porphyria (AHP) | Patients will be administered givosiran 2.5 mg/kg as a subcutaneous (SC) injection once monthly. | Fujii Norihisa | NULL | Not Recruiting | >= 12age old | Not applicable | Both | 10 | N/A | Japan |
| 8 | EUCTR2019-004226-16-GB (EUCTR) | 04/11/2020 | 29/05/2020 | A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents with Erythropoietic Protoporphyria or x-linked Protoporphyria | A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria - RESPITE | Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 21.1;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Symptoms and general pathology [C23] | Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive name: MT-7117 | Mitsubishi Tanabe Pharma Development America (MTDA), Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 159 | Phase 3 | United States;Canada;Finland;Spain;Australia;Norway;Germany;Japan;Italy;United Kingdom;Sweden | ||
| 9 | EUCTR2019-004226-16-IT (EUCTR) | 08/10/2020 | 21/10/2020 | Study to Evaluate Efficacy, Safety, and Tolerability of MT 7117 in Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria | A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT 7117 in Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria - RESPITE | Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 21.1;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Symptoms and general pathology [C23] | Product Name: Dersimelagon Product Code: [MT-7117] Other descriptive name: A novel synthetic, orally-administered, non-peptide small molecule, which acts as | Mitsubishi Tanabe Development America Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 159 | Phase 3 | United States;Canada;Finland;Spain;Australia;Norway;Germany;United Kingdom;Japan;Italy;Sweden | ||
| 10 | EUCTR2019-004226-16-SE (EUCTR) | 06/07/2020 | 26/05/2020 | A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents with Erythropoietic Protoporphyria or x-linked Protoporphyria | A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria - RESPITE | Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 24.0;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Symptoms and general pathology [C23] | Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive name: MT-7117 | Mitsubishi Tanabe Pharma Development America (MTDA), Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 184 | Phase 3 | United States;Canada;Finland;Spain;Australia;Norway;Germany;United Kingdom;Italy;Japan;Sweden | ||
| 11 | NCT04402489 (ClinicalTrials.gov) | June 1, 2020 | 20/5/2020 | Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoetic Protoporphyria or X-Linked Protoporphyria | A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents With Erythropoietic Protoporphyria or X-Linked Protoporphyria | EPP;XLP | Drug: Placebo;Drug: MT-7117 Low Dose;Drug: MT-7117 High Dose | Mitsubishi Tanabe Pharma Development America, Inc. | NULL | Active, not recruiting | 12 Years | 75 Years | All | 184 | Phase 3 | United States;Australia;Canada;Germany;Italy;Japan;Norway;Spain;Sweden;United Kingdom;Finland |
| 12 | NCT04056481 (ClinicalTrials.gov) | August 20, 2019 | 12/8/2019 | Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria | Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria (AHP) | Acute Hepatic Porphyria | Drug: Givosiran | Alnylam Pharmaceuticals | NULL | Approved for marketing | 12 Years | N/A | All | Sweden;Australia;Belgium;Canada;France;Germany;Luxembourg;Netherlands;Spain;United States | ||
| 13 | NCT03520036 (ClinicalTrials.gov) | July 5, 2018 | 23/4/2018 | Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria | A Phase II, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria | Erythropoietic Protoporphyria (EPP) | Drug: MT-7117 low dose;Drug: MT-7117 high dose;Drug: Placebo | Mitsubishi Tanabe Pharma Development America, Inc. | NULL | Completed | 18 Years | 75 Years | All | 102 | Phase 2 | United States |
| 14 | EUCTR2017-002432-17-NL (EUCTR) | 21/06/2018 | 19/02/2018 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias - ENVISION | Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid) | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Poland;Belgium;Brazil;Australia;Denmark;South Africa;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 15 | EUCTR2017-002432-17-FI (EUCTR) | 21/05/2018 | 30/11/2017 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias - ENVISION | Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid) | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Australia;Denmark;Bulgaria;Germany;Netherlands;Japan;Sweden;Korea, Republic of | ||
| 16 | NCT03505853 (ClinicalTrials.gov) | April 26, 2018 | 13/4/2018 | A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP) | A Drug-Drug Interaction Study to Investigate the Effect of Givosiran on the Pharmacokinetics (PK) of Midazolam, Caffeine, Losartan, Omeprazole, and Dextromethorphan in Patients With Acute Intermittent Porphyria (AIP) Who Are Asymptomatic High Excreters (ASHE) | Acute Intermittent Porphyria (AIP);Acute Hepatic Porphyria (AHP);Porphyria, Acute Intermittent;Acute Porphyria | Drug: Givosiran;Drug: 5-probe cocktail | Alnylam Pharmaceuticals | NULL | Completed | 18 Years | 65 Years | All | 10 | Phase 1 | Sweden |
| 17 | EUCTR2017-002432-17-PL (EUCTR) | 12/04/2018 | 16/01/2018 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias - ENVISION | Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid) | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Czechia;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Poland;Brazil;Belgium;Australia;Denmark;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 18 | EUCTR2017-002432-17-BG (EUCTR) | 19/02/2018 | 09/11/2017 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias - ENVISION | Acute Hepatic Porphyrias (AHP) MedDRA version: 24.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid) | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Australia;Denmark;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 19 | EUCTR2017-002432-17-IT (EUCTR) | 19/02/2018 | 02/02/2021 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias - ENVISION | Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 | ALNYLAM PHARMACEUTICALS, INC. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Czechia;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Poland;Brazil;Belgium;Denmark;Australia;South Africa;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 20 | EUCTR2017-002432-17-DE (EUCTR) | 03/01/2018 | 04/10/2017 | ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias - ENVISION | ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias - ENVISION | Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid) | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Australia;Denmark;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 21 | EUCTR2017-002432-17-BE (EUCTR) | 29/12/2017 | 19/12/2017 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias - ENVISION | Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid) | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Poland;Belgium;Brazil;Australia;Denmark;South Africa;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 22 | EUCTR2017-002432-17-SE (EUCTR) | 21/12/2017 | 07/11/2017 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias - ENVISION | Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid) | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Australia;Denmark;Bulgaria;Germany;Netherlands;Japan;Sweden;Korea, Republic of | ||
| 23 | EUCTR2016-002638-54-SE (EUCTR) | 20/12/2017 | 09/08/2016 | Long-term Safety and Clinical Activity of ALN-AS1 in Patients with Acute Intermittent Porphyria (AIP), a rare metabolic disorder. | A Multicenter, Open-label Extension Study to Evaluate the Long-term Safety and Clinical Activity of Subcutaneously Administered ALN-AS1 in Patients with Acute Intermittent Porphyria who have Completed a Previous Clinical Study with ALN-AS1 | Acute Intermittent Porphyria (AIP) MedDRA version: 20.0;Level: LLT;Classification code 10000818;Term: Acute intermittent porphyria;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid) | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 24 | Phase 1;Phase 2 | United States;United Kingdom;Sweden | ||
| 24 | EUCTR2017-002432-17-DK (EUCTR) | 18/12/2017 | 25/10/2017 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias - ENVISION | Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid) | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Denmark;Australia;Bulgaria;Germany;Netherlands;Japan;Sweden;Korea, Republic of | ||
| 25 | EUCTR2017-002432-17-ES (EUCTR) | 11/12/2017 | 17/10/2017 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias - ENVISION | Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid) | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Poland;Belgium;Brazil;Australia;Denmark;South Africa;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 26 | EUCTR2017-002432-17-GB (EUCTR) | 22/11/2017 | 26/09/2017 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias - ENVISION | Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid) | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;Italy;United Kingdom;Switzerland;France;Czech Republic;Mexico;Canada;Poland;Belgium;Brazil;Australia;Denmark;Bulgaria;Netherlands;Germany;Japan;Korea, Republic of;Sweden | ||
| 27 | NCT03338816 (ClinicalTrials.gov) | November 16, 2017 | 7/11/2017 | ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP) | ENVISION: A Phase 3 Randomized, Double-blind, Placebo-Controlled Multicenter Study With an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients With Acute Hepatic Porphyrias | Acute Hepatic Porphyria;Acute Intermittent Porphyria;Porphyria, Acute Intermittent;Acute Porphyria;Hereditary Coproporphyria (HCP);Variegate Porphyria (VP);ALA Dehydratase Deficient Porphyria (ADP) | Drug: Givosiran;Drug: Placebo | Alnylam Pharmaceuticals | NULL | Completed | 12 Years | N/A | All | 94 | Phase 3 | United States;Australia;Bulgaria;Canada;Denmark;Finland;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Spain;Sweden;Taiwan;United Kingdom;Belgium;Switzerland |
| 28 | NCT03118674 (ClinicalTrials.gov) | September 6, 2017 | 13/4/2017 | Harvoni Treatment Porphyria Cutanea Tarda | Newer Direct-Acting Anti-Viral Agents as Sole Therapy of Porphyria Cutanea Tarda in Subjects With Chronic Hepatitis C | Porphyria Cutanea Tarda;Hepatitis C | Drug: Harvoni | Wake Forest University Health Sciences | Gilead Sciences;National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);National Institutes of Health (NIH) | Active, not recruiting | 18 Years | N/A | All | 25 | Phase 2 | United States |
| 29 | NCT02979249 (ClinicalTrials.gov) | December 2016 | 29/11/2016 | Oral Iron for Erythropoietic Protoporphyrias | Effect of Oral Iron Therapy on Erythrocyte Protoporphyrin Levels in the Erythropoietic Protoporphyrias | Erythropoietic Protoporphyria;EPP;X-linked Protoporphyria;XLP | Drug: Oral Iron | Icahn School of Medicine at Mount Sinai | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Completed | 18 Years | N/A | All | 16 | N/A | United States |
| 30 | NCT02943213 (ClinicalTrials.gov) | November 2016 | 21/10/2016 | Assessment of Intra-subject Variability in the Bioavailability of Chlorpromazine Hydrochloride | A Single Center, Single Dose, Open-Label, Two-Period Replicate Pilot Study to Investigate Intra-subject Variability in the Bioavailability of a Formulation Containing Chlorpromazine Hydrochloride (25 mg Sugar Coated Tablets) in at Least 16 Healthy Males and Females Under Fasting Conditions | Anti-Psychotic;Management of Manifestations of Psychotic Disorders;Treatment of Schizophrenia;Control Nausea and Vomiting;Relief of Restlessness and Apprehension Before Surgery;Acute Intermittent Porphyria;Adjunct in the Treatment of Tetanus;Control Manifestations of the Manic Type of Mani-depressive Illness;Relief of Intractable Hiccups | Drug: Chlorpromazine Hydrochloride | Cycle Pharmaceuticals Ltd. | Parexel | Completed | 18 Years | 65 Years | All | 20 | Phase 1 | South Africa |
| 31 | NCT02949830 (ClinicalTrials.gov) | October 2016 | 28/10/2016 | A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP) | A Multicenter, Open-label Extension Study to Evaluate the Long-term Safety and Clinical Activity of Subcutaneously Administered ALN-AS1 in Patients With Acute Intermittent Porphyria Who Have Completed a Previous Clinical Study With ALN-AS1 | Acute Intermittent Porphyria | Drug: givosiran (ALN-AS1) | Alnylam Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 16 | Phase 1/Phase 2 | United States;Sweden;United Kingdom |
| 32 | EUCTR2016-002638-54-GB (EUCTR) | 30/08/2016 | 18/08/2016 | Long-term Safety and Clinical Activity of ALN-AS1 in Patients with Acute Intermittent Porphyria (AIP), a rare metabolic disorder. | A Multicenter, Open-label Extension Study to Evaluate the Long-term Safety and Clinical Activity of Subcutaneously Administered ALN-AS1 in Patients with Acute Intermittent Porphyria who have Completed a Previous Clinical Study with ALN-AS1 | Acute Intermittent Porphyria (AIP) MedDRA version: 20.0;Level: LLT;Classification code 10000818;Term: Acute intermittent porphyria;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CAS# in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid) | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;United Kingdom;Sweden | ||
| 33 | NCT02922413 (ClinicalTrials.gov) | October 30, 2015 | 1/1/2016 | Panhematin for Prevention of Acute Attacks of Porphyria | Safety and Efficacy of Panhematin™ for Prevention of Acute Attacks of Porphyria | Acute Intermittent Porphyria;Hereditary Coproporphyria;Variegate Porphyria | Biological: Hemin for injection;Other: Placebo | The University of Texas Medical Branch, Galveston | NULL | Recruiting | 18 Years | N/A | All | 20 | Phase 2 | United States |
| 34 | NCT02452372 (ClinicalTrials.gov) | May 6, 2015 | 19/5/2015 | A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP) | A Phase 1, Single-ascending Dose, Multiple-ascending Dose, and Multi-dose Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN AS1 in Patients With Acute Intermittent Porphyria (AIP) | Acute Intermittent Porphyria | Drug: givosiran (ALN-AS1);Drug: Sterile Normal Saline (0.9% NaCl) | Alnylam Pharmaceuticals | NULL | Completed | 18 Years | 65 Years | All | 40 | Phase 1 | United States;Sweden;United Kingdom |
| 35 | NCT02180412 (ClinicalTrials.gov) | April 28, 2014 | 25/6/2014 | Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria | A Double-blind, Randomized, Placebo-controlled, Parallel Group Trial on the Efficacy and Safety of PanhematinTM in the Treatment of Acute Attacks of Porphyria | Acute Porphyrias | Biological: Panhematin;Other: Glucose | The University of Texas Medical Branch, Galveston | NULL | Active, not recruiting | 18 Years | 100 Years | All | 40 | Phase 2 | United States |
| 36 | NCT02935400 (ClinicalTrials.gov) | April 28, 2014 | 13/10/2016 | Acute Porphyria Biomarkers for Disease Activity | Acute Porphyrias: Biomarkers for Disease Activity and Response to Treatment | Acute Intermittent Porphyria;Hereditary Coproporphyria;Variegate Porphyria | Drug: Hemin | The University of Texas Medical Branch, Galveston | NULL | Active, not recruiting | 18 Years | N/A | All | 50 | United States | |
| 37 | NCT01605136 (ClinicalTrials.gov) | May 2012 | 22/5/2012 | Phase III Confirmatory Study in Erythropoietic Protoporphyria | A Phase III, Multicentre, Double-Blind, Randomized, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients With Erythropoietic Protoporphyria (EPP) | Erythropoietic Protoporphyria | Drug: Afamelanotide;Drug: Placebo | Clinuvel Pharmaceuticals Limited | NULL | Completed | 18 Years | N/A | All | 93 | Phase 3 | United States |
| 38 | NCT01550705 (ClinicalTrials.gov) | March 2012 | 5/3/2012 | Effect of Isoniazid on Protoporphyrin Levels in Erythropoietic Protoporphyria | Quantification of the Effects of Isoniazid Treatment on Erythrocyte and Plasma Protoporphyrin IX Concentration and Plasma Aminolevulinic Acid in Patients With Erythropoietic Protoporphyria | Erythropoietic Protoporphyria (EPP);X Linked Erythropoietic Protoporphyria | Drug: Isoniazid | University of Utah | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);Icahn School of Medicine at Mount Sinai;University of Alabama at Birmingham;University of California, San Francisco;University of Texas | Terminated | 18 Years | N/A | All | 11 | N/A | United States |
| 39 | NCT04578496 (ClinicalTrials.gov) | July 11, 2011 | 5/10/2020 | A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP) | A Phase III, Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP) | Erythropoietic Protoporphyria | Drug: Afamelanotide | Clinuvel Pharmaceuticals Limited | NULL | Completed | 18 Years | 75 Years | All | 16 | Phase 3 | NULL |
| 40 | NCT01422915 (ClinicalTrials.gov) | May 2011 | 23/8/2011 | Sorbent Therapy of the Cutaneous Porphyrias | Sorbent Therapy of the Cutaneous Porphyrias | Erythropoietic Protoporphyria | Drug: Colestipol | Brigham and Women's Hospital | NULL | Completed | 22 Years | 60 Years | All | 4 | Phase 2/Phase 3 | United States |
| 41 | NCT01284946 (ClinicalTrials.gov) | January 2011 | 26/1/2011 | Safety and Efficacy of Oral Deferasirox in Patients With Porphyria Cutanea Tarda | A Phase II, Open Label Clinical Trial Exploring the Safety and the Efficacy of Oral Deferasirox in Patients Newly Diagnosed With Porphyria Cutanea Tarda (PCT) and Non-transfusion Iron Overload | Porphyria Cutanea Tarda | Drug: Exjade | Assistance Publique - Hôpitaux de Paris | Association pour l'Etude des Fonctions Digestives (AEFD) | Recruiting | 18 Years | N/A | Both | 45 | Phase 2 | France |
| 42 | NCT01097044 (ClinicalTrials.gov) | April 2010 | 30/3/2010 | Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP) | A Phase II, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients With Erythropoietic Protoporphyria (EPP) | Erythropoietic Protoporphyria | Drug: Afamelanotide;Drug: Placebo | Clinuvel Pharmaceuticals Limited | NULL | Completed | 18 Years | N/A | All | 77 | Phase 2 | United States |
| 43 | EUCTR2009-011018-51-IE (EUCTR) | 09/03/2010 | 20/01/2010 | A Phase III, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients with Erythropoietic Protoporphyria (EPP). - Multicentre Phase III EPP Study 2 | A Phase III, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients with Erythropoietic Protoporphyria (EPP). - Multicentre Phase III EPP Study 2 | Erythropoietic Protoporphyria (EPP). MedDRA version: 12.1;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria | Product Name: afamelanotide Product Code: CUV1647 INN or Proposed INN: Afamelanotide | Clinuvel Pharmaceuticals Limited | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | Finland;United Kingdom;Netherlands;Ireland | ||
| 44 | NCT00979745 (ClinicalTrials.gov) | September 2009 | 17/9/2009 | Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP) | A Phase III, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients With Erythropoietic Protoporphyria (EPP) | Erythropoietic Protoporphyria | Drug: Afamelanotide;Drug: Placebo | Clinuvel Pharmaceuticals Limited | NULL | Completed | 18 Years | 70 Years | Both | 70 | Phase 3 | Finland;France;Germany;Ireland;Netherlands;United Kingdom |
| 45 | EUCTR2009-011018-51-FI (EUCTR) | 18/08/2009 | 20/04/2009 | A Phase III, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients with Erythropoietic Protoporphyria (EPP). - Multicentre Phase III EPP Study 2 | A Phase III, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients with Erythropoietic Protoporphyria (EPP). - Multicentre Phase III EPP Study 2 | Erythropoietic Protoporphyria (EPP) MedDRA version: 9.1;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria | Product Name: afamelanotide Product Code: CUV1647 | Clinuvel Pharmaceuticals Limited | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | Finland;United Kingdom;Netherlands;Ireland | ||
| 46 | EUCTR2009-011018-51-NL (EUCTR) | 06/08/2009 | 03/04/2009 | A Phase III, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients with Erythropoietic Protoporphyria (EPP). - Multicentre Phase III EPP Study 2 | A Phase III, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients with Erythropoietic Protoporphyria (EPP). - Multicentre Phase III EPP Study 2 | Erythropoietic Protoporphyria (EPP) MedDRA version: 9.1;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria | Product Name: afamelanotide Product Code: CUV1647 | Clinuvel Pharmaceuticals Limited | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | Finland;United Kingdom;Netherlands;Ireland | ||
| 47 | EUCTR2009-011018-51-GB (EUCTR) | 15/06/2009 | 27/04/2009 | A Phase III, Safety Extension Study in Patients withErythropoietic Protoporphyria (EPP) - Phase III EPP Safety Extension Study | A Phase III, Safety Extension Study in Patients withErythropoietic Protoporphyria (EPP) - Phase III EPP Safety Extension Study | Erythropoietic Protoporphyria (EPP) MedDRA version: 13.1;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17] | Product Name: Afamelanotide Product Code: CUV1647 INN or Proposed INN: afamelanotide Other descriptive name: Trade Name: SCENESSE | Clinuvel Pharmaceuticals Limited | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 3 | Finland;United Kingdom;Netherlands;Ireland | ||
| 48 | EUCTR2007-000636-13-NL (EUCTR) | 18/09/2008 | 07/01/2009 | A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable CUV1647 Implants in Patients with Erythropoietic Protoporphyria (EPP) - Multicentre Phase III EPP Study | A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable CUV1647 Implants in Patients with Erythropoietic Protoporphyria (EPP) - Multicentre Phase III EPP Study | Erythropoietic Protoporphyria (EPP) MedDRA version: 9.1;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria | Product Name: CUV1647 Product Code: CUV1647 | Clinuvel Pharmaceuticals Limited | NULL | Not Recruiting | Female: yes Male: yes | 80 | Phase 3 | Germany;United Kingdom;Netherlands;France;Italy;Sweden | ||
| 49 | EUCTR2007-000636-13-FR (EUCTR) | 08/07/2008 | 09/05/2008 | A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable CUV1647 Implants in Patients with Erythropoietic Protoporphyria (EPP) - Multicentre Phase III EPP Study | A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable CUV1647 Implants in Patients with Erythropoietic Protoporphyria (EPP) - Multicentre Phase III EPP Study | Erythropoietic Protoporphyria (EPP) MedDRA version: 9.1;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria | Product Name: CUV1647 Product Code: CUV1647 | Clinuvel Pharmaceuticals Limited | NULL | Not Recruiting | Female: yes Male: yes | 80 | Phase 3 | France;Netherlands;Germany;Italy;United Kingdom;Sweden | ||
| 50 | EUCTR2007-000636-13-GB (EUCTR) | 17/06/2008 | 29/02/2008 | A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable CUV1647 Implants in Patients with Erythropoietic Protoporphyria (EPP) - Multicentre Phase III EPP Study | A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable CUV1647 Implants in Patients with Erythropoietic Protoporphyria (EPP) - Multicentre Phase III EPP Study | Erythropoietic Protoporphyria (EPP) MedDRA version: 9.1;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria | Product Name: CUV1647 Product Code: CUV1647 | Clinuvel Pharmaceuticals Limited | NULL | Not Recruiting | Female: yes Male: yes | 80 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Netherlands;Germany;Italy;United Kingdom;Sweden | ||
| 51 | EUCTR2007-000636-13-SE (EUCTR) | 27/05/2008 | 03/04/2008 | A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable CUV1647 Implants in Patients with Erythropoietic Protoporphyria (EPP) - Multicentre Phase III EPP Study | A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable CUV1647 Implants in Patients with Erythropoietic Protoporphyria (EPP) - Multicentre Phase III EPP Study | Erythropoietic Protoporphyria (EPP) MedDRA version: 9.1;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria | Product Name: CUV1647 Product Code: CUV1647 | Clinuvel Pharmaceuticals Limited | NULL | Not Recruiting | Female: yes Male: yes | 80 | Phase 3 | Germany;United Kingdom;Netherlands;France;Italy;Sweden | ||
| 52 | NCT00599326 (ClinicalTrials.gov) | January 2008 | 10/1/2008 | Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda | Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda | Porphyria Cutanea Tarda | Drug: Deferasirox | University of Texas Southwestern Medical Center | Novartis Pharmaceuticals | Completed | 18 Years | N/A | All | 10 | Phase 3 | United States |
| 53 | EUCTR2007-002863-28-GB (EUCTR) | 26/09/2007 | 15/08/2007 | Investigation into the use of colestyramine as a therapy for patients with erythropoietic protoporphyria - EPP001 | Investigation into the use of colestyramine as a therapy for patients with erythropoietic protoporphyria - EPP001 | Erythropoietic protoporphyria (EPP) MedDRA version: 9.1;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria | Trade Name: Questran Product Name: Questran INN or Proposed INN: COLESTYRAMINE | Guy's & St Thomas' NHS Foundation Trust | NULL | Not Recruiting | Female: yes Male: yes | 21 | Phase 4 | United Kingdom | ||
| 54 | EUCTR2007-000636-13-IT (EUCTR) | 27/07/2007 | 15/04/2008 | A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable CUV1647 Implants in Patients with Erythropoietic Protoporphyria (EPP) - Multicentre Phase III EPP Study | A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable CUV1647 Implants in Patients with Erythropoietic Protoporphyria (EPP) - Multicentre Phase III EPP Study | Erythropoietic Protoporphyria (EPP) MedDRA version: 9.1;Level: LLT;Classification code 10036181;Term: Porphyria | Product Name: CUV1647 INN or Proposed INN: Nle4-D-Phe7-alpha-MSH | CLINUVEL PHARMACUETICALS LTD | NULL | Not Recruiting | Female: yes Male: yes | 80 | Phase 3 | Germany;United Kingdom;Netherlands;France;Italy;Sweden | ||
| 55 | NCT04053270 (ClinicalTrials.gov) | May 2007 | 8/8/2019 | Multicentre Phase III Erythropoietic Protoporphyria Study | A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutanenous Bioresorbable CUV1647 Implants in Patients With Erythropoietic Protoporphyria (EPP) | Erythropoietic Protoporphyria | Drug: Afamelanotide;Drug: Placebo | Clinuvel Pharmaceuticals Limited | NULL | Completed | 18 Years | 70 Years | All | 100 | Phase 3 | NULL |
| 56 | NCT01573754 (ClinicalTrials.gov) | March 21, 2006 | 5/4/2012 | Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda | A Prospective Comparison of Low Dose Hydroxychloroquine and Phlebotomy in the Treatment of Porphyria Cutanea Tarda. IRB 02-435 | Porphyria Cutanea Tarda | Drug: Hydroxychloroquine;Procedure: Phlebotomy | The University of Texas Medical Branch, Galveston | NULL | Active, not recruiting | 18 Years | 100 Years | All | 100 | Phase 2 | United States |
| 57 | NCT00418795 (ClinicalTrials.gov) | June 11, 2003 | 4/1/2007 | Porphozym in the Treatment of Acute Attacks in AIP | A Multi-centre, Double-blind, Randomized, Placebo-controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Porphozym (Recombinant Human Porphobilinogen Deaminase) in the Treatment of Acute Attacks in AIP | Acute Intermittent Porphyria | Drug: recombinant human porphobilinogen deaminase (Porphozym) | Zymenex A/S | NULL | Completed | 18 Years | N/A | All | 36 | Phase 2/Phase 3 | United States |
| 58 | NCT00004397 (ClinicalTrials.gov) | January 1998 | 18/10/1999 | Phase I Study of Tin Mesoporphyrin in Patients on Long Term Heme Therapy for Prevention of Acute Attacks of Porphyria | Porphyria | Drug: heme arginate;Drug: tin mesoporphyrin | National Center for Research Resources (NCRR) | University of Texas | Completed | 18 Years | N/A | Both | 20 | Phase 1 | NULL | |
| 59 | NCT00004398 (ClinicalTrials.gov) | January 1998 | 18/10/1999 | Phase I Study of Heme Arginate With or Without Tin Mesoporphyrin in Patients With Acute Attacks of Porphyria | Porphyria | Drug: heme arginate;Drug: tin mesoporphyrin | National Center for Research Resources (NCRR) | University of Texas | Completed | 18 Years | N/A | Both | 24 | Phase 1 | NULL | |
| 60 | NCT00004396 (ClinicalTrials.gov) | September 1997 | 18/10/1999 | Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute Porphyrias | Porphyria | Drug: heme arginate;Drug: tin mesoporphyrin | National Center for Research Resources (NCRR) | University of Texas | Completed | 18 Years | N/A | Both | 32 | Phase 2 | United States | |
| 61 | NCT00004831 (ClinicalTrials.gov) | October 1996 | 24/2/2000 | Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria | Erythropoietic Protoporphyria | Drug: cysteine hydrochloride | FDA Office of Orphan Products Development | St. Luke's-Roosevelt Hospital Center | Completed | 18 Years | 65 Years | Both | 20 | N/A | NULL | |
| 62 | NCT00004940 (ClinicalTrials.gov) | May 1996 | 24/2/2000 | Phase III Study of L-Cysteine in Patients With Erythropoietic Protoporphyria | Erythropoietic Protoporphyria | Drug: cysteine hydrochloride | Brigham and Women's Hospital | NULL | Completed | 18 Years | 65 Years | Both | 50 | Phase 3 | NULL | |
| 63 | NCT00004789 (ClinicalTrials.gov) | July 1993 | 24/2/2000 | Phase I/II Study of Heme Arginate and Tin Mesoporphyrin for Acute Porphyria | Porphyria | Drug: heme arginate;Drug: tin mesoporphyrin | National Center for Research Resources (NCRR) | University of Texas | Completed | 18 Years | N/A | Both | 59 | Phase 1/Phase 2 | NULL | |
| 64 | NCT00004330 (ClinicalTrials.gov) | March 1987 | 18/10/1999 | Studies in Porphyria IV: Gonadotropin-Releasing Hormone (GnRH) Analogues for Prevention of Cyclic Attacks | Porphyria | Drug: luteinizing hormone-releasing factor | National Center for Research Resources (NCRR) | University of Texas | Completed | 18 Years | 55 Years | Female | N/A | United States | ||
| 65 | EUCTR2021-001831-17-SE (EUCTR) | 11/01/2022 | An open label, long-term extension study to determine how safe and tolerable oral Dersimelagon is in subjects with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) | A Phase 3, Multicenter, Open-label, Long-term, Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) | Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 24.0;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Symptoms and general pathology [C23] | Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive name: MT-7117 | Mitsubishi Tanabe Pharma Development America (MTDA), Inc. | NULL | NA | Female: yes Male: yes | 184 | Phase 3 | United States;Canada;Spain;Australia;Norway;Germany;United Kingdom;Japan;Italy;Sweden | |||
| 66 | EUCTR2021-001831-17-DE (EUCTR) | 22/11/2021 | An open label, long-term extension study to determine how safe and tolerable oral Dersimelagon is in subjects with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) | A Phase 3, Multicenter, Open-label, Long-term, Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) | Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 24.0;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Symptoms and general pathology [C23] | Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive name: MT-7117 | Mitsubishi Tanabe Pharma Development America (MTDA), Inc. | NULL | NA | Female: yes Male: yes | 184 | Phase 3 | United States;Canada;Spain;Australia;Norway;Germany;United Kingdom;Japan;Italy;Sweden | |||
| 67 | EUCTR2019-004226-16-NO (EUCTR) | 20/05/2020 | A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents with Erythropoietic Protoporphyria or x-linked Protoporphyria | A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria - RESPITE | Erythropoietic Protoporphyria or X-Linked Protoporphyria MedDRA version: 24.0;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Symptoms and general pathology [C23] | Product Name: Dersimelagon Product Code: MT-7117 INN or Proposed INN: Dersimelagon Other descriptive name: MT-7117 | Mitsubishi Tanabe Pharma Development America (MTDA), Inc. | NULL | NA | Female: yes Male: yes | 184 | Phase 3 | United States;Canada;Finland;Spain;Australia;Germany;Norway;United Kingdom;Italy;Japan;Sweden | |||
| 68 | EUCTR2017-002432-17-FR (EUCTR) | 15/01/2018 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias - ENVISION | Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid) | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Poland;Belgium;Brazil;Australia;Denmark;South Africa;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of | |||
| 69 | EUCTR2007-000636-13-DE (EUCTR) | 12/11/2008 | A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable CUV1647 Implants in Patients with Erythropoietic Protoporphyria (EPP) - Multicentre Phase III EPP Study | A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable CUV1647 Implants in Patients with Erythropoietic Protoporphyria (EPP) - Multicentre Phase III EPP Study | Erythropoietic Protoporphyria (EPP) MedDRA version: 12.0;Level: LLT;Classification code 10015289;Term: Erythropoietic protoporphyria | Product Name: Afamelanotide Implant Product Code: CUV1647 INN or Proposed INN: afamelanotide Other descriptive name: [Nle4,D-Phe7]-a-melanocyte stimulating hormone or NDP-a-MSH | Clinuvel Pharmaceuticals Limited | NULL | Not Recruiting | Female: yes Male: yes | 80 | Phase 3 | United Kingdom;Germany;Netherlands;France;Italy;Sweden | |||
| 70 | EUCTR2017-002432-17-CZ (EUCTR) | 02/11/2017 | Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias | ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias - ENVISION | Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Porphyria acute;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10036184;Term: Porphyria hepatic;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid) | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;Italy;Switzerland;United Kingdom;France;Czech Republic;Mexico;Canada;Poland;Belgium;Brazil;Australia;Denmark;Bulgaria;Netherlands;Germany;Japan;Korea, Republic of;Sweden |