254. Porphyria
59 clinical trials, 52 drugs (DrugBank: 17 drugs), 17 drug target genes, 33 drug target pathways
Searched query = "Porphyria", "CEP", "Erythropoietic protoporphyria", "EPP", "X-linked dominant protoporphyria", "XLDP", "PCT", "HEP", "AIP", "ADP", "VP", "Hereditary coproporphyria", "HCP"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;Italy;Switzerland;United Kingdom;France;Czech Republic;Mexico;Canada;Poland;Belgium;Brazil;Denmark;Australia;South Africa;Bulgaria;Germany;Netherlands;Japan;Korea, Republic of;Sweden | ||
| 2 | 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 | Authorised-recruitment may be ongoing or finished | 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 | ||
| 3 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;Italy;Switzerland;United Kingdom;France;Czech Republic;Mexico;Canada;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Japan;Korea, Republic of;Sweden | ||
| 4 | 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: 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;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Australia;Denmark;Bulgaria;Germany;Netherlands;Japan;Sweden;Korea, Republic of | ||
| 5 | EUCTR2017-002432-17-DE (EUCTR) | 03/01/2018 | 04/10/2017 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 74 | Phase 3 | Australia;Denmark;Bulgaria;Germany;Netherlands;Japan;Sweden;Korea, Republic of;United States;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland | ||
| 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 |
| 6 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;Italy;Switzerland;United Kingdom;France;Czech Republic;Mexico;Canada;Poland;Belgium;Brazil;Denmark;Australia;South Africa;Bulgaria;Netherlands;Germany;Japan;Korea, Republic of;Sweden | ||
| 7 | 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] | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 1;Phase 2 | United States;United Kingdom;Sweden | |||
| 8 | 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 | ||
| 9 | 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 | Active, not recruiting | 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 |
| 10 | 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 | Active, not recruiting | 18 Years | N/A | All | 17 | Phase 1;Phase 2 | United States;Sweden;United Kingdom |
| 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 |
| 11 | 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] | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 1;Phase 2 | United States;United Kingdom;Sweden | |||
| 12 | 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 |
| 13 | 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 | |||
| 14 | 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 | NA | Female: yes Male: yes | 74 | Phase 3 | United States;Taiwan;Finland;Spain;Italy;Switzerland;United Kingdom;France;Czech Republic;Mexico;Canada;Poland;Belgium;Brazil;Denmark;Australia;South Africa;Bulgaria;Netherlands;Germany;Japan;Korea, Republic of;Sweden |