254. ポルフィリン症 Porphyria Clinical trials / Disease details


臨床試験数 : 70 薬物数 : 54 - (DrugBank : 19) / 標的遺伝子数 : 19 - 標的パスウェイ数 : 35

  
No.TrialIDDate_
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registration
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PhaseCountries
1EUCTR2021-001831-17-ES
(EUCTR)
01/03/202216/12/2021An 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
184Phase 3United States;Canada;Spain;Germany;United Kingdom;Japan
2NCT05020184
(ClinicalTrials.gov)
December 202126/7/2021Effect of Oral Cimetidine in the ProtoporphyriasEffect of Oral Cimetidine in the ProtoporphyriasErythropoietic Protoporphyria;X-linked ProtoporphyriaDrug: Cimetidine;Drug: PlaceboAmy K. Dickey, M.D.University of TexasNot yet recruiting15 YearsN/AAll20Phase 2United States
3NCT05005975
(ClinicalTrials.gov)
August 10, 202128/7/2021Extension 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;XLPDrug: MT-7117Mitsubishi Tanabe Pharma Development America, Inc.NULLRecruiting12 Years75 YearsAll175Phase 3United States;Australia;Canada;Germany;Italy;Japan;Norway;Spain;Sweden;United Kingdom
4NCT04923516
(ClinicalTrials.gov)
June 20218/6/2021Prevalence of Acute Hepatic PorphyriaPrevalence of Acute Hepatic Porphyria in Population With Suggestive Clinical PictureAcute Hepatic PorphyriaOther: Dosage of Delta-aminolevulinic acid and PorphobilinogenAssociation pour la Recherche en Medecine InterneNULLNot yet recruiting18 Years60 YearsAll500NULL
5EUCTR2019-004226-16-FI
(EUCTR)
29/04/202105/10/2020A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents with Erythropoietic Protoporphyria or x-linked ProtoporphyriaA 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.NULLNot RecruitingFemale: yes
Male: yes
159Phase 3United States;Canada;Finland;Spain;Australia;Norway;Germany;United Kingdom;Japan;Italy;Sweden
6EUCTR2019-004226-16-DE
(EUCTR)
20/04/202115/09/2020A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents with Erythropoietic Protoporphyria or x-linked ProtoporphyriaA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
184Phase 3United States;Canada;Finland;Spain;Australia;Norway;Germany;United Kingdom;Italy;Japan;Sweden
7JPRN-jRCT2071200074
02/02/202124/12/2020Expanded Access Protocol of Givosiran for Patients with Acute Hepatic PorphyriaExpanded 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 NorihisaNULLNot Recruiting>= 12age oldNot applicableBoth10N/AJapan
8EUCTR2019-004226-16-GB
(EUCTR)
04/11/202029/05/2020A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents with Erythropoietic Protoporphyria or x-linked ProtoporphyriaA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
159Phase 3United States;Canada;Finland;Spain;Australia;Norway;Germany;Japan;Italy;United Kingdom;Sweden
9EUCTR2019-004226-16-IT
(EUCTR)
08/10/202021/10/2020Study to Evaluate Efficacy, Safety, and Tolerability of MT 7117 in Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked ProtoporphyriaA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
159Phase 3United States;Canada;Finland;Spain;Australia;Norway;Germany;United Kingdom;Japan;Italy;Sweden
10EUCTR2019-004226-16-SE
(EUCTR)
06/07/202026/05/2020A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents with Erythropoietic Protoporphyria or x-linked ProtoporphyriaA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
184Phase 3United States;Canada;Finland;Spain;Australia;Norway;Germany;United Kingdom;Italy;Japan;Sweden
11NCT04402489
(ClinicalTrials.gov)
June 1, 202020/5/2020Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoetic Protoporphyria or X-Linked ProtoporphyriaA 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 ProtoporphyriaEPP;XLPDrug: Placebo;Drug: MT-7117 Low Dose;Drug: MT-7117 High DoseMitsubishi Tanabe Pharma Development America, Inc.NULLActive, not recruiting12 Years75 YearsAll184Phase 3United States;Australia;Canada;Germany;Italy;Japan;Norway;Spain;Sweden;United Kingdom;Finland
12NCT04056481
(ClinicalTrials.gov)
August 20, 201912/8/2019Expanded Access Protocol of Givosiran for Patients With Acute Hepatic PorphyriaExpanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria (AHP)Acute Hepatic PorphyriaDrug: GivosiranAlnylam PharmaceuticalsNULLApproved for marketing12 YearsN/AAllSweden;Australia;Belgium;Canada;France;Germany;Luxembourg;Netherlands;Spain;United States
13NCT03520036
(ClinicalTrials.gov)
July 5, 201823/4/2018Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic ProtoporphyriaA Phase II, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic ProtoporphyriaErythropoietic Protoporphyria (EPP)Drug: MT-7117 low dose;Drug: MT-7117 high dose;Drug: PlaceboMitsubishi Tanabe Pharma Development America, Inc.NULLCompleted18 Years75 YearsAll102Phase 2United States
14EUCTR2017-002432-17-NL
(EUCTR)
21/06/201819/02/2018Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United 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
15EUCTR2017-002432-17-FI
(EUCTR)
21/05/201830/11/2017Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Australia;Denmark;Bulgaria;Germany;Netherlands;Japan;Sweden;Korea, Republic of
16NCT03505853
(ClinicalTrials.gov)
April 26, 201813/4/2018A 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 PorphyriaDrug: Givosiran;Drug: 5-probe cocktailAlnylam PharmaceuticalsNULLCompleted18 Years65 YearsAll10Phase 1Sweden
17EUCTR2017-002432-17-PL
(EUCTR)
12/04/201816/01/2018Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United 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
18EUCTR2017-002432-17-IT
(EUCTR)
19/02/201802/02/2021Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United 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
19EUCTR2017-002432-17-BG
(EUCTR)
19/02/201809/11/2017Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Australia;Denmark;Bulgaria;Netherlands;Germany;Japan;Sweden;Korea, Republic of
20EUCTR2017-002432-17-DE
(EUCTR)
03/01/201804/10/2017ENVISION: 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.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United 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
21EUCTR2017-002432-17-BE
(EUCTR)
29/12/201719/12/2017Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United 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
22EUCTR2017-002432-17-SE
(EUCTR)
21/12/201707/11/2017Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Australia;Denmark;Bulgaria;Germany;Netherlands;Japan;Sweden;Korea, Republic of
23EUCTR2016-002638-54-SE
(EUCTR)
20/12/201709/08/2016Long-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.NULLNot RecruitingFemale: yes
Male: yes
24Phase 1;Phase 2United States;United Kingdom;Sweden
24EUCTR2017-002432-17-DK
(EUCTR)
18/12/201725/10/2017Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium;Poland;Denmark;Australia;Bulgaria;Germany;Netherlands;Japan;Sweden;Korea, Republic of
25EUCTR2017-002432-17-ES
(EUCTR)
11/12/201717/10/2017Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United 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
26EUCTR2017-002432-17-GB
(EUCTR)
22/11/201726/09/2017Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
74Phase 3United 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
27NCT03338816
(ClinicalTrials.gov)
November 16, 20177/11/2017ENVISION: 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 PorphyriasAcute Hepatic Porphyria;Acute Intermittent Porphyria;Porphyria, Acute Intermittent;Acute Porphyria;Hereditary Coproporphyria (HCP);Variegate Porphyria (VP);ALA Dehydratase Deficient Porphyria (ADP)Drug: Givosiran;Drug: PlaceboAlnylam PharmaceuticalsNULLCompleted12 YearsN/AAll94Phase 3United States;Australia;Bulgaria;Canada;Denmark;Finland;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Spain;Sweden;Taiwan;United Kingdom;Belgium;Switzerland
28NCT03118674
(ClinicalTrials.gov)
September 6, 201713/4/2017Harvoni Treatment Porphyria Cutanea TardaNewer Direct-Acting Anti-Viral Agents as Sole Therapy of Porphyria Cutanea Tarda in Subjects With Chronic Hepatitis CPorphyria Cutanea Tarda;Hepatitis CDrug: HarvoniWake Forest University Health SciencesGilead Sciences;National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);National Institutes of Health (NIH)Active, not recruiting18 YearsN/AAll25Phase 2United States
29NCT02979249
(ClinicalTrials.gov)
December 201629/11/2016Oral Iron for Erythropoietic ProtoporphyriasEffect of Oral Iron Therapy on Erythrocyte Protoporphyrin Levels in the Erythropoietic ProtoporphyriasErythropoietic Protoporphyria;EPP;X-linked Protoporphyria;XLPDrug: Oral IronIcahn School of Medicine at Mount SinaiNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Completed18 YearsN/AAll16N/AUnited States
30NCT02943213
(ClinicalTrials.gov)
November 201621/10/2016Assessment of Intra-subject Variability in the Bioavailability of Chlorpromazine HydrochlorideA 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 ConditionsAnti-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 HiccupsDrug: Chlorpromazine HydrochlorideCycle Pharmaceuticals Ltd.ParexelCompleted18 Years65 YearsAll20Phase 1South Africa
31NCT02949830
(ClinicalTrials.gov)
October 201628/10/2016A 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-AS1Acute Intermittent PorphyriaDrug: givosiran (ALN-AS1)Alnylam PharmaceuticalsNULLCompleted18 YearsN/AAll16Phase 1/Phase 2United States;Sweden;United Kingdom
32EUCTR2016-002638-54-GB
(EUCTR)
30/08/201618/08/2016Long-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.NULLAuthorised-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): noUnited States;United Kingdom;Sweden
33NCT02922413
(ClinicalTrials.gov)
October 30, 20151/1/2016Panhematin for Prevention of Acute Attacks of PorphyriaSafety and Efficacy of Panhematin™ for Prevention of Acute Attacks of PorphyriaAcute Intermittent Porphyria;Hereditary Coproporphyria;Variegate PorphyriaBiological: Hemin for injection;Other: PlaceboThe University of Texas Medical Branch, GalvestonNULLRecruiting18 YearsN/AAll20Phase 2United States
34NCT02452372
(ClinicalTrials.gov)
May 6, 201519/5/2015A 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 PorphyriaDrug: givosiran (ALN-AS1);Drug: Sterile Normal Saline (0.9% NaCl)Alnylam PharmaceuticalsNULLCompleted18 Years65 YearsAll40Phase 1United States;Sweden;United Kingdom
35NCT02180412
(ClinicalTrials.gov)
April 28, 201425/6/2014Controlled Trial of Panhematin in Treatment of Acute Attacks of PorphyriaA Double-blind, Randomized, Placebo-controlled, Parallel Group Trial on the Efficacy and Safety of PanhematinTM in the Treatment of Acute Attacks of PorphyriaAcute PorphyriasBiological: Panhematin;Other: GlucoseThe University of Texas Medical Branch, GalvestonNULLActive, not recruiting18 Years100 YearsAll40Phase 2United States
36NCT02935400
(ClinicalTrials.gov)
April 28, 201413/10/2016Acute Porphyria Biomarkers for Disease ActivityAcute Porphyrias: Biomarkers for Disease Activity and Response to TreatmentAcute Intermittent Porphyria;Hereditary Coproporphyria;Variegate PorphyriaDrug: HeminThe University of Texas Medical Branch, GalvestonNULLActive, not recruiting18 YearsN/AAll50United States
37NCT01605136
(ClinicalTrials.gov)
May 201222/5/2012Phase III Confirmatory Study in Erythropoietic ProtoporphyriaA 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 ProtoporphyriaDrug: Afamelanotide;Drug: PlaceboClinuvel Pharmaceuticals LimitedNULLCompleted18 YearsN/AAll93Phase 3United States
38NCT01550705
(ClinicalTrials.gov)
March 20125/3/2012Effect of Isoniazid on Protoporphyrin Levels in Erythropoietic ProtoporphyriaQuantification of the Effects of Isoniazid Treatment on Erythrocyte and Plasma Protoporphyrin IX Concentration and Plasma Aminolevulinic Acid in Patients With Erythropoietic ProtoporphyriaErythropoietic Protoporphyria (EPP);X Linked Erythropoietic ProtoporphyriaDrug: IsoniazidUniversity of UtahNational 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 TexasTerminated18 YearsN/AAll11N/AUnited States
39NCT04578496
(ClinicalTrials.gov)
July 11, 20115/10/2020A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)A Phase III, Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)Erythropoietic ProtoporphyriaDrug: AfamelanotideClinuvel Pharmaceuticals LimitedNULLCompleted18 Years75 YearsAll16Phase 3NULL
40NCT01422915
(ClinicalTrials.gov)
May 201123/8/2011Sorbent Therapy of the Cutaneous PorphyriasSorbent Therapy of the Cutaneous PorphyriasErythropoietic ProtoporphyriaDrug: ColestipolBrigham and Women's HospitalNULLCompleted22 Years60 YearsAll4Phase 2/Phase 3United States
41NCT01284946
(ClinicalTrials.gov)
January 201126/1/2011Safety and Efficacy of Oral Deferasirox in Patients With Porphyria Cutanea TardaA 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 OverloadPorphyria Cutanea TardaDrug: ExjadeAssistance Publique - Hôpitaux de ParisAssociation pour l'Etude des Fonctions Digestives (AEFD)Recruiting18 YearsN/ABoth45Phase 2France
42NCT01097044
(ClinicalTrials.gov)
April 201030/3/2010Phase 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 ProtoporphyriaDrug: Afamelanotide;Drug: PlaceboClinuvel Pharmaceuticals LimitedNULLCompleted18 YearsN/AAll77Phase 2United States
43EUCTR2009-011018-51-IE
(EUCTR)
09/03/201020/01/2010A 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 2A 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 LimitedNULLNot RecruitingFemale: yes
Male: yes
70Phase 3Finland;United Kingdom;Netherlands;Ireland
44NCT00979745
(ClinicalTrials.gov)
September 200917/9/2009Phase 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 ProtoporphyriaDrug: Afamelanotide;Drug: PlaceboClinuvel Pharmaceuticals LimitedNULLCompleted18 Years70 YearsBoth70Phase 3Finland;France;Germany;Ireland;Netherlands;United Kingdom
45EUCTR2009-011018-51-FI
(EUCTR)
18/08/200920/04/2009A 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 2A 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 LimitedNULLNot RecruitingFemale: yes
Male: yes
70Phase 3Finland;United Kingdom;Netherlands;Ireland
46EUCTR2009-011018-51-NL
(EUCTR)
06/08/200903/04/2009A 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 2A 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 LimitedNULLNot RecruitingFemale: yes
Male: yes
70Phase 3Finland;United Kingdom;Netherlands;Ireland
47EUCTR2009-011018-51-GB
(EUCTR)
15/06/200927/04/2009A Phase III, Safety Extension Study in Patients withErythropoietic Protoporphyria (EPP) - Phase III EPP Safety Extension StudyA 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 LimitedNULLNot RecruitingFemale: yes
Male: yes
15Phase 3Finland;United Kingdom;Netherlands;Ireland
48EUCTR2007-000636-13-NL
(EUCTR)
18/09/200807/01/2009A 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 StudyA 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 LimitedNULLNot RecruitingFemale: yes
Male: yes
80Phase 3Germany;United Kingdom;Netherlands;France;Italy;Sweden
49EUCTR2007-000636-13-FR
(EUCTR)
08/07/200809/05/2008A 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 StudyA 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 LimitedNULLNot RecruitingFemale: yes
Male: yes
80Phase 3France;Netherlands;Germany;Italy;United Kingdom;Sweden
50EUCTR2007-000636-13-GB
(EUCTR)
17/06/200829/02/2008A 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 StudyA 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 LimitedNULLNot 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): noFrance;Netherlands;Germany;Italy;United Kingdom;Sweden
51EUCTR2007-000636-13-SE
(EUCTR)
27/05/200803/04/2008A 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 StudyA 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 LimitedNULLNot RecruitingFemale: yes
Male: yes
80Phase 3Germany;United Kingdom;Netherlands;France;Italy;Sweden
52NCT00599326
(ClinicalTrials.gov)
January 200810/1/2008Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea TardaPilot Trial of Deferasirox in the Treatment of Porphyria Cutanea TardaPorphyria Cutanea TardaDrug: DeferasiroxUniversity of Texas Southwestern Medical CenterNovartis PharmaceuticalsCompleted18 YearsN/AAll10Phase 3United States
53EUCTR2007-002863-28-GB
(EUCTR)
26/09/200715/08/2007Investigation into the use of colestyramine as a therapy for patients with erythropoietic protoporphyria - EPP001Investigation 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 TrustNULLNot RecruitingFemale: yes
Male: yes
21Phase 4United Kingdom
54EUCTR2007-000636-13-IT
(EUCTR)
27/07/200715/04/2008A 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 StudyA 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 LTDNULLNot RecruitingFemale: yes
Male: yes
80Phase 3Germany;United Kingdom;Netherlands;France;Italy;Sweden
55NCT04053270
(ClinicalTrials.gov)
May 20078/8/2019Multicentre Phase III Erythropoietic Protoporphyria StudyA 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 ProtoporphyriaDrug: Afamelanotide;Drug: PlaceboClinuvel Pharmaceuticals LimitedNULLCompleted18 Years70 YearsAll100Phase 3NULL
56NCT01573754
(ClinicalTrials.gov)
March 21, 20065/4/2012Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea TardaA Prospective Comparison of Low Dose Hydroxychloroquine and Phlebotomy in the Treatment of Porphyria Cutanea Tarda. IRB 02-435Porphyria Cutanea TardaDrug: Hydroxychloroquine;Procedure: PhlebotomyThe University of Texas Medical Branch, GalvestonNULLActive, not recruiting18 Years100 YearsAll100Phase 2United States
57NCT00418795
(ClinicalTrials.gov)
June 11, 20034/1/2007Porphozym in the Treatment of Acute Attacks in AIPA 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 AIPAcute Intermittent PorphyriaDrug: recombinant human porphobilinogen deaminase (Porphozym)Zymenex A/SNULLCompleted18 YearsN/AAll36Phase 2/Phase 3United States
58NCT00004397
(ClinicalTrials.gov)
January 199818/10/1999Phase I Study of Tin Mesoporphyrin in Patients on Long Term Heme Therapy for Prevention of Acute Attacks of PorphyriaPorphyriaDrug: heme arginate;Drug: tin mesoporphyrinNational Center for Research Resources (NCRR)University of TexasCompleted18 YearsN/ABoth20Phase 1NULL
59NCT00004398
(ClinicalTrials.gov)
January 199818/10/1999Phase I Study of Heme Arginate With or Without Tin Mesoporphyrin in Patients With Acute Attacks of PorphyriaPorphyriaDrug: heme arginate;Drug: tin mesoporphyrinNational Center for Research Resources (NCRR)University of TexasCompleted18 YearsN/ABoth24Phase 1NULL
60NCT00004396
(ClinicalTrials.gov)
September 199718/10/1999Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute PorphyriasPorphyriaDrug: heme arginate;Drug: tin mesoporphyrinNational Center for Research Resources (NCRR)University of TexasCompleted18 YearsN/ABoth32Phase 2United States
61NCT00004831
(ClinicalTrials.gov)
October 199624/2/2000Study of Cysteine Hydrochloride for Erythropoietic ProtoporphyriaErythropoietic ProtoporphyriaDrug: cysteine hydrochlorideFDA Office of Orphan Products DevelopmentSt. Luke's-Roosevelt Hospital CenterCompleted18 Years65 YearsBoth20N/ANULL
62NCT00004940
(ClinicalTrials.gov)
May 199624/2/2000Phase III Study of L-Cysteine in Patients With Erythropoietic ProtoporphyriaErythropoietic ProtoporphyriaDrug: cysteine hydrochlorideBrigham and Women's HospitalNULLCompleted18 Years65 YearsBoth50Phase 3NULL
63NCT00004789
(ClinicalTrials.gov)
July 199324/2/2000Phase I/II Study of Heme Arginate and Tin Mesoporphyrin for Acute PorphyriaPorphyriaDrug: heme arginate;Drug: tin mesoporphyrinNational Center for Research Resources (NCRR)University of TexasCompleted18 YearsN/ABoth59Phase 1/Phase 2NULL
64NCT00004330
(ClinicalTrials.gov)
March 198718/10/1999Studies in Porphyria IV: Gonadotropin-Releasing Hormone (GnRH) Analogues for Prevention of Cyclic AttacksPorphyriaDrug: luteinizing hormone-releasing factorNational Center for Research Resources (NCRR)University of TexasCompleted18 Years55 YearsFemaleN/AUnited States
65EUCTR2019-004226-16-NO
(EUCTR)
20/05/2020A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents with Erythropoietic Protoporphyria or x-linked ProtoporphyriaA 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.NULLNAFemale: yes
Male: yes
184Phase 3United States;Canada;Finland;Spain;Australia;Germany;Norway;United Kingdom;Italy;Japan;Sweden
66EUCTR2017-002432-17-CZ
(EUCTR)
02/11/2017Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United 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
67EUCTR2021-001831-17-DE
(EUCTR)
22/11/2021An 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.NULLNAFemale: yes
Male: yes
184Phase 3United States;Canada;Spain;Australia;Norway;Germany;United Kingdom;Japan;Italy;Sweden
68EUCTR2021-001831-17-SE
(EUCTR)
11/01/2022An 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.NULLNAFemale: yes
Male: yes
184Phase 3United States;Canada;Spain;Australia;Norway;Germany;United Kingdom;Japan;Italy;Sweden
69EUCTR2017-002432-17-FR
(EUCTR)
15/01/2018Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: 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.NULLNot RecruitingFemale: yes
Male: yes
74Phase 3United 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
70EUCTR2007-000636-13-DE
(EUCTR)
12/11/2008A 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 StudyA 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 LimitedNULLNot RecruitingFemale: yes
Male: yes
80Phase 3United Kingdom;Germany;Netherlands;France;Italy;Sweden