254. ポルフィリン症 Porphyria Clinical trials / Disease details
臨床試験数 : 72 / 薬物数 : 53 - (DrugBank : 16) / 標的遺伝子数 : 19 - 標的パスウェイ数 : 35
Showing 1 to 10 of 20 diseases
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 |
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1 | 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 | Complete | >= 12age old | Not applicable | Both | 10 | N/A | Japan |
2 | 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 ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-labe ... | 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] Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Por ... | 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) Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CA ... | 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 United States;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Can ... | ||
3 | 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 ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-labe ... | 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] Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Por ... | 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) Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CA ... | 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 United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium; ... | ||
4 | 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 Study to Investigate the Interaction Between Givosiranand a 5-probe Drug Cocktail in Patients With ... | 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) A Drug-Drug Interaction Study to Investigate the Effect of Givosiranon the Pharmacokinetics (PK) of ... | Acute Intermittent Porphyria (AIP);Acute Hepatic Porphyria (AHP);Porphyria, Acute Intermittent;Acute Porphyria Acute Intermittent Porphyria (AIP);Acute Hepatic Porphyria (AHP);Porphyria, Acute Intermittent;Acute ... | Drug: Givosiran;Drug: 5-probe cocktail | Alnylam Pharmaceuticals | NULL | Completed | 18 Years | 65 Years | All | 10 | Phase 1 | Sweden |
5 | 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 ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-labe ... | 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] Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Por ... | 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) Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CA ... | 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 United States;Czechia;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Me ... | ||
6 | 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 ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-labe ... | 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] Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Por ... | 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 United States;Czechia;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Me ... | ||
7 | 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 ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-labe ... | 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] Acute Hepatic Porphyrias (AHP) MedDRA version: 24.0;Level: PT;Classification code 10036182;Term: Por ... | 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) Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CA ... | 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 United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium; ... | ||
8 | 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-labe ... | 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-labe ... | 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] Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Por ... | 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) Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CA ... | 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 United States;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Can ... | ||
9 | 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 ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-labe ... | 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] Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Por ... | 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) Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CA ... | 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 United States;Taiwan;Finland;Spain;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Can ... | ||
10 | 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 ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-labe ... | 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] Acute Hepatic Porphyrias (AHP) MedDRA version: 20.0;Level: PT;Classification code 10036182;Term: Por ... | 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) Product Name: Givosiran Product Code: ALN-AS1 INN or Proposed INN: ALN-60519 Other descriptive name: CA ... | 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 United States;Taiwan;Finland;Spain;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Belgium; ... |