234. Peroxisomal disease (except Adrenoleukodystrophy) Clinical trials / Disease details
Clinical trials : 39 / Drugs : 35 - (DrugBank : 12) / Drug target genes : 13 - Drug target pathways : 45
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 | NCT04125472 (ClinicalTrials.gov) | June 2, 2021 | 10/10/2019 | Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1 | Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria | Drug: Lumasiran | Alnylam Pharmaceuticals | NULL | Approved for marketing | N/A | N/A | All | Belgium;Canada;France;French Polynesia;Germany;Italy;Netherlands;Spain;Switzerland;United Kingdom;United States Belgium;Canada;France;French Polynesia;Germany;Italy;Netherlands;Spain;Switzerland;United Kingdom;Un ... | ||
2 | EUCTR2019-001346-17-IT (EUCTR) | 14/05/2021 | 07/06/2021 | A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hyperoxaluria Type 1 A Phase 3 study of an Investigational Drug, Lumasiran(ALN-GO1) in patients with advanced primary hyp ... | ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1) - ILLUMINATE-C ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamic ... | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: ... | Product Name: lumasiran Product Code: [ALN-GO1] INN or Proposed INN: Lumasiran | ALNYLAM PHARMACEUTICALS, INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 3 | United Arab Emirates;United States;Lebanon;Turkey;Israel;United Kingdom;Switzerland;Italy;India;Egypt;France;Jordan;Belgium;Australia;Netherlands;Germany United Arab Emirates;United States;Lebanon;Turkey;Israel;United Kingdom;Switzerland;Italy;India;Egyp ... | ||
3 | EUCTR2019-001346-17-GB (EUCTR) | 22/01/2020 | 23/07/2019 | A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hyperoxaluria Type 1 A Phase 3 study of an Investigational Drug, Lumasiran(ALN-GO1) in patients with advanced primary hyp ... | ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1) ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamic ... | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: ... | Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: Lumasiran | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 16 | Phase 3 | United Arab Emirates;United States;Lebanon;Turkey;Israel;United Kingdom;Switzerland;India;Egypt;France;Jordan;Belgium;Australia;Netherlands;Germany United Arab Emirates;United States;Lebanon;Turkey;Israel;United Kingdom;Switzerland;India;Egypt;Fran ... | ||
4 | NCT04152200 (ClinicalTrials.gov) | January 21, 2020 | 31/10/2019 | A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 | ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (PH1) ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamic ... | Primary Hyperoxaluria Type 1;Primary Hyperoxaluria | Drug: Lumasiran | Alnylam Pharmaceuticals | NULL | Active, not recruiting | N/A | N/A | All | 21 | Phase 3 | United States;Australia;Belgium;France;Israel;Italy;Jordan;Lebanon;Netherlands;Turkey;United Arab Emirates;Switzerland;United Kingdom United States;Australia;Belgium;France;Israel;Italy;Jordan;Lebanon;Netherlands;Turkey;United Arab Em ... |
5 | EUCTR2019-001346-17-NL (EUCTR) | 05/12/2019 | 22/08/2019 | A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hyperoxaluria Type 1 A Phase 3 study of an Investigational Drug, Lumasiran(ALN-GO1) in patients with advanced primary hyp ... | ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1) ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamic ... | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: ... | Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: LUMASIRAN | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 16 | Phase 3 | United States;United Arab Emirates;Lebanon;Turkey;Israel;Switzerland;United Kingdom;India;Egypt;France;Jordan;Belgium;Australia;Netherlands;Germany United States;United Arab Emirates;Lebanon;Turkey;Israel;Switzerland;United Kingdom;India;Egypt;Fran ... | ||
6 | EUCTR2019-001346-17-BE (EUCTR) | 07/10/2019 | 21/08/2019 | A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hyperoxaluria Type 1 A Phase 3 study of an Investigational Drug, Lumasiran(ALN-GO1) in patients with advanced primary hyp ... | ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1) ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamic ... | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: ... | Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: LUMASIRAN | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 16 | Phase 3 | United States;Egypt;United Kingdom;United Arab Emirates;Switzerland;India;Lebanon;Netherlands;Turkey;Belgium;Italy;Israel;Australia;France;Jordan;Germany United States;Egypt;United Kingdom;United Arab Emirates;Switzerland;India;Lebanon;Netherlands;Turkey ... | ||
7 | EUCTR2018-004014-17-DE (EUCTR) | 23/04/2019 | 27/12/2018 | A Study of an Investigational Drug, Lumasiran (ALN-GO1) in Infants and Young Children with Primary Hyperoxaluria Type 1 A Study of an Investigational Drug, Lumasiran(ALN-GO1) in Infants and Young Children with Primary Hy ... | ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children with Primary Hyperoxaluria Type 1 ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacody ... | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: ... | Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: LUMASIRAN | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 3 | France;United States;Israel;Germany;United Kingdom | ||
8 | NCT03905694 (ClinicalTrials.gov) | April 22, 2019 | 1/4/2019 | A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1 | ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1 ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacody ... | Primary Hyperoxaluria;Primary Hyperoxaluria Type 1 (PH1) | Drug: Lumasiran | Alnylam Pharmaceuticals | NULL | Active, not recruiting | 0 Years | 5 Years | All | 18 | Phase 3 | United States;France;Germany;Israel;United Kingdom |
9 | EUCTR2018-001981-40-NL (EUCTR) | 21/03/2019 | 21/01/2019 | A Phase 3 Study of an Investigational Drug, Lumasiran (ALN-GO1) with an Extended Dosing Period in Children and Adults with Primary Hyperoxaluria Type 1 Disease A Phase 3 Study of an Investigational Drug, Lumasiran(ALN-GO1) with an Extended Dosing Period in Chi ... | ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study with an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults with Primary Hyperoxaluria Type 1 ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study with an Extended Dosing P ... | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: ... | Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: lumasiran | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 3 | France;United States;United Arab Emirates;Jordan;Israel;Germany;Netherlands;United Kingdom;Japan;Switzerland France;United States;United Arab Emirates;Jordan;Israel;Germany;Netherlands;United Kingdom;Japan;Swi ... | ||
10 | EUCTR2018-004014-17-GB (EUCTR) | 01/03/2019 | 19/12/2018 | A Study of an Investigational Drug, Lumasiran (ALN-GO1) in Infants and Young Children with Primary Hyperoxaluria Type 1 A Study of an Investigational Drug, Lumasiran(ALN-GO1) in Infants and Young Children with Primary Hy ... | ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children with Primary Hyperoxaluria Type 1 ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacody ... | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: ... | Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: LUMASIRAN | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | France;United States;Israel;Germany;United Kingdom |