234. Peroxisomal disease (except Adrenoleukodystrophy)
38 clinical trials, 37 drugs (DrugBank: 12 drugs), 12 drug target genes, 42 drug target pathways
Searched query = "Peroxisomal disease (except Adrenoleukodystrophy)", "Peroxisomal disease", "Peroxisomal disorder", "Peroxisome biogenesis disorder", "PEX gene disord ... show all
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 | EUCTR2016-003134-24-NL (EUCTR) | 21/06/2018 | 28/09/2017 | Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 | A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.0;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: ALN-GO1 Product Code: ALN-GO1 INN or Proposed INN: ALN-65585 Other descriptive name: ALN-65585 | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | France;United States;United Arab Emirates;Jordan;Israel;Germany;Netherlands;United Kingdom | ||
| 2 | EUCTR2016-003134-24-DE (EUCTR) | 28/12/2017 | 11/07/2017 | Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 | A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 | 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] | Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: lumasiran Other descriptive name: ALN-65585 | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | United States;United Arab Emirates;France;Jordan;Israel;Netherlands;Germany;United Kingdom | ||
| 3 | EUCTR2016-003134-24-GB (EUCTR) | 20/12/2017 | 30/08/2017 | Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 | A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 | 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] | Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: lumasiran Other descriptive name: ALN-65585 | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | France;Israel;Netherlands;Germany;United Kingdom | ||
| 4 | EUCTR2016-003134-24-FR (EUCTR) | 09/10/2017 | 20/10/2017 | Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 | A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.0;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: ALN-GO1 Product Code: ALN-GO1 INN or Proposed INN: ALN-65585 Other descriptive name: ALN-65585 | Alnylam Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | United States;United Arab Emirates;France;Jordan;Israel;Netherlands;Germany;United Kingdom | ||
| 5 | EUCTR2015-004407-23-NL (EUCTR) | 06/06/2017 | 15/08/2016 | The First-in-Human Study of an Investigational Drug, ALN-GO1, in Healthy Adult Subjects and Patients with Primary Hyperoxaluria Type 1 Disease | A Phase 1/2, Single-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered ALN-GO1 in Healthy Adult Subjects, and Patients with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 19.0;Level: PT;Classification code 10020703;Term: Hyperoxaluria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: ALN-GO1 Product Code: ALN-GO1 INN or Proposed INN: ALN-65585 | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 1;Phase 2 | United States;France;Jordan;Israel;Germany;Netherlands;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 |
| 6 | EUCTR2015-004407-23-GB (EUCTR) | 10/02/2016 | 21/12/2015 | The First-in-Human Study of an Investigational Drug, ALN-GO1, in Healthy Adult Subjects and Patients with Primary Hyperoxaluria Type 1 Disease | A Phase 1/2, Single-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered ALN-GO1 in Healthy Adult Subjects, and Patients with Primary Hyperoxaluria Type 1 | 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] | Product Name: ALN-GO1 Product Code: ALN-GO1 INN or Proposed INN: ALN-65585 | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 52 | Phase 1;Phase 2 | United States;France;Jordan;Israel;Netherlands;Germany;United Kingdom |