254. Porphyria Clinical trials / Disease details
Clinical trials : 72 / Drugs : 53 - (DrugBank : 16) / Drug target genes : 19 - Drug target pathways : 35
| 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 | 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 | Completed | 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 |
| 2 | 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 | Completed | 18 Years | N/A | All | 16 | Phase 1/Phase 2 | United States;Sweden;United Kingdom |
| 3 | 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 |