Clinical trials: Acute Intermittent Porphyria (AIP)
Acute Intermittent Porphyria
A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)
The purpose of this study is to evaluate the safety and tolerability of givosiran (ALN-AS1) in AIP patients as well as to characterize pharmacokinetics (PK) and pharmacodynamics (PD) of ALN-AS1 in AIP patients.
- Trial name
- NCT02452372
- Testing
- givosiran (ALN-AS1), Sterile Normal Saline (0.9% NaCl)
- Where
- 6 sites in United States, Sweden and 1 more
- Volunteers
- 40 people
- Sponsor
- Alnylam Pharmaceuticals
Acute Hepatic Porphyria, Acute Intermittent Porphyria
ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)
The purpose of this study is to evaluate the effect of subcutaneous givosiran (ALN-AS1), compared to placebo, on the rate of porphyria attacks in patients with Acute Hepatic Porphyrias (AHP).
- Conditions
- Acute Hepatic Porphyria, Acute Intermittent Porphyria, Porphyria, Acute Intermittent, Acute Porphyria
- Trial name
- NCT03338816
- Testing
- Givosiran
- Where
- 36 sites in United States, Australia and 16 more
- Volunteers
- 94 people
- Sponsor
- Alnylam Pharmaceuticals
Acute Hepatic Porphyria
Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
The purpose of this study is to provide expanded access of givosiran to patients with Acute Hepatic Porphyria (AHP).
- Trial name
- NCT04056481
- Testing
- Givosiran
- Sponsor
- Alnylam Pharmaceuticals
Acute Intermittent Porphyria
A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)
The purpose of this study is to determine the long-term safety, tolerability and pharmacokinetics of givosiran (ALN-AS1) in AIP patients who completed study ALN-AS1-001 (NCT02452372).
- Trial name
- NCT02949830
- Testing
- Givosiran
- Where
- 5 sites in United States, Sweden and 1 more
- Volunteers
- 16 people
- Sponsor
- Alnylam Pharmaceuticals
Acute Intermittent Porphyria
Observational Study of Acute Intermittent Porphyria Patients
This is an observational prospective study that will allow evaluating the clinical and laboratory parameters evolution of at least eight patients with AIP.
- Trial name
- NCT02076763
- Where
- 2 sites in Spain
- Volunteers
- 9 people
- Sponsor
- Digna Biotech S.L.
Acute Intermittent Porphyria
Phase I Gene Therapy Clinical Trial Using the Vector rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria
This is a Phase I trial aimed to determine the safety of the investigational gene therapy product (rAAV2/5-PBGD) for the treatment of Acute Intermittent Porphyria (AIP).
- Trial name
- NCT02082860
- Testing
- rAAV2/5-PBGD vector dosage 1, rAAV2/5-PBGD vector dosage 2, rAAV2/5-PBGD vector dosage 3
- Where
- 2 sites in Spain
- Volunteers
- 8 people
- Sponsor
- Digna Biotech S.L.
Anti-Psychotic, Management of Manifestations of Psychotic Disorders
Assessment of Intra-subject Variability in the Bioavailability of Chlorpromazine Hydrochloride
Cycle Pharmaceuticals Ltd. (Cycle) is developing an oral tablet formulation of Chlorpromazine Hydrochloride and intends to conduct bioequivalence trials to demonstrate its similarity to the RLD.
- Conditions
- Anti-Psychotic, Management of Manifestations of Psychotic Disorders, Treatment of Schizophrenia, Control Nausea and Vomiting
- Trial name
- NCT02943213
- Testing
- Chlorpromazine Hydrochloride
- Where
- South Africa
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Cycle Pharmaceuticals Ltd.
Acute Hepatic Porphyria, Hepatic Porphyrias
INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP
This study will use specific diagnostic tests on a group of patients who are experiencing symptoms typical of acute hepatic porphyria (AHP) to determine how many have the condition, and to potentially help improve the diagnostic…
- Conditions
- Acute Hepatic Porphyria, Hepatic Porphyrias, Acute Intermittent Porphyria (AIP), Hereditary Coproporphyria (HCP)
- Trial name
- NCT03547297
- Where
- 12 sites in United States
- Volunteers
- 212 people
- Sponsor
- Alnylam Pharmaceuticals
Acute Intermittent Porphyria (AIP), Acute Hepatic Porphyria (AHP)
A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP)
The purpose of this study is to evaluate the effect of givosiran on the pharmacokinetics of the 5-probe cocktail of midazolam, caffeine, losartan, omeprazole, and dextromethorphan, and their metabolites, in asymptomatic patients…
- Conditions
- Acute Intermittent Porphyria (AIP), Acute Hepatic Porphyria (AHP), Porphyria, Acute Intermittent, Acute Porphyria
- Trial name
- NCT03505853
- Testing
- Givosiran, 5-probe cocktail
- Where
- Sweden
- Volunteers
- 10 people
- Sponsor
- Alnylam Pharmaceuticals
Acute Intermittent Porphyria
Porphozym in the Treatment of Acute Attacks in AIP
A 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 AIP.
- Trial name
- NCT00418795
- Testing
- recombinant human porphobilinogen deaminase (Porphozym)
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Zymenex A/S