Clinical Trials Logo

Porphyria, Acute Intermittent clinical trials

View clinical trials related to Porphyria, Acute Intermittent.

Filter by:
  • Completed  
  • Page 1 ·  Next »

NCT ID: NCT05882136 Completed - Porphyrias, Hepatic Clinical Trials

Acute Intermittent Porphyria Related Abnormalities in Cardiovascular System

AIPRACUS
Start date: April 5, 2019
Phase:
Study type: Observational

This study aims to assess the changes in the cardiovascular system in patients with acute intermittent porphyria (AIP).

NCT ID: NCT03505853 Completed - Clinical trials for Acute Intermittent Porphyria (AIP)

A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP)

Start date: April 26, 2018
Phase: Phase 1
Study type: Interventional

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 with Acute Intermittent Porphyria.

NCT ID: NCT03338816 Completed - Clinical trials for Acute Intermittent Porphyria

ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)

Start date: November 16, 2017
Phase: Phase 3
Study type: Interventional

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).

NCT ID: NCT02949830 Completed - Clinical trials for Acute Intermittent Porphyria

A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)

Start date: October 2016
Phase: Phase 1/Phase 2
Study type: Interventional

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).

NCT ID: NCT02943213 Completed - Clinical trials for Acute Intermittent Porphyria

Assessment of Intra-subject Variability in the Bioavailability of Chlorpromazine Hydrochloride

Start date: November 2016
Phase: Phase 1
Study type: Interventional

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. The aim of this pilot study is to investigate intrasubject variability in the bioavailability of Chlorpromazine Hydrochloride 25 mg sugar coated tablets. Cycle aims to demonstrate that Chlorpromazine Hydrochloride has a shallow dose response curve and a wide safety margin. This will then allow for the modification of bioequivalence acceptance criteria in future pivotal studies which will reduce the number of participants required whilst still maintaining assurance of safety and efficacy. Pilot Subjects (n): 20 Periods: 2 (2xR) Dosing: Single-dose Strength: 25 mg Test Product: N/A Reference: USL PHARMA Chlorpromazine Hydrochloride Analytes (in plasma): Chlorpromazine; 7-Hydroxychlorpromazine Bioequivalence based on 90% CI (Cmax, AUC): Standard; 80.00 - 125.00%

NCT ID: NCT02452372 Completed - Clinical trials for Acute Intermittent Porphyria

A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)

Start date: May 6, 2015
Phase: Phase 1
Study type: Interventional

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.

NCT ID: NCT02180412 Completed - Acute Porphyrias Clinical Trials

Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria

Start date: April 28, 2014
Phase: Phase 2
Study type: Interventional

This study aims to provide high quality evidence for the effectiveness and safety of hemin (PanhematinTM , Recordati) for treatment of acute attacks of porphyria. These types of studies have not been done before with either PanhematinTM or the hemin preparation available in Europe (NormosangTM, Orphan Europe). There are two treatment groups in this study. One group will be treated with PanhematinTM plus glucose, and the other group will be treated with glucose plus an inactive salt solution (called a "placebo"). To avoid prejudice, the treatment given to each participant will be blinded (meaning the participants and most of the hospital staff will not know which treatment the participant will receive) and randomized (meaning participants will have an equal chance of receiving either treatment, like the flip of a coin). A placebo-controlled, randomized study is the standard method used to prove treatments are effective and safe. PanhematinTM and glucose will be given in the same manner as is usual for treating an attack of porphyria. For participants who are chosen to receive the placebo, their treatment will be switched to real PanhematinTM at any time if their symptoms do not improve. This is called "rescue" treatment, and assures that they study is safe and patients who need hemin will receive it. Treatment with hemin will be for 4 days, or longer if needed. Since the study treatment is started as soon as possible after symptoms appear, there will be very little delay in providing hemin to those who need it. Funding Source - Office of Orphan Products Development (FDA OOPD)

NCT ID: NCT02082860 Completed - Clinical trials for Acute Intermittent Porphyria

Phase I Gene Therapy Clinical Trial Using the Vector rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria

Start date: November 2012
Phase: Phase 1
Study type: Interventional

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). Up to eight patients fulfilling the eligibility criteria will participate in this multicentre, open label, single dose, dose-ranging Phase I clinical trial. The enrolled patients will be followed up to assess the safety profile of the investigational gene therapy product and to establish the maximum therapeutic safe dose to be administered in future confirmatory/pivotal clinical trial(s). In addition, the biological and clinical response to the treatment with rAAV2/5-PBGD in AIP patients will be assessed. A complete evaluation of the clinical (symptoms and quality of life assessment) and laboratory (blood and urine) data will be performed.

NCT ID: NCT02076763 Completed - Clinical trials for Acute Intermittent Porphyria

Observational Study of Acute Intermittent Porphyria Patients

Start date: August 2011
Phase: N/A
Study type: Observational

This is an observational prospective study that will allow evaluating the clinical and laboratory parameters evolution of at least eight patients with AIP. This study will allow establishing a baseline for the evaluation of the eight patients that are planned to be included in a gene therapy clinical trial (AAVPBGD-AIP-001) for the AIP treatment using a rAAV5-AAT-cohPBGD expression. Patients fulfilling the study inclusion criteria will undergo a clinical and laboratory evaluation for a minimum of 6 months (with one inclusion visit, one final visit and at least two visits of follow up) up to a maximum of 24 months until their inclusion in the subsequent clinical trial. A complete evaluation of the clinical (symptoms and quality of life assessment) and laboratory (blood and urine) data will be collected.

NCT ID: NCT01568554 Completed - Clinical trials for Acute Intermittent Porphyria (AIP)

Clinical Diagnosis of Acute Porphyria

Start date: December 2011
Phase:
Study type: Observational

The purpose of this study is to test whether a focused questionnaire and laboratory tests can better define risk factors associated with possible genetic porphyria. The investigators hypothesize that the genetic carrier state of acute porphyria is distinctive enough that the Genetic Carrier Profile the investigators devise through this study will be useful in identifying carriers of genetic porphyria among the large population with undiagnosed abdominal pain.