Clinical trials: Acute Porphyria
Acute Intermittent Porphyria, Hereditary Coproporphyria
Panhematin for Prevention of Acute Attacks of Porphyria
The purpose of this study is to determine if Panhematin is safe and effective for prevention of acute attacks of porphyria. The study aims to provide high quality evidence for the use on hemin for prevention of acute attacks of…
- Conditions
- Acute Intermittent Porphyria, Hereditary Coproporphyria, Variegate Porphyria
- Trial name
- NCT02922413
- Testing
- Hemin for injection
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- The University of Texas Medical Branch, Galveston
Porphyria
Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute Porphyrias
OBJECTIVES: I. Compare the efficacy of heme arginate, singly or in combination with tin mesoporphyrin, in lowering porphyrin precursors in patients with asymptomatic acute intermittent porphyria. II.
- Trial name
- NCT00004396
- Testing
- heme arginate, tin mesoporphyrin
- Where
- 2 sites in United States
- Volunteers
- 32 people
- Sponsor
- National Center for Research Resources (NCRR)
Postural Orthostatic Tachycardia Syndrome, Acute Hepatic Porphyria
Evaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia Syndrome
Postural Tachycardia Syndrome (POTS) is the most common autonomic disorder and is estimated to affect 3,000,000 individuals in the United States, with 80-85% of patients being women.
- Trial name
- NCT05344599
- Testing
- Autonomic Function Testing, Genetic Testing, Urine Testing
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Vanderbilt University Medical Center
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 Intermittent Porphyria (AIP)
Identification of Acute Intermittent Porphyria Modifying Genes
This study proposes to identify the predisposing/protective modifying genes that underlie the acute attacks in symptomatic patients with Acute Intermittent Porphyria (AIP), an autosomal dominant inborn error of heme biosynthesis.
- Trial name
- NCT05502133
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Acute Hepatic Porphyria
EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)
The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks.
- Trial name
- NCT02240784
- Where
- 30 sites in United States, Australia and 18 more
- Volunteers
- 136 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
Porphyria
Phase I Study of Heme Arginate With or Without Tin Mesoporphyrin in Patients With Acute Attacks of Porphyria
OBJECTIVES: I. Evaluate the efficacy of tin mesoporphyrin in patients with acute porphyria attacks who are also treated with a standard course of heme arginate. II.
- Trial name
- NCT00004398
- Testing
- heme arginate, tin mesoporphyrin
- Volunteers
- 24 people
- Sponsor
- National Center for Research Resources (NCRR)
Acute Porphyrias, Cutaneous Porphyrias
Longitudinal Study of the Porphyrias
The objective of this protocol is to conduct a longitudinal multidisciplinary investigation of the human porphyrias including the natural history, morbidity, pregnancy outcomes, and mortality in people with these disorders.
- Trial name
- NCT01561157
- Where
- 16 sites in United States
- Volunteers
- 1,500 people
- Sponsor
- The American Porphyrias Expert Collaborative
Porphyria, Acute Intermittent
Clinical Research on Acute Intermittent Porphyria and the Use of Carbohydrate-Rich Diet as a Treatment
The main aim of this clinical trial is to learn about the effect of carbohydrate-rich diet as a treatment for AIP (acute intermittent porphyria).
- Trial name
- NCT06273644
- Testing
- Carbohydrates
- Where
- 3 sites in Norway, Sweden
- Volunteers
- 50 people
- Sponsor
- Nordlandssykehuset HF
Acute Hepatic Porphyria
Prevalence of Acute Hepatic Porphyria
To determine the proportion of patients suffering from acute hepatic porphyria (AHP) from different hospital departments and referred to an internist referent for a suggestive clinical picture with a first negative etiological…
- Trial name
- PHA (NCT04923516)
- Testing
- Dosage of Delta-aminolevulinic acid and Porphobilinogen
- Where
- France
- Volunteers
- 150 people
- Sponsor
- Association pour la Recherche en Medecine Interne
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.
Porphyria
Phase I/II Study of Heme Arginate and Tin Mesoporphyrin for Acute Porphyria
OBJECTIVES: I. Evaluate the efficacy and safety of heme arginate in the treatment of 20 patients with acute attacks of porphyria. II.
- Trial name
- NCT00004789
- Testing
- heme arginate, tin mesoporphyrin
- Volunteers
- 59 people
- Sponsor
- National Center for Research Resources (NCRR)
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
Porphyria
Phase I Study of Tin Mesoporphyrin in Patients on Long Term Heme Therapy for Prevention of Acute Attacks of Porphyria
OBJECTIVES: I. Evaluate the effectiveness of tin mesoporphyrin in reducing the number of heme infusions needed to prevent acute attacks of porphyria. II. Evaluate the safety and tolerability of tin mesoporphyrin in these patients.
- Trial name
- NCT00004397
- Testing
- heme arginate, tin mesoporphyrin
- Volunteers
- 20 people
- Sponsor
- National Center for Research Resources (NCRR)
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.
Acute Porphyrias
Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria
This study aims to provide high quality evidence for the effectiveness and safety of hemin (PanhematinTM , Recordati) for treatment of acute attacks of porphyria.
- Trial name
- NCT02180412
- Testing
- Panhematin, Glucose
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- The University of Texas Medical Branch, Galveston
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 Intermittent Porphyria, Hereditary Coproporphyria
Acute Porphyria Biomarkers for Disease Activity
The long term objective of the research is to identify new biomarkers of disease activity in the human acute porphyrias. This pilot study is intended to provide pilot and feasibility data needed to plan larger and more definitive…
- Conditions
- Acute Intermittent Porphyria, Hereditary Coproporphyria, Variegate Porphyria
- Trial name
- NCT02935400
- Testing
- Hemin
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- The University of Texas Medical Branch, Galveston
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