Clinical Trials on Porphyrias

Total 64 results

Icahn School of Medicine at Mount Sinai

Recruiting

Longitudinal Study of the Porphyrias

Acute Porphyrias | Cutaneous Porphyrias

United States
Alnylam Pharmaceuticals

Terminated

INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP

Acute Hepatic Porphyria | Acute Intermittent Porphyria (AIP) | Hereditary Coproporphyria (HCP) | Variegate Porphyria (VP) | ALA Dehydratase Deficient Porphyria (ADP) | Hepatic Porphyrias | Porphyria Acute

United States
The University of Texas Medical Branch, Galveston

Terminated

Panhematin for Prevention of Acute Attacks of Porphyria

Hereditary Coproporphyria | Acute Intermittent Porphyria | Variegate Porphyria

United States
The University of Texas Medical Branch, Galveston

Completed

Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria

Acute Porphyrias

United States
University of California, San Francisco
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); University... and other collaborators

Completed

Clinical Diagnosis of Acute Porphyria

Acute Intermittent Porphyria (AIP) | Hereditary Coproporphyria (HCP) | Variegate Porphyria (VP)

United States
University of Texas Southwestern Medical Center
Novartis Pharmaceuticals

Completed

Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

Porphyria Cutanea Tarda

United States
Alnylam Pharmaceuticals

Completed

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

Acute Intermittent Porphyria

United States, United Kingdom, Sweden
Zymenex A/S

Completed

Porphozym in the Treatment of Acute Attacks in AIP

Acute Intermittent Porphyria

United States
Alnylam Pharmaceuticals

Completed

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

Porphyria, Acute Intermittent | Acute Intermittent Porphyria (AIP) | Acute Hepatic Porphyria (AHP) | Acute Porphyria

Sweden
Clinuvel (UK) Ltd.

Completed

Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)

Variegate Porphyria

Netherlands
The University of Texas Medical Branch, Galveston

Active, not recruiting

Acute Porphyria Biomarkers for Disease Activity

Hereditary Coproporphyria | Acute Intermittent Porphyria | Variegate Porphyria

United States
Icahn School of Medicine at Mount Sinai

Recruiting

Identification of Acute Intermittent Porphyria Modifying Genes

Acute Intermittent Porphyria (AIP)

United States
Alnylam Pharmaceuticals

No longer available

Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria

Acute Hepatic Porphyria
Alnylam Pharmaceuticals

Completed

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

Acute Hepatic Porphyria | Acute Intermittent Porphyria | Porphyria, Acute Intermittent | Acute Porphyria | Hereditary Coproporphyria (HCP) | Variegate Porphyria (VP) | ALA Dehydratase Deficient Porphyria (ADP)

United States, Spain, United Kingdom, Korea, Republic of, Australia, Bulgaria, Canada, Denmark, Finland, France, Germany, Italy, Japan, Mexico, Netherlands, Poland, Sweden, Taiwan
Nordlandssykehuset HF
University of Oslo; Norwegian University of Science and Technology; The University... and other collaborators

Recruiting

Clinical Research on Acute Intermittent Porphyria and the Use of Carbohydrate-Rich Diet as a Treatment

Porphyria, Acute Intermittent

Norway
Alnylam Pharmaceuticals

Completed

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

Acute Intermittent Porphyria

United States, United Kingdom, Sweden
Digna Biotech S.L.
University of Navarra; Porphyria Centre Sweden; UniQure N.V.; Nationales Centrum...

Completed

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

Acute Intermittent Porphyria

Spain
Digna Biotech S.L.
University of Navarra; Porphyria Centre Sweden; UniQure N.V.

Completed

Observational Study of Acute Intermittent Porphyria Patients

Acute Intermittent Porphyria

Spain
University of South Florida
University of Miami; University of Alabama at Birmingham; Icahn School of Medicine... and other collaborators

Completed

Evidence-based Assessment of Medication Sensitivity in Acute Hepatic Porphyria

Porphyrias

United States
Association pour la Recherche en Medecine Interne

Not yet recruiting

Prevalence of Acute Hepatic Porphyria (PHA)

Acute Hepatic Porphyria
Alnylam Pharmaceuticals

Recruiting

ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP) (ELEVATE)

Acute Hepatic Porphyria

Belgium, France, Germany, United Kingdom, United States, Italy, Sweden
Alnylam Pharmaceuticals

Completed

EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)

Acute Hepatic Porphyria

United States, Belgium, Bulgaria, France, Italy, Mexico, Spain, Taiwan, United Kingdom, Netherlands, Korea, Republic of, Switzerland, Czechia, Poland, Finland, Australia, Germany, Israel, Norway, South Africa
National Institute of Cardiology, Warsaw, Poland
Institute of Hematology and Transfusion Medicine, Warsaw

Completed

Acute Intermittent Porphyria Related Abnormalities in Cardiovascular System (AIPRACUS)

Porphyrias, Hepatic

Poland
University Hospital, Strasbourg, France

Completed

Risk Factors of Porphyria Cutanea Tarda (PCT)

Porphyria Cutanea Tarda
The University of Texas Medical Branch, Galveston

Completed

Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda

Porphyria Cutanea Tarda

United States
Assistance Publique - Hôpitaux de Paris
Association pour l'Etude des Fonctions Digestives (AEFD)

Unknown

Safety and Efficacy of Oral Deferasirox in Patients With Porphyria Cutanea Tarda

Porphyria Cutanea Tarda

France
National Center for Research Resources (NCRR)
University of Texas

Completed

Study of the Pathogenesis of Porphyria Cutanea Tarda

Porphyria Cutanea Tarda

United States
Nordlandssykehuset HF
The Royal Norwegian Ministry of Health

Active, not recruiting

Dental Health, Diet, Inflammation and Biomarkers in Patients With Acute Intermittent Porphyria(AIP)

Acute Intermittent Porphyria

Norway
Brigham and Women's Hospital

Completed

Sorbent Therapy of the Cutaneous Porphyrias (EPP)

Erythropoietic Protoporphyria

United States
National Center for Research Resources (NCRR)
University of Texas

Completed

Phase I Study of Heme Arginate With or Without Tin Mesoporphyrin in Patients With Acute Attacks of Porphyria

Porphyria
University of Alabama at Birmingham
Porphyria Rare Disease Clinical Research Consortium

Completed

Mitoferrin-1 Expression in Erythropoietic Protoporphyria (Porphyria Rare Disease Clinical Research Consortium (RDCRC))

Erythropoietic Protoporphyria (EPP)

United States
Wake Forest University Health Sciences
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Gilead... and other collaborators

Completed

Harvoni Treatment Porphyria Cutanea Tarda

Hepatitis C | Porphyria Cutanea Tarda

United States
Vanderbilt University Medical Center

Recruiting

Evaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia Syndrome

Postural Orthostatic Tachycardia Syndrome | Acute Hepatic Porphyria

United States
National Center for Research Resources (NCRR)
University of Texas

Completed

Phase I Study of Tin Mesoporphyrin in Patients on Long Term Heme Therapy for Prevention of Acute Attacks of Porphyria

Porphyria
National Center for Research Resources (NCRR)
University of Texas

Completed

Study of Nutritional Factors in Porphyria

Porphyria
National Center for Research Resources (NCRR)
University of Texas

Completed

Studies in Porphyria IV: Gonadotropin-Releasing Hormone (GnRH) Analogues for Prevention of Cyclic Attacks

Porphyria

United States
National Center for Research Resources (NCRR)
University of Texas

Unknown

Studies in Porphyria I: Characterization of Enzyme Defects

Porphyria

United States
National Center for Research Resources (NCRR)
University of Texas

Completed

Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute Porphyrias

Porphyria

United States
National Center for Research Resources (NCRR)
University of Texas

Completed

Phase I/II Study of Heme Arginate and Tin Mesoporphyrin for Acute Porphyria

Porphyria
Fundación Grupo de Investigación en Cuidados Intensivos...
Recordati Rare Diseases

Active, not recruiting

Colombia National Porphyria Registry

Rare Diseases | Porphyrias

Colombia
Cycle Pharmaceuticals Ltd.
Parexel

Completed

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

Acute Intermittent Porphyria | Anti-Psychotic | Management of Manifestations of Psychotic Disorders | Treatment of Schizophrenia | Control Nausea and Vomiting | Relief of Restlessness and Apprehension Before Surgery | Adjunct in the Treatment of Tetanus | Control Manifestations of the Manic Type... and other conditions

South Africa
National Center for Research Resources (NCRR)
Icahn School of Medicine at Mount Sinai

Completed

Diagnostic and Screening Study of Genetic Disorders

Metabolism, Inborn Errors | Leukodystrophy, Globoid Cell | Tay-Sachs Disease | Porphyria, Erythropoietic

United States
Disc Medicine, Inc

Enrolling by invitation

Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP

Erythropoietic Protoporphyria

United States, Australia
Clinuvel Pharmaceuticals Limited

Completed

Multicentre Phase III Erythropoietic Protoporphyria Study

Erythropoietic Protoporphyria
Bispebjerg Hospital

Completed

Light Exposure Patterns and Symptoms Among Patients With Erythropoietic Protoporphyria

Erythropoietic Protoporphyria

Denmark
Amy K. Dickey, M.D.
Wake Forest University Health Sciences; University of Texas

Recruiting

Effect of Oral Cimetidine in the Protoporphyrias

Erythropoietic Protoporphyria | X-linked Protoporphyria

United States
Mitsubishi Tanabe Pharma America Inc.

Completed

Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria

Erythropoietic Protoporphyria (EPP)

United States
FDA Office of Orphan Products Development
St. Luke's-Roosevelt Hospital Center

Completed

Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria

Erythropoietic Protoporphyria
Disc Medicine, Inc

Active, not recruiting

Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP

Erythropoietic Protoporphyria

United States
Clinuvel Pharmaceuticals Limited

Completed

Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)

Erythropoietic Protoporphyria

Netherlands, Finland, France, Germany, Ireland, United Kingdom

Clinical Trials on Porphyrias

Sponsors and Collaborators

Medical Conditions

Drug Interventions

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CROs in Burundi

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Rare Diseases

Drug Interventions

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