Clinical Trials on Beta-ketothiolase Deficiency

Total 9441 results

Vanderbilt University

Approved for marketing

Long Term Treatment With L-DOPS

Dopamine Beta Hydroxylase (DBH) Deficiency

United States
West Kazakhstan Medical University

Recruiting

Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan

Ornithine Transcarbamylase Deficiency | Biotinidase Deficiency | Citrullinemia | Glutaric Acidemia Type II | Argininosuccinic Aciduria | Maple Syrup Urine Disease | Primary Carnitine Deficiency | Homocystinuria | Carnitine Palmitoyltransferase II Deficiency | Arginase Deficiency | Very Long-chain Acyl-CoA... and other conditions

Kazakhstan
Vanderbilt University
National Heart, Lung, and Blood Institute (NHLBI)

Terminated

Treatment of Orthostatic Hypotension

Autonomic Nervous System Diseases | Orthostatic Hypotension | Orthostatic Intolerance | Dopamine Beta-Hydroxylase Deficiency

United States
Vanderbilt University Medical Center

Completed

The Pathophysiology of Orthostatic Hypotension

Autonomic Nervous System Diseases | Orthostatic Hypotension | Orthostatic Intolerance | Dopamine Beta-Hydroxylase Deficiency

United States
Assiut University

Unknown

Red Cell Distribution Width Index Versus Red Cell Distribution Width as Discriminating Guide for Iron Deficiency Anaemia and Beta Thalassemia Trait .

Microcytic Hypochromic Anemia
Newcastle-upon-Tyne Hospitals NHS Trust
Newcastle University; DSM Nutritional Products, Inc.

Completed

Effect of SNPs in the BCMO1 Enzyme (BETASNP2)

Vitamin A Deficiency | Beta-carotene Bioavailability

United Kingdom
M.D. Anderson Cancer Center

Withdrawn

Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies

Sickle Cell Disease | Sickle Beta Thalassemia | Beta Thalassemia Major | Sickle Cell-SS Disease | Sickle Beta 0 Thalassemia | Sickle Beta Plus Thalassemia

United States
Shanghai 10th People's Hospital

Completed

Related Mechanisms of RBP4 in Glycolipid Metabolism

Beta Cell Dysfunction

China
Universidad Francisco de Vitoria
University of Seville; University of Greenwich

Completed

Ingestion of Beta-alanine Effects in Well-trained Tennis Players (BETA_TENNIS)

Beta-alanine | Placebo

Spain
Celgene

Terminated

Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.

Beta Thalassemia Intermedia | Beta Thalassemia Major

France, United Kingdom, Italy, Greece
Singapore Institute for Clinical Sciences
DSM Nutritional Products, Inc.

Completed

Beta-cryptoxanthin Supplement: Absorption and Function (BETA+)

Beta-cryptoxanthin Supplementation

Singapore
Brown University
National Institute of General Medical Sciences (NIGMS)

Recruiting

Beta Events and Sensory Perception

Beta Rhythm | Tactile Perception

United States
Groupe Hospitalier Paris Saint Joseph

Recruiting

Evaluation of Pharmacokinetic / Pharmacodynamic and Tolerance Assessment of Dermal Administration of Beta-lactams in the Elderly (BETALACUTANE)

Beta-Lactams | Aged Subject

France
Hospital de Base

Unknown

The Effects of Esmolol on the Hemodynamic Response to Orotracheal Extubation

Hemodynamic Instability | Beta Blocker

Brazil
University of British Columbia

Completed

Evaluation of Low-cost Techniques for Detecting Sickle Cell Disease and β-thalassemia in Nepal and Canada

Sickle Cell Disease | Beta-Thalassemia | Sickle Cell Trait | Sickle Cell-Beta Thalassemia | Sickle Cell-SS Disease

Canada, Nepal
Leiden University Medical Center

Completed

EEG Theta-Beta Ratio Neurofeedback-training (TBR-NFT)

Elevated EEG Theta/Beta Ratio
University of Americas

Completed

Acute Supplementation With Beta-Alanine Improves Performance in Aerobic-anaerobic Transition Zones in Endurance Athletes

Beta-alanine

Chile
Children's Hospital of Philadelphia

Not yet recruiting

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

Beta-Thalassemia

United States
Bristol-Myers Squibb

Recruiting

A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.

Beta-thalassemia

China
Assiut University

Recruiting

The Prevelence of HBB c.93-21 G-A in β Thalassemia Patients

Beta-Thalassemia

Egypt
Celgene
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway...

Recruiting

Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia

Beta-Thalassemia

United States, Turkey, Italy, Thailand, Germany, Greece, China, India, Lebanon
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Active, not recruiting

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia

Hematologic Diseases | Genetic Diseases, Inborn | Hemoglobinopathies | Thalassemia | Beta-Thalassemia

United States, United Kingdom, Canada, Germany, Italy
CorrectSequence Therapeutics Co., Ltd
First Affiliated Hospital of Guangxi Medical University

Enrolling by invitation

CS-101 in Patients With β-thalassemia

Beta-Thalassemia

China
Rare Partners srl Impresa Sociale
Università degli Studi di Ferrara

Completed

Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (SIRTHALACLIN)

Beta-Thalassemia

Italy
Acceleron Pharma Inc. (a wholly owned subsidiary...

Completed

ACE-536 Extension Study - Beta Thalassemia

Beta-Thalassemia

Greece, Italy
University Hospital of Patras

Completed

Choroidal Thickness in Beta-thalassemia Patients

Beta-Thalassemia

Greece
IRCCS San Raffaele
Orchard Therapeutics; Fondazione Telethon

Unknown

Gene Therapy for Transfusion Dependent Beta-thalassemia (TIGET-BTHAL)

Beta-Thalassemia

Italy
Hoffmann-La Roche

Completed

A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent (NTD) Beta-Thalassemia

Beta-Thalassemia

Italy, Lebanon, Thailand
Prolong Pharmaceuticals

Completed

A Study of EPEG in Beta Thalassemia Patients

Beta-Thalassemia

Thailand
Shenzhen Geno-Immune Medical Institute

Unknown

Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector

Beta-Thalassemia

China
University of Washington
National Heart, Lung, and Blood Institute (NHLBI); George Papanicolaou Hospital

Completed

Evaluating the Safety of G-CSF Mobilization in Individuals With Beta Thalassemia Major

Beta-Thalassemia

Greece
Lantu Biopharma

Not yet recruiting

An Open-label Study of a Gene Therapy Product in Beta-Thalassemia

Beta-Thalassemia
bluebird bio
Center for International Blood and Marrow Transplant Research

Recruiting

A Study of Participants With β-Thalassemia Treated With Betibeglogene Autotemcel

Beta-Thalassemia

United States
Children's Hospital of Fudan University
CorrectSequence Therapeutics Co., Ltd

Recruiting

Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major

Beta-Thalassemia

China
Assiut University

Not yet recruiting

The Prevelence of IVS 1-6 (T-C) [HBB:c.92 +6 T-C] Gene Mutation in Suspected Cases of β Thalassemia in Assiut University Hospitals

Beta-Thalassemia
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Recruiting

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

Hematologic Diseases | Genetic Diseases, Inborn | Hemoglobinopathies | Thalassemia | Beta-Thalassemia

United States, Germany, Italy, United Kingdom, Canada
Assiut University

Not yet recruiting

Iron Status in BTM With Blood Transfusion

Beta-Thalassemia
CorrectSequence Therapeutics Co., Ltd
First Affiliated Hospital of Guangxi Medical University

Active, not recruiting

A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia

Beta-Thalassemia

China
bluebird bio

Completed

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

Beta-Thalassemia

United States, Germany, United Kingdom, France, Greece, Italy
bluebird bio

Completed

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

Beta-Thalassemia

France, United Kingdom, United States, Thailand, Italy, Germany
IRCCS San Raffaele
Telethon Institute for Gene Therapy (OSR-TIGET)

Active, not recruiting

Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL)

Beta Thalassaemia

Italy
Assiut University

Unknown

Dermatological Abnormalities in Beta-thalassemia Major

Beta-Thalassemia
University of Washington
National Heart, Lung, and Blood Institute (NHLBI); Genzyme, a Sanofi Company; George Papanicolaou Hospital

Completed

Evaluating the Safety and Effectiveness of Mozobil Mobilization in Adults With Beta-Thalassemia Major

Beta-Thalassemia

Greece
Boston University

Completed

Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia

Beta-Thalassemia

United States, United Kingdom
Royal Brompton & Harefield NHS Foundation Trust
CORDA, The Heart Charity; Apotex Inc.; The Cooley's Anemia Foundation,; The UK...

Unknown

Combined Chelation Treatment With Deferiprone and Deferoxamine in Thalassemia Major

Beta-Thalassemia

Italy
Aga Khan University

Completed

Anthropometric Measurements in Children Having Transfusion-dependent Beta Thalassemia

Beta Thalassemia Major
China Medical University Hospital

Completed

Clinical Trial of Deferasirox Combination Treatment With Deferiprone In Thalassaemia Patients

Beta-thalassemia Major
Assiut University

Not yet recruiting

Thrombophilia In Beta Thalassemia

Beta-Thalassemia
Boston University
Mahidol University

Completed

Study of SDMB (2,2 Dimethylbutyrate, Sodium Salt) in Beta Thalassemia Intermedia in Thailand (ST20-P2T)

Beta Thalassemia Intermedia

Thailand
Institute of Hematology & Blood Diseases Hospital
R&D Kanglin Biotech

Recruiting

Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.

Transfusion-dependent Beta-Thalassemia

China

Clinical Trials on Beta-ketothiolase Deficiency

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Macedonia

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Rare Diseases

Drug Interventions

Dietary Supplements

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