Search clinical trials for: Metabolism, Inborn

Total 14047 results

SpliSense Ltd.

Recruiting

Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients With Cystic Fibrosis

Cystic Fibrosis

United States
University of North Carolina, Chapel Hill
North Carolina Translational and Clinical Sciences Institute

Not yet recruiting

PeLear CCC: Proyecto Latino Contra Cancer Colorectal (PeLear CCC)

Colorectal Cancer | Rectal Cancer | Rectal Neoplasms | Colon Cancer | Colon Adenocarcinoma | Colon Rectal Cancer | Hereditary Nonpolyposis Colon Cancer

United States
University of Cologne
The Clinical Trials Centre Cologne; ESCRS (European Society of Cataract and...

Not yet recruiting

Trial Comparing Cataract Surgery With Triple-DMEK in Patients With Cataract and Fuchs Endothelial Corneal Dystrophy

Cataract Surgery | Cataract and Fuchs Endothelial Corneal Dystrophy

Denmark, Germany, Netherlands, Spain
University of Mons

Recruiting

Quantitative Assessment of Orofacial Muscle Function in FSHD

Facioscapulohumeral Muscular Dystrophy

Belgium
Amryt Pharma
Chiesi Farmaceutici S.p.A.

Not yet recruiting

A Study to Assess the Incidence of Skin Cancers in Patients With Epidermolysis Bullosa Receiving Filsuvez (FOSteR)

Epidermolysis Bullosa, Junctional | Epidermolysis Bullosa, Dystrophic
Takeda

Recruiting

A Study Evaluating Soticlestat in Participants With Dravet Syndrome or Lennox-Gastaut Syndrome Who Have Been Exposed to Fenfluramine

Dravet Syndrome (DS) | Lennox-Gastaut Syndrome (LGS)

United Kingdom, Denmark
Rocket Pharmaceuticals Inc.

Not yet recruiting

Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

Pyruvate Kinase Deficiency

Spain, United States
AbbVie

Not yet recruiting

An Observational Study to Assess Real-World Use of Upadacitinib Tablets in Adolescent and Adult Participants With Moderate to Severe Atopic Dermatitis in China (REACH-AD)

Atopic Dermatitis
Leiden University Medical Center
The Dutch Brain Foundation (funding)

Not yet recruiting

Stimulating Amyloid Clearance in Cerebral Amyloid Angiopathy (Clear-Brain)

Cerebral Amyloid Angiopathy
Regeneron Pharmaceuticals

Not yet recruiting

A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body (FERVENT-1)

Non-transfusion Dependent Beta-thalassemia (NTDT)
Instituto Nacional de Investigacao em Saude, Angola
FUNDAÇAO CALOUSTE GULBENKIAN; FUNDACIÓN BANCARIA CAIXA D'ESTALVIS I PENSIONS... and other collaborators

Recruiting

LEARNER- Low dosE AspiRiN prEterm tRial (Angola) (LEARNER)

Pregnancy Complications | Pregnancy Related | Sickle Cell Disease | Pre-Eclampsia

Angola
Sanofi
Regeneron Pharmaceuticals

Not yet recruiting

Study in Paediatric Patients With Atopic Dermatitis Treated With Dupilumab in Spain (PROADAP)

Atopic Dermatitis
AstraZeneca

Recruiting

Hereditary Transthyretin Amyloidosis Polyneuropathy in Patients With Carpal Tunnel Syndrome in Russia (LOCUS)

Carpal Tunnel Syndrome | Amyloidosis | Polyneuropathy | Hereditary Transthyretin Amyloidosis

Russian Federation
Ultragenyx Pharmaceutical Inc

Not yet recruiting

Long-term Extension of GTX-102 in Angelman Syndrome

Angelman Syndrome

United States, Australia, Canada, Germany, United Kingdom, Israel, France, Spain
Poitiers University Hospital

Recruiting

Study to Evaluate Music Therapy on Irritability and Impulsivity in Patients With Huntington's Disease (MUSIC-HD) (MUSIC-HD)

Huntington Disease

France
Jiangsu HengRui Medicine Co., Ltd.

Not yet recruiting

Clinical Trial of HR091506 Tablets in Treatment of Gout With Hyperuricemia in Adults

Gout With Hyperuricemia in Adults
Spinogenix
Avance Clinical Pty Ltd.

Not yet recruiting

Study of SPG601 in Adult Men With Fragile X Syndrome

Fragile X Syndrome

United States
Children's Hospital Los Angeles

Not yet recruiting

Maralixibat in Patients With Cystic Fibrosis and Constipation, A Within-Subjects Pilot Study

Cystic Fibrosis | Constipation Chronic Idiopathic

United States
IRCCS Ospedale San Raffaele

Not yet recruiting

Validation of Human Drugs Target of Repurposed Drugs and Novel Therapies

Rare Diseases | Corneal Neovascularization | Limbal Stem Cell Deficiency | Aniridia | Neurotrophic Keratopathy | Ocular Cicatricial Pemphigoid | Ocular Graft-versus-host Disease | EEC Syndrome
Ospedale San Raffaele
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Recruiting

Identification of New Biological Markers for the Progression of Mycobacterium Abscessus-induced Lung Disease in Cystic Fibrosis (BioMol-MA)

Non-Tuberculous Mycobacterial Pneumonia | Cystic Fibrosis Lung

Italy
All India Institute of Medical Sciences, Bhubaneswar

Not yet recruiting

Study on Efficacy of Add on Selenium in Mild-to-moderate Graves Ophthalmopathy

Thyroid Eye Disease | Graves Ophthalmopathy

India
Avidity Biosciences, Inc.

Not yet recruiting

Global Study of Del-desiran for the Treatment of DM1 (HARBOR)

Myotonic Dystrophy | Myotonic Disorders | Myotonia | Myotonic Dystrophy Type 1 (DM1) | Myotonic Dystrophy 1 | Myotonic Muscular Dystrophy | DM1 | Dystrophy Myotonic | Steinert Disease | Steinert
QED Therapeutics, Inc.

Recruiting

Prospective Clinical Assessment Study in Children With Hypochondroplasia (HCH)

Hypochondroplasia

United States, Australia, Norway, Portugal, Singapore, Spain
Nido Biosciences, Inc.

Recruiting

A Study of NIDO-361 in Patients With SBMA (PIONEER KD)

Spinal and Bulbar Muscular Atrophy | Kennedy's Disease

Korea, Republic of, Denmark, Italy, United Kingdom
Johns Hopkins University
National Center for Complementary and Integrative Health (NCCIH); Bowdoin College

Recruiting

Remote Delivery of a Mindfulness-based Intervention for Tics (MBIT)

Tourette Syndrome | Tic Disorders | Tourette's Disorder | Persistent Tic Disorder | Chronic Motor Tic Disorder | Chronic Vocal Tic Disorder | Persistent Motor Tic Disorder | Persistent Vocal Tic Disorder

United States
Riphah International University

Not yet recruiting

Effects of Lower Limb Fitness Program in Children With DS

Down Syndrome

Pakistan
Sanofi

Recruiting

A Study to Evaluate the Treatment Response and Safety of Two Dose Regimens of Subcutaneous Amlitelimab Monotherapy Compared With Treatment Withdrawal in Participants Aged 12 Years and Older With Moderate-to-severe Atopic Dermatitis (ESTUARY)

Dermatitis Atopic

United States
Wave Life Sciences Ltd.

Not yet recruiting

A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD (RestorAATion-2)

Alpha-1 Antitrypsin Deficiency
Tisento Therapeutics

Not yet recruiting

A Phase 2b Study of Zagociguat in Patients With MELAS (PRIZM)

Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)

United States, Australia, Italy, Germany, Canada, United Kingdom
Başak Çağla Arslan
Hacettepe University

Recruiting

Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy | Occupational Therapy | Gamification

Turkey
Amgen

Not yet recruiting

A Study of AMG 732 in Healthy Participants and Participants With Thyroid Eye Disease

Thyroid Eye Disease
Assiut University

Not yet recruiting

The Role of Muscle Ultrasound in Assessment of Sample of Patients With Limb-girdle Muscular Dystrophy

Limb-girdle Muscular Dystrophy
Children's Oncology Group

Not yet recruiting

A Study Using Risk Factors to Determine Treatment for Children With Favorable Histology Wilms Tumors (FHWT)

Stage I Kidney Wilms Tumor | Stage II Kidney Wilms Tumor | Stage III Kidney Wilms Tumor | Stage IV Kidney Wilms Tumor
Istanbul University - Cerrahpasa (IUC)

Not yet recruiting

Comparing Efficacies of Median Nerve Hydrodissection With Dexamethasone and Dextrose in Carpal Tunnel Syndrome

Carpal Tunnel Syndrome | Entrapment Neuropathy | Median Nerve Entrapment

Turkey
Boston Children's Hospital

Not yet recruiting

Effect of S. Aureus Skin Decolonization on Disease Severity in Atopic Dermatitis Patients

Atopic Dermatitis
Groupe Hospitalier de la Region de Mulhouse et...

Not yet recruiting

Frequency-time Analysis of Pathological Lung Sounds: Detection and Quantification of Pathological Sounds in Patients With Cystic Fibrosis, Pulmonary Fibrosis or COPD (Chronic Obstructive Pulmonary Disease) (ATF/SPP)

Chronic Obstructive Pulmonary Disease | Pulmonary Fibrosis | Cystic Fibrosis
Essen Biotech

Not yet recruiting

Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease (BAH243)

Sickle Cell Disease | Sickle-Cell Disease With Crisis

China
CSL Behring

Not yet recruiting

Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy

Hemophilia B
Oneness Biotech Co., Ltd.

Not yet recruiting

Evaluate Efficacy, Pharmacokinetics, and Safety of Repeat Subcutaneous Doses of FB825 in Adults With Moderate-to-Severe Atopic Dermatitis

Atopic Dermatitis
Takeda

Not yet recruiting

A Study on Dravet Syndrome (DS) and Lennox-Gastaut Syndrome (LGS) in Children, Teenagers and Adults in Portugal (DRALEGA-PT)

Dravet Syndrome (DS) | Lennox-Gastaut Syndrome (LGS)
Academisch Medisch Centrum - Universiteit van Amsterdam...

Recruiting

The Role of Red Cell Characteristics, Angiogenesis, Viscosity and Oxygenation in the Pathophysiology of Sickle Cell Related Retinopathy (RAVOS)

Sickle Cell Disease | Sickle Cell SC Disease | Sickle Cell-SS Disease | Sickle Cell Retinopathy

Netherlands
Vanderbilt University Medical Center
Aminu Kano Teaching Hospital

Not yet recruiting

Prevention of Malnutrition in Children With Sickle Cell Disease (PMC-SCD)

Malnutrition | Sickle Cell Disease
Institute of Liver and Biliary Sciences, India

Recruiting

'Glycogen Storage Diseases (GSDs) in Indian Children- Establishing an Indian GSD (I-GSD) Registry' (I-GSD)

Glycogen Storage Diseases

India
Steminent US, Inc.

Not yet recruiting

Stemchymal® for Polyglutamine Spinocerebellar Ataxia

Spinocerebellar Ataxias
Apogee Therapeutics, Inc.

Recruiting

A Study Evaluating APG777 in Atopic Dermatitis

Atopic Dermatitis

United States, Canada
Alnylam Pharmaceuticals

Recruiting

A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy (cAPPricorn-1)

Cerebral Amyloid Angiopathy

Canada
Janjira Petsuksiri
Mahidol University

Not yet recruiting

Orbital Radiotherapy in Grave's Ophthalmopathy 1 Week vs 2 Weeks (OraGO-1 Trial)

Thyroid Associated Ophthalmopathy

Thailand
Natural Resources Institute Finland
Tampere University

Recruiting

Sand Play - the Effect of Biodiversity Exposure on Atopic Dermatitis (BIWE)

Microbial Colonization | Atopic Dermatitis | Immune System and Related Disorders | Nature, Human

Finland
Peking University First Hospital

Recruiting

Establishment of a Multidimensional Prediction Model for the Natural Course of VHL Disease-related Renal Cell Carcinoma

Renal Cell Carcinoma | VHL Syndrome

China
Peking Union Medical College Hospital

Not yet recruiting

A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy. (GEN6050XIIT)

Duchenne Muscular Dystrophy (DMD)

Search clinical trials for: Metabolism, Inborn

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