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Results for “Genetic Disease, Inborn”

16,950 trials

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Showing 20 of 16,950 results

Early research (Phase 1)Study completedNCT02919800
What this trial is testing

A Single-dose, Dose-escalation Study of a Long-acting MOD-5014 in Healthy Adult Male

Who this might be right for
Hemophilia A or B With Inhibitors
OPKO Health, Inc. 32
Not applicableStudy completedNCT00026000
What this trial is testing

Brain Activation in Vocal and Motor Tics

Who this might be right for
Tourette's SyndromeChronic Motor Tic DisorderChronic Motor Vocal Disorder
National Institute of Neurological Disorders and Stroke (NINDS) 30
Not applicableLooking for participantsNCT01601171
What this trial is testing

Genetics of Reproductive Disorders (Including Kallmann Syndrome) and Cleft Lip and/or Palate

Who this might be right for
Kallmann SyndromeHypogonadotropic HypogonadismHypothalamic Amenorrhea+5 more
Centre Hospitalier Universitaire Vaudois 2,000
Early research (Phase 1)Looking for participantsNCT06298071
What this trial is testing

A Phase Ib Clinical Trial of Peguricase for Injection With Methotrexate in Patients With Uncontrolled Gout.

Who this might be right for
Gout
Shanghai Institute Of Biological Products 36
Testing effectiveness (Phase 2)Active Not RecruitingNCT03326336
What this trial is testing

Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa

Who this might be right for
Non-syndromic Retinitis Pigmentosa
GenSight Biologics 10
Large-scale testing (Phase 3)Ended earlyNCT01794000
What this trial is testing

Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD)

Who this might be right for
Sickle Cell Disease
Eli Lilly and Company 341
Not applicableStudy completedNCT04198207
What this trial is testing

The Effectiveness and Safety of Vagus Nerve Stimulation for TRE

Who this might be right for
Tuberous Sclerosis ComplexEpilepsy
Beijing Children's Hospital 70
Not applicableStudy completedNCT01632111
What this trial is testing

Pulmonary Involvement in Patients With Fabry Disease

Who this might be right for
Fabry Disease
University of Zurich 110
Not applicableStudy completedNCT07756944
What this trial is testing

Effects of Bubble Cushion Exercises on Postural Control in Children With Down Syndrome

Who this might be right for
Down Syndrome
Riphah International University 22
Not applicableEnrolling By InvitationNCT04579211
What this trial is testing

Prospective Analysis of Urine LAM to Eliminate NTM Sputum Screening

Who this might be right for
Cystic Fibrosis
Jerry A. Nick, M.D. 220
Large-scale testing (Phase 3)Study completedNCT01305200
What this trial is testing

Supersaturated Calcium Phosphate Rinse in Preventing Oral Mucositis in Young Patients Undergoing Autologous or Donor Stem Cell Transplant

Who this might be right for
Childhood Acute Lymphoblastic Leukemia in RemissionChildhood Acute Myeloid Leukemia in RemissionChildhood Chronic Myelogenous Leukemia+24 more
Children's Oncology Group 226
Not applicableStudy completedNCT05971836
What this trial is testing

The Molecular Basis of Inherited Reproductive Disorders

Who this might be right for
Hypogonadotropic HypogonadismReproductive DisorderKallmann Syndrome+1 more
Stephanie B. Seminara, MD 854
Not applicableLooking for participantsNCT04982744
What this trial is testing

Registry of Li Fraumeni and Li Fraumeni Like Syndromes

Who this might be right for
Li-Fraumeni SyndromeLi-Fraumeni-Like Syndrome
Istituto Ortopedico Rizzoli 200
Not applicableStudy completedNCT04809207
What this trial is testing

Acceptability, Feasibility and Preliminary Efficacy of CFWP to Treat Clinically Elevated Fatigue in Adults With CF

Who this might be right for
Cystic FibrosisFatigue
Johns Hopkins University 40
Testing effectiveness (Phase 2)WithdrawnNCT05471492
What this trial is testing

Study to Evaluate the Efficacy and Safety of PF-06480605 in Adult Participants With Moderate to Severe Crohn's Disease

Who this might be right for
Crohn Disease
Telavant, Inc.
Not applicableStudy completedNCT03845400
What this trial is testing

Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II in North America

Who this might be right for
Hereditary Angioedema (HAE)
Shire 168
Not applicableStudy completedNCT02100371
What this trial is testing

Study of BMS-833923 in Two Specific Patients With Basal Cell Nevus Syndrome

Who this might be right for
Basal Cell Nevus Syndrome
University Health Network, Toronto 2
Testing effectiveness (Phase 2)Not Yet RecruitingNCT06487000
What this trial is testing

Determine the Dose-dependent Efficacy and Safety of EC-18 in Patients With Moderate to Severe Atopic Dermatitis

Who this might be right for
Atopic Dermatitis
Enzychem Lifesciences Corporation 150
Early research (Phase 1)Study completedNCT02251600
What this trial is testing

A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
PTC Therapeutics 24
Testing effectiveness (Phase 2)Study completedNCT01761292
What this trial is testing

Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD

Who this might be right for
Duchenne Muscular Dystrophy (DMD)
Italfarmaco 20
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