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Results for “Genetic Disorder”

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Enrolling by Invitation

Showing 20 of 0 results

Post-approval studies (Phase 4)Study completedNCT01519661
What this trial is testing

Long Term Safety of Tobramycin Inhalation Powder in Patients With Cystic Fibrosis

Who this might be right for
Pulmonary InfectionsPseudomonas Aeruginosa in Cystic Fibrosis
Novartis Pharmaceuticals 157
Not applicableActive Not RecruitingNCT05388370
What this trial is testing

PASS of Paediatric Patients Initiating Selumetinib

Who this might be right for
Neurofibromatosis Type 1
AstraZeneca 124
Testing effectiveness (Phase 2)Enrolling By InvitationNCT07393373
What this trial is testing

Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia

Who this might be right for
Hypochondroplasia
QED Therapeutics, a BridgeBio company 135
Not applicableStudy completedNCT01150305
What this trial is testing

Epidemiology of Non-syndromic Dominant Deafness

Who this might be right for
Hearing Impairment
Assistance Publique - Hôpitaux de Paris 183
Not applicableStudy completedNCT05047705
What this trial is testing

Investigating Distress Tolerance in Adults With Tourette Syndrome

Who this might be right for
Tourette SyndromeTic DisordersTics
Johns Hopkins University 9
Not applicableUnknownNCT06271005
What this trial is testing

Neurological Dry Eye Study Stimulating Both Lacrimal and Vagus Nerves

Who this might be right for
Dry Eye DiseaseDry Eye
Genetic Disease Investigators 75
Not applicableActive Not RecruitingNCT01633866
What this trial is testing

Pediatric Radio Frequency Coils Generic

Who this might be right for
Duchenne Muscular DystrophyMusculoskeletal Abnormalities
Children's Hospital Medical Center, Cincinnati 75
Testing effectiveness (Phase 2)UnknownNCT02722122
What this trial is testing

Study to Evaluate the Safety,Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of AIR DNase™in Patients With Cystic Fibrosis Previously Treated With Pulmozyme®

Who this might be right for
Cystic Fibrosis
Protalix 15
Not applicableStudy completedNCT00159029
What this trial is testing

Genetics of Alpha Thalassemia in Israeli Ethnic Groups

Who this might be right for
Alpha Thalassemia
Deborah Rund 100
Not applicableStudy completedNCT06582537
What this trial is testing

LAMA2 Genetic Correction

Who this might be right for
MDC1A
Maastricht University 7
Large-scale testing (Phase 3)Not Yet RecruitingNCT07184645
What this trial is testing

Trial of OPA-15406 Foam in Children and Infants With Atopic Dermatitis

Who this might be right for
Atopic Dermatitis (AD)
Otsuka Pharmaceutical Co., Ltd. 198
Not applicableActive Not RecruitingNCT05145062
What this trial is testing

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

Who this might be right for
Blood and Lymphatic Diseases
Sangamo Therapeutics 8
Testing effectiveness (Phase 2)Ended earlyNCT05116046
What this trial is testing

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia

Who this might be right for
Achondroplasia
Pfizer 35
Not applicableStudy completedNCT00047970
What this trial is testing

The Sister Study: Genetic and Environmental Risk Factors for Breast Cancer

Who this might be right for
Breast Cancer
National Institute of Environmental Health Sciences (NIEHS) 50,884
Testing effectiveness (Phase 2)Looking for participantsNCT06518005
What this trial is testing

Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome

Who this might be right for
Crigler-Najjar Syndrome
Genethon 3
Very early researchStudy completedNCT00657202
What this trial is testing

Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1

Who this might be right for
Neurofibromatosis Type 1Cutaneous Neurofibromas
Massachusetts General Hospital 11
Not applicableUnknownNCT03310502
What this trial is testing

Variation of Genes Controlling Carbohydrate and Lipid Metabolism

Who this might be right for
Diabetes Mellitus, Type 2Cardiovascular DiseasesObesity+3 more
Turku University Hospital 2,050
Not applicableStudy completedNCT00343577
What this trial is testing

Antiproteinuric Agents and Fabry Disease

Who this might be right for
Fabry DiseaseProteinuria
University of Alabama at Birmingham 12
Not applicableStudy completedNCT00252577
What this trial is testing

Genetic Predictors of Lithium Response in Bipolar Disorder

Who this might be right for
Bipolar Disorder
VA Office of Research and Development 130
Large-scale testing (Phase 3)Study completedNCT03936777
What this trial is testing

Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome

Who this might be right for
Dravet SyndromeLennox Gastaut SyndromeEpileptic Encephalopathy
Zogenix, Inc. 412
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