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Results for “Recessive”

266 trials

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Showing 20 of 266 results

Not applicableUnknownNCT05116839
What this trial is testing

Pressure Support Ventilation Versus Continuous Positive Air Way Pressure (CPAP) Using I Gelin Adult Patients,

Who this might be right for
Ventilator Lung
Mansoura University 38
Not applicableStudy completedNCT01776164
What this trial is testing

Early and Longitudinal Assessment of Neurodegeneration in the Brain and Spinal Cord in Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
University of Minnesota 85
Not applicableLooking for participantsNCT06147414
What this trial is testing

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders

Who this might be right for
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, IncludingSickle Cell DiseaseCystic Fibrosis+12 more
Assistance Publique - Hôpitaux de Paris 550
Early research (Phase 1)Study completedNCT02493816
What this trial is testing

Safety Study of Gene-modified Autologous Fibroblasts in Recessive Dystrophic Epidermolysis Bullosa

Who this might be right for
Recessive Dystrophic Epidermolysis Bullosa
King's College London 5
Large-scale testing (Phase 3)UnknownNCT01222000
What this trial is testing

Treatment of the Recessive Nonbullous Congenital Ichthyosis by the Epigallocatechine Cutaneous

Who this might be right for
Lamellar Ichthyosis
Centre Hospitalier Universitaire de Nice 8
Testing effectiveness (Phase 2)Study completedNCT04686175
What this trial is testing

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency

Who this might be right for
Ectonucleotide Pyrophosphatase/phosphodiesterase1 DeficiencyAutosomal Recessive Hypophosphatemic RicketsGeneralized Arterial Calcification of Infancy
Inozyme Pharma 9
Not applicableStudy completedNCT05102084
What this trial is testing

The Effect of Listening to Music During CPAP on the Agitation Levels and Compliance.

Who this might be right for
COVID-19COVID-19 Acute Respiratory Distress Syndrome
SÜMEYYE BİLGİLİ 70
Early research (Phase 1)Active Not RecruitingNCT06713434
What this trial is testing

Pilot Study of ELK-003 Eye Drops for Treating Ocular Manifestations of Epidermolysis Bullosa

Who this might be right for
Junctional Epidermolysis BullosaEpidermolysis Bullosa (EB)Dystrophic Epidermolysis Bullosa+1 more
Fundación DEBRA Chile, Niños Piel de Cristal 20
Not applicableEnded earlyNCT02926963
What this trial is testing

Generation of Powerful Biological Tools for Understanding the Pathophysiology of Chronic Granulomatous Disease.

Who this might be right for
Chronic Granulomatous Disease
University Hospital, Grenoble 3
Testing effectiveness (Phase 2)Study completedNCT03041038
What this trial is testing

The Efficacy and Safety of Secukinumab in Patients With Ichthyoses

Who this might be right for
IchthyosisAutosomal Recessive Congenital IchthyosisLamellar Ichthyosis+3 more
Northwestern University 20
Not applicableStudy completedNCT04157595
What this trial is testing

Mackenzie's Mission: The Australian Reproductive Carrier Screening Project

Who this might be right for
X-Linked Genetic DiseasesAutosomal Recessive Disorder
Murdoch Childrens Research Institute 18,302
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07523581
What this trial is testing

EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety

Who this might be right for
Alport Syndrome, X-LinkedAlport Syndrome, Autosomal Recessive
Eloxx Pharmaceuticals, Inc. 24
Not applicableEnrolling By InvitationNCT05708677
What this trial is testing

A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of RDEB

Who this might be right for
RDEB
Abeona Therapeutics, Inc 22
Large-scale testing (Phase 3)Active Not RecruitingNCT06046820
What this trial is testing

The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency

Who this might be right for
Ectonucleotide Pyrophosphatase/Phosphodiesterase1 DeficiencyAutosomal Recessive Hypophosphatemic RicketsGeneralized Arterial Calcification of Infancy
Inozyme Pharma 27
Not applicableLooking for participantsNCT06563414
What this trial is testing

A Natural History Study of Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa (DEB)

Who this might be right for
Epidermolysis Bullosa DystrophicaEpidermolysis Bullosa Dystrophica, RecessiveEpidermolysis Bullosa Dystrophica Dominans
Krystal Biotech, Inc. 40
Large-scale testing (Phase 3)Looking for participantsNCT07419334
What this trial is testing

Study of ALK-001 on the Progression of Stargardt Disease

Who this might be right for
Stargardt Disease
Alkeus Pharmaceuticals, Inc. 230
Testing effectiveness (Phase 2)Looking for participantsNCT07002398
What this trial is testing

Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)

Who this might be right for
Retinal Dystrophy Due to Biallelic ABCA4 MutationsStargardt Disease 1
VeonGen Therapeutics GmbH 15
Early research (Phase 1)Study completedNCT00008450
What this trial is testing

Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant

Who this might be right for
Adenosine Deaminase DeficiencyAutosomal Recessive DisorderImmune System Disorder+4 more
Fred Hutchinson Cancer Center 6
Early research (Phase 1)Study completedNCT03041363
What this trial is testing

Treatment Development of Triheptanoin (G1D)

Who this might be right for
EpilepsyGLUT1DS1Glut1 Deficiency Syndrome 1, Autosomal Recessive+4 more
Juan Pascual 12
Very early researchStudy completedNCT04177498
What this trial is testing

Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated SCC

Who this might be right for
Recessive Dystrophic Epidermolysis Bullosa
Thomas Jefferson University 3
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