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Results for “Recessive”

259 trials

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Showing 20 of 259 results

Not applicableActive Not RecruitingNCT03440697
What this trial is testing

Pathogenetic Basis of Aortopathy and Aortic Valve Disease

Who this might be right for
AortopathiesThoracic Aortic AneurysmAortic Valve Disease+19 more
Yale University 3,000
Large-scale testing (Phase 3)WithdrawnNCT04786574
What this trial is testing

See if Tolvaptan Can Delay Dialysis in Infants and Children Who at Enrollment Are 28 Days to Less Than 12 Weeks Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Who this might be right for
Autosomal Recessive Polycystic Kidney Disease (ARPKD)
Otsuka Pharmaceutical Development & Commercialization, Inc.
Very early researchLooking for participantsNCT07066085
What this trial is testing

Serial Blood Count Study

Who this might be right for
Cyclic NeutropeniaCongenital NeutropeniaNeutropenia
University of Washington 20
Testing effectiveness (Phase 2)Ended earlyNCT03738800
What this trial is testing

A Safety, Efficacy and Systemic Exposure Study of CD5789 Cream in Adults and Adolescents With Lamellar Ichthyosis

Who this might be right for
Lamellar Ichthyosis
Mayne Pharma International Pty Ltd 65
Not applicableStudy completedNCT05411328
What this trial is testing

Efficacy of Virtual Reality on Balance in Children With Ataxic Cerebral Palsy Randomized Controlled Trail

Who this might be right for
Ataxic Cerebral Palsy
Delta University for Science and Technology 64
Not applicableStudy completedNCT04372446
What this trial is testing

Understanding the Spectrum of ENPP1 Deficiency and Acute ABCC6 Deficiency

Who this might be right for
Generalized Arterial Calcification in InfancyAutosomal Recessive Hypophosphatemic Rickets Type 2
Inozyme Pharma 38
Testing effectiveness (Phase 2)Study completedNCT04520022
What this trial is testing

Safety and Effectiveness Study of Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With RDEB

Who this might be right for
Recessive Dystrophic Epidermolysis Bullosa
Gangnam Severance Hospital 5
Not applicableStudy completedNCT02587832
What this trial is testing

High Flow Nasal Cannula in Comparison With Nasal Continuous Positive Airway Pressure in the Management of Respiratory Distress Syndrome

Who this might be right for
Respiratory Distress Syndrome
Sidra Medicine 60
Testing effectiveness (Phase 2)WithdrawnNCT04301284
What this trial is testing

Study of CAD-1883 for Spinocerebellar Ataxia

Who this might be right for
Spinocerebellar AtaxiasSpinocerebellar Ataxia Type 1Spinocerebellar Ataxia Type 2+7 more
Cadent Therapeutics
Not applicableLooking for participantsNCT06605378
What this trial is testing

Prevalence of Antibodies and Cytokines in Participants With Chronic Granulomatous Disease

Who this might be right for
Chronic Granulomatous Disease (CGD)
Ensoma 60
Not applicableLooking for participantsNCT04261127
What this trial is testing

Validation of the RADIAL Algorithm for Diagnosis of Autosomal Recessive Cerebellar Ataxia

Who this might be right for
Autosomal Recessive Cerebellar Ataxia
University Hospital, Strasbourg, France 400
Testing effectiveness (Phase 2)Not Yet RecruitingNCT04844177
What this trial is testing

Total Lymphoid Irradiation Pre-HSCT in Severe Congenital Neutropenia

Who this might be right for
Severe Congenital NeutropeniaGATA2 Deficiency
Federal Research Institute of Pediatric Hematology, Oncology and Immunology 10
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
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
Not applicableAvailableNCT01995305
What this trial is testing

Use Massive Parallel Sequencing and Exome Capture Technology to Sequence the Exome of Fanconi Anemia Children and Their Patents

Who this might be right for
Fanconi AnemiaAutosomal or Sex Linked Recessive Genetic DiseaseBone Marrow Hematopoiesis Failure, Multiple Congenital Abnormalities, and Susceptibility to Neoplastic Diseases.+1 more
Xiaofan Zhu
Very early researchActive Not RecruitingNCT04171661
What this trial is testing

Self-Assembled Skin Substitute for the Treatment of Epidermolysis Bullosa

Who this might be right for
Epidermolysis Bullosa Dystrophica
CHU de Quebec-Universite Laval 1
Not applicableActive Not RecruitingNCT04126005
What this trial is testing

Natural History Study of Patients With Canavan Disease (CANinform Study)

Who this might be right for
Canavan Disease
Aspa Therapeutics 70
Not applicableStudy completedNCT06343324
What this trial is testing

Effect of Web-Based Adaptation and Insomnia Severity in Individuals Using Noninvasive Ventilators at Home

Who this might be right for
Noninvasive VentilationCOPD
KTO Karatay University 80
Testing effectiveness (Phase 2)UnknownNCT04153630
What this trial is testing

Safety Study and Preliminary Efficacy of Infusion Haploidentical Mesenchymal Stem Cells Derived From Bone Marrow for Treating Recessive Dystrophic Epidermolysis Bullosa

Who this might be right for
Epidermolysis Bullosa Dystrophica, Recessive
Instituto de Investigación Hospital Universitario La Paz 9
Not applicableStudy completedNCT01470729
What this trial is testing

Biomarkers in Autosomal Dominant Cerebellar Ataxia

Who this might be right for
Spinocerebellar Ataxia Type 1Spinocerebellar Ataxia Type 2Spinocerebellar Ataxia, Autosomal Recessive 3+1 more
Assistance Publique - Hôpitaux de Paris 102
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