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Results for “Autosomal Recessive”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Looking for participantsNCT04782258
What this trial is testing

See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Who this might be right for
Autosomal Recessive Polycystic Kidney (ARPKD)
Otsuka Pharmaceutical Development & Commercialization, Inc. 20
Testing effectiveness (Phase 2)Active Not RecruitingNCT06462547
What this trial is testing

ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency

Who this might be right for
Gene MutationsPseudoxanthoma ElasticumArterial Calcification+2 more
Inozyme Pharma 22
Testing effectiveness (Phase 2)Study completedNCT00730314
What this trial is testing

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

Who this might be right for
Sickle Cell DiseaseThalassemiaAnemia+9 more
Children's Hospital Los Angeles 25
Not applicableStudy completedNCT02977923
What this trial is testing

Decreasing REcurrent Pain and Anxiety in Medical Procedures With a Pediatric Population: a Pilot Study

Who this might be right for
PainAnxietyBurns+2 more
St. Justine's Hospital 20
Not applicableStudy completedNCT03273764
What this trial is testing

Clinical RD (Respiratory Distress) Score for Objective Decision Making for Surfactant Therapy

Who this might be right for
Respiratory Distress Syndrome in Premature InfantSurfactantCPAP
Lady Hardinge Medical College 209
Not applicableStudy completedNCT07133256
What this trial is testing

Effects of Aerobic Versus Resistance Training on Bone Metabolism in Stage 3-4 Chronic Kidney Disease

Who this might be right for
OsteopeniaBone Metabolism DisordersChronic Kidney Disease (Stage 3-4)+1 more
Sinai University 60
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
Not applicableStudy completedNCT01599780
What this trial is testing

Language Development in Preterm Children

Who this might be right for
Language Development
Stanford University 100
Testing effectiveness (Phase 2)UnknownNCT05735158
What this trial is testing

Topical KB105 for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)

Who this might be right for
Autosomal Recessive Ichthyosis
Krystal Biotech, Inc. 15
Not applicableUnknownNCT04170192
What this trial is testing

Multi-center Clinical Study of Cord Blood Stem Cell Transplantation for IBD Caused by IL-10R Gene Deficiency

Who this might be right for
Inflammatory Bowel Diseases
Children's Hospital of Fudan University 50
Not applicableStudy completedNCT04153110
What this trial is testing

Cerebello-Spinal tDCS as Rehabilitative Intervention in Neurodegenerative Ataxia

Who this might be right for
AtaxiaSpinocerebellar AtaxiasSpinocerebellar Ataxia Type 1+5 more
Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia 61
Not applicableNot Yet RecruitingNCT07079527
What this trial is testing

Effects of Manual Positive End Expiratory Pressure and Non-invasive Continuous Positive Air Pressure in COPD Patients

Who this might be right for
COPD
Riphah International University 42
Not applicableLooking for participantsNCT05354622
What this trial is testing

Hereditary Spastic Paraplegia Genomic Sequencing Initiative (HSPseq)

Who this might be right for
Hereditary Spastic ParaplegiaNeurodegenerative DiseasesPediatric Disorder+3 more
Boston Children's Hospital 200
Testing effectiveness (Phase 2)Study completedNCT03364153
What this trial is testing

Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)

Who this might be right for
Stargardt's Macular Dystrophy
Astellas Pharma Global Development, Inc. 121
Not applicableLooking for participantsNCT06639802
What this trial is testing

Effects of Hand Arm Bimanual Intensive Therapy Including Lower Extremity on Balance and Coordination in Ataxic Cerebral Palsy

Who this might be right for
Ataxic Cerebral PalsyCoordination and Balance Disturbances
Foundation University Islamabad 23
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
Testing effectiveness (Phase 2)Study completedNCT00301834
What this trial is testing

Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders

Who this might be right for
Congenital Amegakaryocytic ThrombocytopeniaDiamond-blackfan AnemiaLeukemia+2 more
University of California, San Francisco 35
Not applicableLooking for participantsNCT06535542
What this trial is testing

Integrating Whole Genome Sequencing and Digital Twins Into the Management of Hypercholesterolemia in Emiratis

Who this might be right for
Hypercholesterolemia, Autosomal DominantHypercholesterolemia, Autosomal RecessiveFamilial Combined Hypercholesterolemia
Abu Dhabi Health Services Company 40
Early research (Phase 1)Ended earlyNCT00272415
What this trial is testing

Intravenous INO-1001 Plus Oral Temozolomide to Evaluate Tolerability, Safety, and Pharmacokinetics in Patients With Melanoma

Who this might be right for
Melanoma
Genentech, Inc. 39
Not applicableUnknownNCT03449225
What this trial is testing

Prenatal Computer-Aided Genetics Education Module

Who this might be right for
Autosomal Recessive DisorderAneuploidy
Wayne State University 100
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