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Results for “Recessive Hereditary Disorder (Autosomal)”

86 trials

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Showing 20 of 86 results

Not applicableLooking for participantsNCT06967831
What this trial is testing

Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells

Who this might be right for
Leigh Syndrome (Maternally Inherited, MILS)Leigh Syndrome (AR, AD, XR)
Charite University, Berlin, Germany 80
Not applicableUnknownNCT03758534
What this trial is testing

Natural History of GACI With or Without ARHR2 or PXE

Who this might be right for
Generalized Arterial Calcification in InfancyAutosomal Recessive Hypophosphatemic RicketsPseudoxanthoma Elasticum
Universität Münster 80
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 applicableStudy completedNCT05621200
What this trial is testing

Transcranial Alternating Current Stimulation (tACS) in Patients With Ataxia

Who this might be right for
AtaxiaSpinocerebellar AtaxiasMultiple System Atrophy+2 more
Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia 26
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 applicableUnknownNCT05107830
What this trial is testing

Phenotyping of Primary Hyperoxaluria

Who this might be right for
Primary Hyperoxaluria
University Hospital, Strasbourg, France 186
Not applicableStudy completedNCT03120013
What this trial is testing

Rehabilitative Trial With Cerebello-Spinal tDCS in Neurodegenerative Ataxia

Who this might be right for
Ataxia, CerebellarCerebellar AtaxiaSpinocerebellar Ataxias+8 more
Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia 21
Not applicableStudy completedNCT01902940
What this trial is testing

Natural History in CCFDN and IBM Syndromes

Who this might be right for
Inclusion Body Myositis, SporadicInclusion Body Myopathy, Autosomal-recessiveInclusion Body Myopathy, Autosomal-dominant+1 more
Ludwig-Maximilians - University of Munich 350
Not applicableNo Longer AvailableNCT03172455
What this trial is testing

Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT)

Who this might be right for
Acute-graft-versus-host DiseaseSteroid Refractory Acute Graft Versus Host DiseaseGraft-versus-host-disease+5 more
Impatients N.V. trading as myTomorrows
Testing effectiveness (Phase 2)Study completedNCT00305708
What this trial is testing

Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission

Who this might be right for
Congenital Amegakaryocytic ThrombocytopeniaDiamond-blackfan AnemiaFanconi Anemia+3 more
University of California, San Francisco 40
Testing effectiveness (Phase 2)Active Not RecruitingNCT02402660
What this trial is testing

Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease

Who this might be right for
Stargardt DiseaseStargardt Macular DegenerationStargardt Macular Dystrophy+1 more
Alkeus Pharmaceuticals, Inc. 160
Not applicableStudy completedNCT00244010
What this trial is testing

Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias

Who this might be right for
Anemia, AplasticAmegakaryocytic ThrombocytopeniaDiamond-Blackfan Anemia+1 more
St. Jude Children's Research Hospital 4
Testing effectiveness (Phase 2)Study completedNCT01529827
What this trial is testing

Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies

Who this might be right for
Accelerated Phase Chronic Myelogenous LeukemiaAdult Acute Lymphoblastic Leukemia in RemissionAdult Acute Myeloid Leukemia in Remission+75 more
Roswell Park Cancer Institute 94
Testing effectiveness (Phase 2)Study completedNCT04489511
What this trial is testing

Study of STG-001 in Subjects With Stargardt Disease

Who this might be right for
Stargardt Disease-1
Stargazer Pharmaceuticals, Inc. 10
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
Early research (Phase 1)Study completedNCT00060749
What this trial is testing

Effect of DHA Supplements on Macular Function in Patients With Stargardt Macular Dystrophy and Stargardt-like Macular Dystrophy

Who this might be right for
Macular Degeneration
National Eye Institute (NEI) 22
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
Testing effectiveness (Phase 2)Active Not RecruitingNCT05095727
What this trial is testing

MRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)

Who this might be right for
Glycogen Storage Disease
ModernaTX, Inc. 15
Not applicableActive Not RecruitingNCT04765345
What this trial is testing

Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F

Who this might be right for
Retinal DegenerationRetinitis PigmentosaEye Diseases, Hereditary
Jaeb Center for Health Research 44
Testing effectiveness (Phase 2)Ended earlyNCT03333486
What this trial is testing

Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer

Who this might be right for
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 PositiveAcute Leukemia in RemissionAcute Lymphoblastic Leukemia+34 more
Roswell Park Cancer Institute 31
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