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Results for “Recessive Hereditary Disorder (Autosomal)”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Active Not RecruitingNCT04239625
What this trial is testing

Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)

Who this might be right for
Stargardt DiseaseStargardt Macular DegenerationStargardt Macular Dystrophy+1 more
Alkeus Pharmaceuticals, Inc. 200
Early research (Phase 1)Study completedNCT01917708
What this trial is testing

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

Who this might be right for
Hurler SyndromeFanconi AnemiaGlanzmann Thrombasthenia+12 more
Emory University 10
Testing effectiveness (Phase 2)Looking for participantsNCT07523581
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
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
Testing effectiveness (Phase 2)Looking for participantsNCT04099966
What this trial is testing

AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

Who this might be right for
Acute LeukemiaSevere Aplastic AnemiaNon-hodgkin Lymphoma+6 more
Mitchell Cairo 20
Not applicableLooking for participantsNCT01401998
What this trial is testing

ARPKD Database Study

Who this might be right for
Hepato/Renal Fibrocystic DiseaseAutosomal Recessive Polycystic Kidney DiseaseJoubert Syndrome+7 more
Children's Hospital of Philadelphia 200
Testing effectiveness (Phase 2)Active Not RecruitingNCT01966367
What this trial is testing

CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

Who this might be right for
Bone Marrow Failure SyndromeSevere Aplastic AnemiaSevere Congenital Neutropenia+13 more
Diane George 37
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07681778
What this trial is testing

Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)

Who this might be right for
Leber Congenital AmaurosisLeber Congenital Amaurosis (LCA)
Opus Genetics, Inc 10
Early research (Phase 1)Study completedNCT03096080
What this trial is testing

A Safety, Pharmacokinetic, Single Ascending Dose Study of Tesevatinib in Pediatric Subjects With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Who this might be right for
Polycystic Kidney, Autosomal Recessive
Kadmon Corporation, LLC 10
Not applicableEnded earlyNCT01319851
What this trial is testing

Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation

Who this might be right for
ThalassemiaSickle Cell DiseaseGlanzmann Thrombasthenia+10 more
Emory University 3
Not applicableLooking for participantsNCT06374719
What this trial is testing

WiTNNess - TNNT1 Myopathy Natural History Study

Who this might be right for
TNNT1-associated MyopathyInfantile-onset Nemaline Rod MyopathyMyopathies, Nemaline+9 more
Clinic for Special Children 40
Not applicableNot Yet RecruitingNCT07778641
What this trial is testing

Genetic Study of OTOF- Related Auditory Neuropathy Spectrum Disorder

Who this might be right for
Auditory Neuropathy Spectrum DisorderAuditory Neuropathy
Sohag University 30
Not applicableLooking for participantsNCT04815005
What this trial is testing

HoFH, the International Clinical Collaborators Registry

Who this might be right for
Homozygous Familial Hypercholesterolemia
University of Pennsylvania 1,000
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
Large-scale testing (Phase 3)Study completedNCT00176852
What this trial is testing

Stem Cell Transplant for Hemoglobinopathy

Who this might be right for
Sickle Cell DiseaseThalassemiaSevere Congenital Neutropenia+2 more
Masonic Cancer Center, University of Minnesota 22
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
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
Testing effectiveness (Phase 2)Enrolling By InvitationNCT04833907
What this trial is testing

RAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease

Who this might be right for
Canavan Disease
Myrtelle Inc. 24
Not applicableStudy completedNCT07745179
What this trial is testing

Natural History Study: ENPP1 Deficiency or the Early-Onset Form of ABCC6 Deficiency

Who this might be right for
Ectonucleotide Pyrophosphatase/phosphodiesterase1 DeficiencyATP-Binding Cassette Subfamily C Member 6 DeficiencyGeneralized Arterial Calcification of Infancy+2 more
Inozyme Pharma 23
Testing effectiveness (Phase 2)Looking for participantsNCT04998396
What this trial is testing

AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

Who this might be right for
Canavan Disease
Aspa Therapeutics 26
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