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Results for “Autosomal Recessive”

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Showing 20 of 0 results

Not applicableUnknownNCT02217397
What this trial is testing

Combinations of Oral Appliance and CPAP for Patients With Severe Obstructive Sleep Apnea Untolerate High-pressure CPAP

Who this might be right for
Obstructive Sleep Apnea
National Taiwan University Hospital 60
Not applicableStudy completedNCT03478839
What this trial is testing

Study of People With Generalized Arterial Calcification of Infancy (GACI) or Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2)

Who this might be right for
Generalized Arterial Calcification of InfancyAutosomal Recessive Hypophosphatemic Rickets Type2
National Human Genome Research Institute (NHGRI) 48
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
Large-scale testing (Phase 3)UnknownNCT05968118
What this trial is testing

Angiogenesis and Blood Perfusion Effect of HGF Gene Therapy in PAD Patients

Who this might be right for
Peripheral Artery Disease
Beijing Northland Biotech. Co., Ltd. 36
Testing effectiveness (Phase 2)Ended earlyNCT01539590
What this trial is testing

Study to Evaluate the Safety and Activity of BB3 to Treat Heart Attack

Who this might be right for
Myocardial Infarction
Angion Biomedica Corp 5
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
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)Study completedNCT03366142
What this trial is testing

Ustekinumab (Anti-IL-12/23p40 Monoclonal Antibody) in Patients With Leukocyte Adhesion Deficiency Type 1 (LAD1) Who Have Inflammatory Pathology

Who this might be right for
LAD1
National Institute of Allergy and Infectious Diseases (NIAID) 6
Not applicableWithdrawnNCT06019481
What this trial is testing

A Natural History Study in Pediatric Participants With Hearing Loss Due to OTOF, GJB2, or GJB2/GJB6 Mutations

Who this might be right for
Congenital Hearing Loss Secondary to Biallelic Mutations in the Otoferlin Gene (OTOF)Biallelic Mutations in the Gap Junction Beta 2 (GJB2) GeneDigenic Mutations in GJB2/Gap Junction Beta 6 (GJB6) Genes
Regeneron Pharmaceuticals
Not applicableNot Yet RecruitingNCT07689435
What this trial is testing

Cholesterol Cream vs Urea Cream for Skin Barrier and Quality of Life in Ichthyosis

Who this might be right for
Autosomal Recessive Congenital IchthyosisSkin Barrier DysfunctionIchthyosis Inherited
Institute of Dermatology, Thailand 24
Not applicableStudy completedNCT04740359
What this trial is testing

Functional Trunk Training in Ataxia Patients

Who this might be right for
Ataxia, SpinocerebellarAtaxias, Hereditary
Hasan Kalyoncu University 20
Not applicableUnknownNCT03618719
What this trial is testing

Impact of Intermittent Hypoxia on the Function of the Phagocytes

Who this might be right for
Obstructive Sleep Apnea of Adult
Taipei Veterans General Hospital, Taiwan 40
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 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)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
Testing effectiveness (Phase 2)Looking for participantsNCT07665021
What this trial is testing

Gene-Modified Stem Cell Therapy for Children With Autosomal Recessive Osteopetrosis (ARO)

Who this might be right for
Osteopetrosis
Fondazione Telethon 8
Not applicableEnrolling By InvitationNCT04681781
What this trial is testing

SLC13A5 Deficiency Natural History Study - Remote Only

Who this might be right for
Citrate Transporter DeficiencyEpilepsyRare Diseases+7 more
TESS Research Foundation 20
Not applicableLooking for participantsNCT06261424
What this trial is testing

Effects of a Supervised Rehabilitation Program on Disease Severity in Spastic Ataxias

Who this might be right for
Autosomal Recessive Spastic Ataxia of Charlevoix-SaguenaySpastic Paraplegia 7
Laval University 84
Not applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
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
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