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Results for “Autosomal Recessive”

209 trials

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Showing 20 of 209 results

Large-scale testing (Phase 3)Active Not RecruitingNCT05627648
What this trial is testing

Phase 3 Study of KP-100LI in Subjects With Vocal Fold Scar

Who this might be right for
Vocal Fold Scar
Kringle Pharma, Inc. 62
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
Post-approval studies (Phase 4)Study completedNCT05759416
What this trial is testing

Evaluation Efficacy of Melatonin Gel as Adjunctive Therapy in Chronic Periodontitis

Who this might be right for
Chronic Periodontitis
Al-Azhar University 40
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Not applicableUnknownNCT05495477
What this trial is testing

Effects of NIV and CPAP on Ventilation Distribution, Measured by EIT, During Deep Sedation in Paediatric Patients

Who this might be right for
Respiratory FailureSedationMechanical Ventilation Complication
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico 20
Large-scale testing (Phase 3)UnknownNCT04475224
What this trial is testing

Phase 3 Study of KP-100IT in Subjects With Acute Spinal Cord Injury

Who this might be right for
Acute Spinal Cord Injury
Kringle Pharma, Inc. 25
Not applicableUnknownNCT03319888
What this trial is testing

Effect of CPAP in the Worsening of Renal Function in Early Stages of Chronic Kidney Disease (CKD)

Who this might be right for
Chronic Kidney InsufficiencySleep Apnea
Sociedad Española de Neumología y Cirugía Torácica 700
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 completedNCT00254059
What this trial is testing

Study to Compare Polysomnography With Overnight Home Oximetry and Auto - CPAP for Sleep Apnea

Who this might be right for
Obstructive Sleep Apnea
University of British Columbia 60
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 participantsNCT07185256
What this trial is testing

Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)

Who this might be right for
ARBBVMDAutosomal-Dominant Bestrophinopathy+1 more
Opus Genetics, Inc 10
Testing effectiveness (Phase 2)UnknownNCT02061800
What this trial is testing

CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant

Who this might be right for
Chronic Myeloid Leukemia (CML)Acute Myelogenous Leukemia (AML)Myelodysplastic Syndrome (MDS)+3 more
Diane George 14
Not applicableEnrolling By InvitationNCT06696456
What this trial is testing

Long Term Follow-up Study of AAVAnc80-hOTOF Gene Therapy

Who this might be right for
Otoferlin Gene-mediated Hearing Loss
Akouos, Inc. 30
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)Looking for participantsNCT05821959
What this trial is testing

Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

Who this might be right for
Sensorineural Hearing Loss, Bilateral
Akouos, Inc. 22
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)Looking for participantsNCT06942572
What this trial is testing

A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)

Who this might be right for
Stargardt DiseaseStargardt Macular DegenerationStargardt Macular Dystrophy
Splice Bio 57
Testing effectiveness (Phase 2)Enrolling By InvitationNCT06253507
What this trial is testing

PCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)

Who this might be right for
p47Autosomal RecessiveChronic Granulomatous Disease
National Institute of Allergy and Infectious Diseases (NIAID) 10
Not applicableStudy completedNCT05479656
What this trial is testing

A Rehabilitation Program to Increase Balance and Mobility in Ataxia of Charlevoix-Saguenay

Who this might be right for
Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay
Élise Duchesne 10
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
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