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Results for “Neonatal Adrenoleukodystrophy”

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Showing 20 of 0 results

Early research (Phase 1)Study completedNCT03500627
What this trial is testing

Evaluate the Safety, Tolerability, and Pharmacokinetics of OP-101 After Intravenous Administration in Healthy Volunteers

Who this might be right for
Healthy
Orpheris, Inc. 8
Not applicableActive Not RecruitingNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Large-scale testing (Phase 3)Study completedNCT01896102
What this trial is testing

The Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

Who this might be right for
Cerebral Adrenoleukodystrophy (CALD)
Genetix Biotherapeutics Inc. 32
Post-approval studies (Phase 4)Active Not RecruitingNCT05003648
What this trial is testing

Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy

Who this might be right for
AdrenoleukodystrophyRestless Legs Syndrome
Massachusetts General Hospital 24
Not applicableStudy completedNCT02699190
What this trial is testing

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Who this might be right for
LeukodystrophyWhite Matter Disease4H Syndrome+61 more
Children's Hospital of Philadelphia 236
Not applicableLooking for participantsNCT04925349
What this trial is testing

Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia

Who this might be right for
AdrenoleukodystrophyAdrenomyeloneuropathyMetachromatic Leukodystrophy+1 more
Assistance Publique - Hôpitaux de Paris 100
Not applicableUnknownNCT00278044
What this trial is testing

Clinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese Children

Who this might be right for
Adrenoleukodystrophy
Far Eastern Memorial Hospital 20
Not applicableStudy completedNCT01626092
What this trial is testing

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

Who this might be right for
Lysosomal Storage DiseasePeroxisomal Disorder
Masonic Cancer Center, University of Minnesota 3
Not applicableWithdrawnNCT03649919
What this trial is testing

Multi-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in Children

Who this might be right for
XlsmaDMDX-ALD+1 more
Children's Hospital of Fudan University
Not applicableLooking for participantsNCT04528355
What this trial is testing

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

Who this might be right for
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+2 more
Paul Szabolcs 50
Not applicableLooking for participantsNCT06224413
What this trial is testing

Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel

Who this might be right for
Cerebral Adrenoleukodystrophy (CALD)
Genetix Biotherapeutics Inc. 120
Large-scale testing (Phase 3)Study completedNCT00176904
What this trial is testing

Stem Cell Transplant for Inborn Errors of Metabolism

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+8 more
Masonic Cancer Center, University of Minnesota 135
Testing effectiveness (Phase 2)Ended earlyNCT03367546
What this trial is testing

Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases

Who this might be right for
Sickle Cell DiseaseThalassemiaHigh Risk Hematologic Disorders+2 more
Masonic Cancer Center, University of Minnesota 5
Not applicableEnded earlyNCT05008874
What this trial is testing

Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia

Who this might be right for
AMNAMN Gene MutationX-ALD
SwanBio Therapeutics, Inc. 65
Large-scale testing (Phase 3)Study completedNCT03231878
What this trial is testing

A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.

Who this might be right for
Adrenoleukodystrophy
Minoryx Therapeutics, S.L. 105
Not applicableStudy completedNCT02952482
What this trial is testing

Newborn Screening for Adrenoleukodystrophy

Who this might be right for
Adrenoleukodystrophy
National Taiwan University Hospital 45,796
Not applicableLooking for participantsNCT03047369
What this trial is testing

The Myelin Disorders Biorepository Project

Who this might be right for
LeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more
Children's Hospital of Philadelphia 12,000
Not applicableLooking for participantsNCT03789721
What this trial is testing

Adrenoleukodystrophy National Registry Study

Who this might be right for
ALD (Adrenoleukodystrophy)AdrenoleukodystrophyCerebral Adrenoleukodystrophy
Masonic Cancer Center, University of Minnesota 1,000
Large-scale testing (Phase 3)Study completedNCT00007020
What this trial is testing

Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

Who this might be right for
Infantile Refsum's DiseaseZellweger SyndromeAdrenoleukodystrophy+2 more
Mirum Pharmaceuticals, Inc. 85
Not applicableUnknownNCT03278899
What this trial is testing

Prospectively Assess Disease Progression in Male Children With X-ALD

Who this might be right for
X-Linked Adrenoleukodystrophy
NeuroVia, Inc. 100
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