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Results for “AMN”

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Showing 20 of 0 results

Not applicableEnded earlyNCT02204904
What this trial is testing

Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)

Who this might be right for
X-Linked Adrenoleukodystrophy (X-ALD)Cerebral Adrenoleukodystrophy (CALD)Adrenoleukodystrophy (ALD)
Genetix Biotherapeutics Inc. 59
Not applicableLooking for participantsNCT04675749
What this trial is testing

Quality of Life in Women With X-linked Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
Leipzig University Medical Center 200
Not applicableStudy completedNCT03627416
What this trial is testing

Repetitive Transcranial Magnetic Stimulation as Therapy in Hereditary Spastic Paraplegia and Adrenomyeloneuropathy

Who this might be right for
Hereditary Spastic ParaplegiaAdrenomyeloneuropathy
Jakub Antczak 15
Large-scale testing (Phase 3)Ended earlyNCT00654433
What this trial is testing

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

Who this might be right for
Inherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more
Aldagen 40
Testing effectiveness (Phase 2)Looking for participantsNCT01962415
What this trial is testing

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Who this might be right for
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+4 more
Paul Szabolcs 100
Early research (Phase 1)WithdrawnNCT01787578
What this trial is testing

Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)

Who this might be right for
X-Linked AdrenoleukodystrophyAdrenomyeloneuropathy
Thomas S. Scanlan
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
Early research (Phase 1)Temporarily pausedNCT06030648
What this trial is testing

MT2022-01: MSCs for ALD

Who this might be right for
Cerebral Adrenoleukodystrophy
Masonic Cancer Center, University of Minnesota 10
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
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