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Results for “Leukodystrophy, Spongiform”

13 trials

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Showing 13 of 13 results

Testing effectiveness (Phase 2)WithdrawnNCT00657748
What this trial is testing

Lithium and Acetate for Canavan Disease

Who this might be right for
Canavan DiseaseInfantileDeficiency Disease+2 more
Assistance Publique - Hôpitaux de Paris
Not applicableUnknownNCT00724802
What this trial is testing

Oral Glyceryl Triacetate (GTA) in Newborns With Canavan

Who this might be right for
Canavan Disease
Shaare Zedek Medical Center 1
Not applicableActive Not RecruitingNCT04126005
What this trial is testing

Natural History Study of Patients With Canavan Disease (CANinform Study)

Who this might be right for
Canavan Disease
Aspa Therapeutics 70
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 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 applicableNo Longer AvailableNCT05317780
What this trial is testing

Canavan-Single Patient IND

Who this might be right for
Canavan Disease
University of Florida
Not applicableEnrolling By InvitationNCT03655223
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
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
Not applicableStudy completedNCT01999257
What this trial is testing

Efficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent.

Who this might be right for
Tay Sachs DiseaseCanavan DiseaseFamilial Dysautonomia
McGill University Health Centre/Research Institute of the McGill University Health Centre 60
Early research (Phase 1)UnknownNCT00278707
What this trial is testing

GTA-Glyceryltriacetate for Canavan Disease

Who this might be right for
Infantile Canavan DiseaseDeficiency Disease, Aspartoacylase
Sheba Medical Center 5
Not applicableStudy completedNCT02851563
What this trial is testing

A Natural History Study of Canavan Disease

Who this might be right for
Canavan Disease
NYU Langone Health 21
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)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