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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Post-approval studies (Phase 4)Study completedNCT00483379
What this trial is testing

High Dose or High Dose Frequency Study of Alglucosidase Alfa

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II (GSD-II)Glycogenesis 2 Acid Maltase Deficiency
Genzyme, a Sanofi Company 13
Not applicableUnknownNCT01482494
What this trial is testing

Detection of Pompe Disease in Adult Patients With Myopathies of Uncertain Origin or With Asymptomatic Hyper-CK-emia

Who this might be right for
Pompe Disease
Jordi Perez Lopez 50
Not applicableEnrolling By InvitationNCT06376279
What this trial is testing

Genetic Diagnosis in Inborn Errors of Metabolism

Who this might be right for
Metabolic DiseaseMitochondrial DiseasesEpilepsy in Children+3 more
Region Stockholm 1,000
Not applicableUnknownNCT03949920
What this trial is testing

Migalastat in Fabry Disease

Who this might be right for
Fabry Disease
Manchester University NHS Foundation Trust 21
Large-scale testing (Phase 3)UnknownNCT04860960
What this trial is testing

Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1

Who this might be right for
Niemann-Pick Disease, Type C1
Cyclo Therapeutics, Inc. 94
Not applicableLooking for participantsNCT07508631
What this trial is testing

Friedreich Ataxia Nerve Ultrasund

Who this might be right for
Friedreich Ataxia
Centre Hospitalier Universitaire de Nice 14
Not applicableLooking for participantsNCT03291223
What this trial is testing

Gaucher Disease Outcome Survey (GOS)

Who this might be right for
Gaucher Disease
Shire 1,257
Testing effectiveness (Phase 2)Study completedNCT02023866
What this trial is testing

Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease

Who this might be right for
Inherited Mitochondrial Disease, Including Leigh Syndrome
Amgen 36
Not applicableActive Not RecruitingNCT05035823
What this trial is testing

COMMAND Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis

Who this might be right for
Neurologic DisorderParalysisParalysis; Stroke+9 more
Synchron Medical, Inc. 6
Not applicableActive Not RecruitingNCT06614569
What this trial is testing

Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease

Who this might be right for
GM2 GangliosidosisTay Sachs DiseaseSandhoff Disease
Terence Flotte 7
Early research (Phase 1)WithdrawnNCT01003912
What this trial is testing

Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases

Who this might be right for
Lysosomal Storage DiseasesInborn Errors of Metabolism
Joanne Kurtzberg, MD
Not applicableEnded earlyNCT04094181
What this trial is testing

VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies

Who this might be right for
Gaucher Disease
Shire 2
Early research (Phase 1)Looking for participantsNCT04532047
What this trial is testing

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Who this might be right for
MPS IMPS IIMPS IVA+6 more
University of California, San Francisco 10
Not applicableLooking for participantsNCT06904027
What this trial is testing

A Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle Disorders

Who this might be right for
Urea Cycle Disorders
Tongji Hospital 40
Post-approval studies (Phase 4)Study completedNCT01288027
What this trial is testing

Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

Who this might be right for
Pompe Disease (Late-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenesis 2 Acid Maltase Deficiency
Genzyme, a Sanofi Company 16
Not applicableLooking for participantsNCT05985486
What this trial is testing

Brain Outcomes With Lifestyle Change in Down Syndrome

Who this might be right for
Down SyndromeAlzheimer DiseaseObesity
University of Kansas Medical Center 81
Not applicableNo Longer AvailableNCT05686564
What this trial is testing

Early Access Program for ALXN1840 in Patients With Wilson Disease

Who this might be right for
Wilson Disease
Alexion Pharmaceuticals, Inc.
Not applicableEnded earlyNCT04836377
What this trial is testing

A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02

Who this might be right for
Type 1 Gaucher Disease
AVROBIO 4
Not applicableUnknownNCT02263781
What this trial is testing

PREPL in Health and Disease

Who this might be right for
Hypotonia-Cystinuria SyndromeMuscle HypotoniaHealthy Volunteers+3 more
Universitaire Ziekenhuizen KU Leuven 125
Large-scale testing (Phase 3)Study completedNCT00158600
What this trial is testing

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Glycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 90
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