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Results for “Mucopolysaccharidosis Type 3 A”

52 trials

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Showing 20 of 52 results

Not applicableStudy completedNCT01047306
What this trial is testing

Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Who this might be right for
Sanfilippo Syndrome Type A
Shire 25
Post-approval studies (Phase 4)Enrolling By InvitationNCT05492799
What this trial is testing

Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE

Who this might be right for
MPS III B
Allievex Corporation 15
Testing effectiveness (Phase 2)Study completedNCT00383448
What this trial is testing

HSCT for High Risk Inherited Inborn Errors

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+6 more
Masonic Cancer Center, University of Minnesota 38
Testing effectiveness (Phase 2)Ended earlyNCT02350816
What this trial is testing

An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.

Who this might be right for
Sanfilippo SyndromeMucopolysaccharidosis (MPS)
Shire 17
Testing effectiveness (Phase 2)Study completedNCT03423186
What this trial is testing

Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients

Who this might be right for
Sanfilippo Syndrome Type A (MPS IIIA)
Swedish Orphan Biovitrum 6
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
Testing effectiveness (Phase 2)Ended earlyNCT04088734
What this trial is testing

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

Who this might be right for
MPS IIIASanfilippo SyndromeSanfilippo A+1 more
Ultragenyx Pharmaceutical Inc 5
Not applicableStudy completedNCT01938014
What this trial is testing

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

Who this might be right for
Mucopolysaccharidosis Type I (MPS I)Mucopolysaccharidosis Type II (MPS II)Mucopolysaccharidosis Type III (MPS III)+2 more
University of Chicago 19
Large-scale testing (Phase 3)UnknownNCT03612869
What this trial is testing

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

Who this might be right for
Mucopolysaccharidosis Type IIIA
LYSOGENE 20
Not applicableUnknownNCT03227042
What this trial is testing

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Who this might be right for
Mucopolysaccharidosis Type IIIB
Allievex Corporation 44
Testing effectiveness (Phase 2)Study completedNCT02060526
What this trial is testing

Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease

Who this might be right for
Sanfilippo Syndrome
Shire 21
Testing effectiveness (Phase 2)Study completedNCT00730314
What this trial is testing

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

Who this might be right for
Sickle Cell DiseaseThalassemiaAnemia+9 more
Children's Hospital Los Angeles 25
Not applicableLooking for participantsNCT03333200
What this trial is testing

Longitudinal Study of Neurodegenerative Disorders

Who this might be right for
MLDKrabbe DiseaseALD+24 more
University of Pittsburgh 1,500
Not applicableNot Yet RecruitingNCT07712003
What this trial is testing

Natural History to Assess Disease in Patients With MPS IIIC

Who this might be right for
Sanfilippo Syndrome Type CMucopolysaccharidosis (MPS) IIIC
Phoenix Nest 35
Large-scale testing (Phase 3)Looking for participantsNCT02716246
What this trial is testing

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Who this might be right for
MPS IIIASanfilippo SyndromeSanfilippo A+1 more
Ultragenyx Pharmaceutical Inc 36
Not applicableStudy completedNCT02493998
What this trial is testing

Mucopolysaccharidosis Type IIIB (MPS IIIB)

Who this might be right for
Mucopolysaccharidosis Type IIIBMucopolysaccharidosis Type 3 BMPS III B+1 more
Allievex Corporation 22
Testing effectiveness (Phase 2)Study completedNCT02053064
What this trial is testing

Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy

Who this might be right for
Mucopolysaccharidosis Type III ASanfilippo Disease Type A
LYSOGENE 4
Testing effectiveness (Phase 2)Study completedNCT01155778
What this trial is testing

Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients

Who this might be right for
Mucopolysaccharidosis (MPS)
Shire 12
Not applicableStudy completedNCT02293382
What this trial is testing

A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

Who this might be right for
MPS IIIB (Sanfilippo Syndrome)
Alexion Pharmaceuticals, Inc. 30
Large-scale testing (Phase 3)Study completedNCT02230566
What this trial is testing

UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Who this might be right for
MPS 7Sly SyndromeMucopolysaccharidosis+1 more
Ultragenyx Pharmaceutical Inc 12
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