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Results for “Mucopolysaccharidosis Type IIIA”

37 trials

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Showing 20 of 37 results

Testing effectiveness (Phase 2)Active Not RecruitingNCT04201405
What this trial is testing

Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA

Who this might be right for
Mucopolysaccharidosis Type IIIA
University of Manchester 5
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 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
Testing effectiveness (Phase 2)Study completedNCT01474343
What this trial is testing

Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

Who this might be right for
Mucopolysaccharidosis Type III ASanfilippo Disease Type A
LYSOGENE 4
Not applicableStudy completedNCT01586871
What this trial is testing

Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network

Who this might be right for
Mucopolysaccharidoses
University of Minnesota 1,147
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
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
Not applicableStudy completedNCT02746341
What this trial is testing

Natural History Study of Patients With MPS IIIA

Who this might be right for
Mucopolysaccharidosis IIIA
LYSOGENE 23
Not applicableStudy completedNCT01873911
What this trial is testing

Neurobehavioral Phenotypes in MPS III

Who this might be right for
Sanfilippo Syndrome Type ASanfilippo Syndrome Type BHurler Syndrome
University of Minnesota 30
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
Early research (Phase 1)Active Not RecruitingNCT06567769
What this trial is testing

Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Who this might be right for
Sanfilippo Syndrome Type A
GC Biopharma Corp 9
Large-scale testing (Phase 3)Enrolling By InvitationNCT04360265
What this trial is testing

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

Who this might be right for
Mucopolysaccharidosis IIIAMPS IIIASanfilippo Syndrome+1 more
Ultragenyx Pharmaceutical Inc 41
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
Testing effectiveness (Phase 2)Study completedNCT02754076
What this trial is testing

A Treatment Study of Mucopolysaccharidosis Type IIIB

Who this might be right for
MPS III BMucopolysaccharidosis Type IIIB
Allievex Corporation 23
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
Testing effectiveness (Phase 2)Ended earlyNCT01299727
What this trial is testing

Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Who this might be right for
Sanfilippo Syndrome
Shire 12
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)Ended earlyNCT01372228
What this trial is testing

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Who this might be right for
Hurler Syndrome (MPS I)Hurler-Scheie SyndromeHunter Syndrome (MPS II)+9 more
Talaris Therapeutics Inc. 3
Testing effectiveness (Phase 2)UnknownNCT03784287
What this trial is testing

A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

Who this might be right for
Mucopolysaccharidosis Type IIIBMPS III B
Allievex Corporation 20
Not applicableUnknownNCT04918641
What this trial is testing

Natural History Observational Study of MPS IIIa in SMC

Who this might be right for
Sanfilippo Syndrome A
Samsung Medical Center 25
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