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Results for “Sanfilippo Syndrome”

61 trials

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Showing 20 of 61 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
Not applicableAvailableNCT07733856
What this trial is testing

Early Access Treatment With Tralesinidase Alfa in Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B)

Who this might be right for
Mucopolysaccharidosis Type IIIBMPS IIIB (Sanfilippo B Syndrome)
Spruce Biosciences
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
Not applicableUnknownNCT03812055
What this trial is testing

Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage Disorders

Who this might be right for
Lysosomal Storage Diseases
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 50
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
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)Ended earlyNCT02618512
What this trial is testing

A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

Who this might be right for
Mucopolysaccharidosis III, Type B (MPS IIIB)Sanfilippo B
Alexion Pharmaceuticals, Inc. 3
Not applicableLooking for participantsNCT06036693
What this trial is testing

MPS (RaDiCo Cohort) (RaDiCo-MPS)

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis III+5 more
Institut National de la Santé Et de la Recherche Médicale, France 1,000
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
Not applicableStudy completedNCT03161171
What this trial is testing

Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III

Who this might be right for
Mucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type III+2 more
University Hospital Heidelberg 34
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
Not applicableEnded earlyNCT04655911
What this trial is testing

A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101

Who this might be right for
Mucopolysaccharidosis III-B
Abeona Therapeutics, Inc 1
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