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Results for “Mucopolysaccharidosis Type IIIA”

35 trials

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Showing 20 of 35 results

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 completedNCT05523206
What this trial is testing

A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)

Who this might be right for
Mucopolysaccharidosis III-A
Sanguine Biosciences 15
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
Early research (Phase 1)Looking for participantsNCT06567769
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
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
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
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)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 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
Testing effectiveness (Phase 2)Study completedNCT04018755
What this trial is testing

Open-label Study of Anakinra in MPS III

Who this might be right for
Mucopolysaccharidosis III
Lynda E Polgreen 24
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
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)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
Not applicableStudy completedNCT02037880
What this trial is testing

Natural History Studies of Mucopolysaccharidosis III

Who this might be right for
Mucopolysaccharidosis Type IIIAMucopolysaccharidosis Type IIIB
Nationwide Children's Hospital 25
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
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
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 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 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
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