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Results for “Mucopolysaccharidosis Type 3 A”

50 trials

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Showing 20 of 50 results

Large-scale testing (Phase 3)Study completedNCT01275066
What this trial is testing

Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Who this might be right for
MPS IV A
BioMarin Pharmaceutical 177
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
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 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)Looking for participantsNCT05371613
What this trial is testing

Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Who this might be right for
Mucopolysaccharidosis II
Denali Therapeutics Inc. 63
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 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
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 applicableWithdrawnNCT02298686
What this trial is testing

Biomarker for Sanfilippo Type A-B-C-D Disease (BioSanfilippo)

Who this might be right for
Mental RetardationDevelopmental Delay
CENTOGENE GmbH Rostock
Large-scale testing (Phase 3)Ended earlyNCT00654433
What this trial is testing

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

Who this might be right for
Inherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more
Aldagen 40
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
Testing effectiveness (Phase 2)Active Not RecruitingNCT06095388
What this trial is testing

Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA

Who this might be right for
Mucopolysaccharidosis III-A
JCR Pharmaceuticals Co., Ltd. 15
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 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
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
Testing effectiveness (Phase 2)Study completedNCT03811028
What this trial is testing

Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment 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)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
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
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 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
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