Clinical trials: Mucopolysaccharidosis III
MPS IIIA, Sanfilippo Syndrome
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
- Conditions
- MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
- Trial name
- NCT02716246
- Testing
- UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 36 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Sanfilippo Syndrome, Mucopolysaccharidosis III
Study of Cannabidiol in Sanfilippo Syndrome
The goal of this clinical trial is to test cannabidiol in Sanfilippo syndrome. The main questions it aims to answer are: 1) determine the safety of cannabidiol in Sanfilippo syndrome, and 2) explore the efficacy of cannabidiol in…
- Trial name
- NCT06333041
- Testing
- Epidiolex
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Mucopolysaccharidosis III-B
A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101
This is a multicenter, non-interventional, long-term follow-up (LTFU) study in participants who have been treated with ABO-101 in a prior trial.
- Trial name
- NCT04655911
- Testing
- ABO-101
- Where
- 3 sites in United States, France and 1 more
- Volunteers
- 1 people
- Sponsor
- Abeona Therapeutics, Inc
Sanfilippo Syndrome
Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Sanfilippo syndrome, or Mucopolysaccharidosis (MPS) III, is a rare lysosomal storage disease (LSD) caused by loss in activity of 1 of 9 enzymes necessary for degradation of the glycosaminoglycan (GAG) heparan sulfate (HS) in…
- Trial name
- NCT01299727
- Testing
- rhHNS-10 mg, rhHNS-45 mg, rhHNS-90 mg
- Where
- 2 sites in Netherlands, United Kingdom
- Volunteers
- 12 people
- Sponsor
- Shire
Sanfilippo Syndrome Type A (MPS IIIA)
A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
MPS IIIA, also known as Sanfilippo A, is an inherited lysosomal storage disease (LSD). MPS IIIA is caused by a deficiency in sulfamidase, one of the enzymes involved in the lysosomal degradation of the glycosaminoglycan (GAG)…
- Trial name
- NCT03811028
- Testing
- SOBI003
- Where
- 3 sites in United States, Turkey (Türkiye)
- Volunteers
- 6 people
- Sponsor
- Swedish Orphan Biovitrum
Mucopolysaccharidosis Type III A, Sanfilippo Disease Type A
Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
The clinical trial P1-SAF-301 is an open-label, single arm, monocentric, phase I/II clinical study evaluating the tolerance and the safety of intracerebral administration of adeno-associated viral vector serotype 10 carrying the…
- Trial name
- NCT01474343
- Testing
- SAF-301
- Where
- 2 sites in France
- Volunteers
- 4 people
- Sponsor
- LYSOGENE
Mucopolysaccharidosis Type IIIB, Mucopolysaccharidosis Type 3 B
A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
Mucopolysaccharidosis type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B) is a severe neurodegenerative disorder. The purpose of this study is to learn more about the health problems in patients with MPS IIIB and how…
- Conditions
- Mucopolysaccharidosis Type IIIB, Mucopolysaccharidosis Type 3 B, MPS III B, MPS 3 B
- Trial name
- NCT02493998
- Where
- 8 sites in United States, Australia and 6 more
- Volunteers
- 22 people
- Sponsor
- Allievex Corporation
Mucopolysaccharidosis III
Open-label Study of Anakinra in MPS III
Sanfilippo syndrome, or mucopolysaccharidosis type III (MPS III), is a disorder of metabolism, associated with insufficient production of a lysosomal enzyme needed for normal cell function.
- Trial name
- NCT04018755
- Testing
- anakinra
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Lynda E Polgreen
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II
Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III
The study quantitates behavioral challenges in mucopolysaccharidosis type I-III and parental coping strategies
- Conditions
- Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type III, Behavior Disorders
- Trial name
- NCT03161171
- Testing
- This is an observational study
- Where
- Germany
- Volunteers
- 34 people
- Sponsor
- University Hospital Heidelberg
MPS IIIA, Sanfilippo Syndrome
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
Open-label, clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein
- Conditions
- MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
- Trial name
- NCT04088734
- Testing
- ABO-102
- Where
- 3 sites in United States, Australia and 1 more
- Volunteers
- 5 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
ScreenPlus is a consented, multi-disorder pilot newborn screening program implemented in conjunction with the New York State Newborn Screening Program that provides families the option to have their newborn(s) screened for a…
- Conditions
- Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2, Cerebrotendinous Xanthomatosis, Fabry Disease
- Trial name
- ScreenPlus (NCT05368038)
- Testing
- Confirmatory Testing
- Where
- 9 sites in United States
- Volunteers
- 100,000 people, healthy volunteers welcome
- Sponsor
- Albert Einstein College of Medicine
Mucopolysaccharidosis III-A
A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)
This protocol is a decentralized, single cohort, natural history and biomarker study enrolling up to 20 participants with MPS IIIA (Sanfilippo syndrome).
- Trial name
- NCT05523206
- Testing
- No intervention
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Sanguine Biosciences
Mucopolysaccharidosis Type IIIA, Mucopolysaccharidosis Type IIIB
Natural History Studies of Mucopolysaccharidosis III
The purpose of this study is to assess rates of decline in motor and cognitive functional measures, and to assess potential biomarkers, in order to identify potential outcome measure appropriate for use in therapeutic clinical…
- Trial name
- NCT02037880
- Testing
- Lumbar puncture, Magnetic Resonance Imaging (MRI) of the brain
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Nationwide Children's Hospital
Mucopolysaccharidosis Type IIIA
Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
MPS IIIA is predominantly a central nervous system disease causing cognitive disability, progressive loss of acquired skills, behavioral and sleep disturbance.
- Trial name
- AAVance (NCT03612869)
- Testing
- LYS-SAF302
- Where
- 8 sites in United States, France and 3 more
- Volunteers
- 20 people
- Sponsor
- LYSOGENE
Morquio B Disease, GM1 Gangliosidosis Type III
Natural History of Morquio B and Late-Onset of GM1 Gangliosidosis
Mucopolysaccharidosis type IVB (Morquio-B disease, MBD) is an autosomal-recessive lysosomal disease caused by mutations in a gene called GLB1.
- Trial name
- NCT04320329
- Where
- Canada
- Volunteers
- 30 people
- Sponsor
- University of British Columbia
Sanfilippo Syndrome Type C
Natural History Study of Participants With Sanfilippo Syndrome Type IIIC
This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with MPS IIIC.
- Trial name
- MPSIIIC (NCT05825131)
- Where
- 2 sites in United States, France
- Volunteers
- 30 people
- Sponsor
- Phoenix Nest
Lysosomal Storage Disease, Peroxisomal Disorder
Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
This study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) in patients with high-risk lysosomal and peroxisomal disorders using a novel…
- Trial name
- NCT01626092
- Testing
- Campath-1H, Clofarabine, Melphalan
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
MPS IIIB
Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)
The primary objectives of this study are to evaluate the effects of Tralesinidase Alfa (TA) on cognition
- Trial name
- TrAnsform (NCT07579910)
- Testing
- Tralesinidase alfa (TA)
- Volunteers
- 14 people
- Sponsor
- Spruce Biosciences
Mucopolysaccharidosis I, Mucopolysaccharidosis II
Clinical Trial of Growth Hormone in MPS I, II, and VI
The purpose of this study is to determine whether growth hormone is a safe and effective treatment for short stature in children with Mucopolysaccharidosis type I, II, and VI.
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI
- Trial name
- NCT00748969
- Testing
- Somatropin (DNA origin)
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center