Clinical trials: Mucopolysaccharidosis Type I
Mucopolysaccharidosis IIIB
A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)
This is a global, open-label, Phase I/II, interventional trial in participants younger than 6 years of age with Mucopolysaccharidosis Type IIIB (MPS IIIB), designed to assess the safety and tolerability of JR-446, determine its…
- Trial name
- NCT07640984
- Testing
- JR-446
- Where
- 5 sites in United States, Germany and 1 more
- Volunteers
- 12 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
MPS-IH (Hurler Syndrome)
A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT
A multi-center randomized clinical trial to compare OTL-203 (gene therapy) with stem cell transplant (standard of care) in patients with MPS-IH (Hurler syndrome).
- Trial name
- HURCULES (NCT06149403)
- Testing
- Experimental: OTL-203, Active Comparator: Allo-HSCT
- Where
- 5 sites in United States, Italy and 2 more
- Volunteers
- 41 people
- Sponsor
- Orchard Therapeutics
Mucopolysaccharidosis I (MPS I)
Mucopolysaccharidosis I (MPS I) Registry
The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I.
- Trial name
- NCT00144794
- Where
- 141 sites in United States, Argentina and 35 more
- Volunteers
- 1,406 people
- Sponsor
- Genzyme, a Sanofi Company
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
- Conditions
- Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III)
- Trial name
- NCT01372228
- Testing
- hematopoietic stem cell infusion
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Talaris Therapeutics Inc.
Metastatic Breast Cancer, Metastatic Triple Negative Breast Cancer
S 81694 Plus Paclitaxel in Metastatic Breast Cancer
The purpose of this study is to determine the safety profile, the maximum tolerated dose (MTD) and the associated dose-limiting toxicities (DLTs) of S 81694 in combination with paclitaxel in metastatic breast cancer (mBC)…
- Trial name
- NCT03411161
- Testing
- Combination therapy (S81694 + paclitaxel) phase I, Paclitaxel, Combination therapy (S81694 + paclitaxel) phase II
- Where
- 6 sites in Belgium, France and 2 more
- Volunteers
- 22 people
- Sponsor
- Institut de Recherches Internationales Servier
MPS I, MPS1-S
Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy
SIG-005-121 is a FIH, Phase 1/2, multi-centre, open-label, sequential dose-escalating study to assess the safety, tolerability, and preliminary efficacy of SIG-005 in adults with MPS-1.
- Conditions
- MPS I, MPS1-S, MPS1-HS
- Trial name
- NCT05665036
- Testing
- SIG-005 (hIDUA Producing Spheres)
- Where
- 3 sites in Brazil, United Kingdom
- Sponsor
- Sigilon Therapeutics, Inc.
Fibrodysplasia Ossificans Progressiva, FOP
A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP)
This study is researching an experimental drug called garetosmab, referred to as "study drug". The study is focused on children and adolescent participants with FOP.
- Trial name
- OPTIMA-2 (NCT07559513)
- Testing
- garetosmab
- Volunteers
- 18 people
- Sponsor
- Regeneron Pharmaceuticals
Mucopolysaccharidosis Type IIIA
Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA
Patients with MPS IIIA have a clinical disorder marked by severe and progressive brain disease and neurological symptoms due to the accumulation of undigested glycosaminoglycans in all cells of the body.
- Trial name
- NCT04201405
- Testing
- Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene
- Where
- United Kingdom
- Volunteers
- 5 people
- Sponsor
- University of Manchester
Inherited Metabolic Disorders (IMD)
A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456
A follow-up study to evaluate the safety and clinical outcomes of patients with inherited metabolic disorders (IMD) who have undergone hematopoietic stem cell transplantation (HSCT) with MGTA-456
- Trial name
- IMD-002 (NCT04008849)
- Testing
- Safety and efficacy assessments
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Magenta Therapeutics, Inc.
Mucopolysaccharidosis (MPS)
Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)
Mucopolysaccharidosis (MPS) type II (MPS II; Hunter syndrome) is a rare, X-linked disease caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase (I2S) and occurs almost exclusively in boys, with an incidence of…
- Trial name
- NCT02095015
- Where
- 14 sites in United States, Canada and 5 more
- Volunteers
- 159 people
- Sponsor
- Shire
Inherited Metabolic Diseases, Lysosomal Storage Disorders
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Eligible research subjects will receive an unrelated umbilical cord blood transfusion as a possible cure for their inherited metabolic disease.
- Conditions
- Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism
- Trial name
- UCBT-002 (NCT00654433)
- Testing
- ALD-101
- Where
- 3 sites in United States
- Volunteers
- 40 people
- Sponsor
- Aldagen
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
Mucopolysaccharidoses
Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI
Approximately 85% of individuals with Mucopolysaccharidosis (MPS) type I, II, or VI report weekly pain and 50-60% have significant limitations in their activities of daily living due to MPS related musculoskeletal disease despite…
- Trial name
- NCT01521429
- Where
- 3 sites in United States
- Volunteers
- 55 people
- Sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
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
Mucopolysaccharidosis Type 3 B
Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB
Open-label, dose-escalation clinical trial of rAAV9.CMV.hNAGLU injected intravenously through a peripheral limb vein
- Trial name
- MPSIIIB (NCT03315182)
- Testing
- rAAV9.CMV.hNAGLU
- Where
- 4 sites in United States, France and 2 more
- Volunteers
- 11 people
- Sponsor
- Abeona Therapeutics, Inc
Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome
RGX-111 Gene Therapy in Patients With MPS I
RGX-111 is a gene therapy which is intended to deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system.
- Conditions
- Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome
- Trial name
- NCT03580083
- Testing
- RGX-111
- Where
- 2 sites in United States, Brazil
- Volunteers
- 21 people
- Sponsor
- REGENXBIO Inc.
MPS I
The Intensively Follow-up Examinations for Asymptomatic MPS I Infants in Taiwan
MPS I newborn screening has been executed in Taiwan nationwide since August 2015. Infants who failed the recheck at recall were referred to MacKay Memorial Hospital for a detailed confirmatory diagnosis.
- Trial name
- NCT04958070
- Testing
- MPS I
- Where
- Taiwan
- Volunteers
- 20 people
- Sponsor
- Mackay Memorial Hospital
Mucopolysaccharidosis I, Hurler's Syndrome
A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
The purpose of this study is to determine if laronidase is present in the breast milk of post-partum women receiving Aldurazyme® (laronidase) and the effects of Aldurazyme (laronidase) on the growth, development, and immunologic…
- Conditions
- Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie
- Trial name
- NCT00418821
- Testing
- Laronidase
- Where
- Italy
- Volunteers
- 2 people
- Sponsor
- Genzyme, a Sanofi Company
Mucopolysaccharidosis Disorders, Hurler Syndrome
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based…
- Conditions
- Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome
- Trial name
- NCT02171104
- Testing
- Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen
- Where
- United States
- Volunteers
- 149 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Mucopolysaccharidosis I, Hurler Syndrome
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
This is a multi-center, open label, study conducted to evaluate the safety of laronidase administered by intravenous drip infusion in Japanese patients with MPS I disease.
- Conditions
- Mucopolysaccharidosis I, Hurler Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
- Trial name
- NCT00258011
- Testing
- Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
- Where
- 2 sites in Japan
- Volunteers
- 3 people
- Sponsor
- Genzyme, a Sanofi Company