Clinical trials: MPS I
Mucopolysaccharidosis I
A Study of JR-171 in Patients With Mucopolysaccharidosis I
Phase I/II, open-label, multicenter, multinational (Japan, Brazil and US),designed to evaluate the safety, pharmacokinetics and explore the efficacy for the treatment of mucopolysaccharidosis type I (MPS I).
- Trial name
- NCT04227600
- Testing
- JR-171 (lepunafusp alfa)
- Where
- 6 sites in United States, Brazil and 1 more
- Volunteers
- 18 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
Mucopolysaccharidosis II, MPS II
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913.
- Trial name
- NCT03041324
- Testing
- SB-913
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Sangamo Therapeutics
Inherited Metabolic Disorders (IMD)
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
This study is designed to assess the safety and efficacy of using MGTA-456 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
- Trial name
- NCT03406962
- Testing
- MGTA-456
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Magenta Therapeutics, Inc.
Hunter Syndrome
A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®
Elaprase (idursulfase), a large molecular protein, is not expected to cross the blood brain barrier at therapeutic levels when administered intravenously.
- Trial name
- NCT00920647
- Testing
- Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg)
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 16 people
- Sponsor
- Shire
MPS I
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I
The purpose of the study is to evaluate the safety, tolerability of ascending doses of SB-318. SB-318 is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing.
- Trial name
- NCT02702115
- Testing
- SB-318
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Sangamo Therapeutics
Mucopolysaccharidosis I, Hurler's Syndrome
A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease
The main purpose of this study is to evaluate differences in the pharmacodynamic response of 4 Aldurazyme® (laronidase) dose regimens in patients with Mucopolysaccharidosis I (MPS I).
- Conditions
- Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
- Trial name
- NCT00144781
- Testing
- Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
- Where
- 5 sites in Brazil, Canada
- Volunteers
- 34 people
- Sponsor
- Genzyme, a Sanofi Company
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
Mucopolysaccharidosis I, Hurlers Syndrome
Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I
This study is being conducted to demonstrate the safety and clinical efficacy of Aldurazyme treatment in MPS I patients
- Conditions
- Mucopolysaccharidosis I, Hurlers Syndrome, Hurler-Scheie Syndrome
- Trial name
- NCT00912925
- Testing
- rhIDU (recombinant human-Alpha-L-Iduronidase)
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 45 people
- Sponsor
- Genzyme, a Sanofi Company
Mucopolysaccharidosis I
Immune Tolerance Study With Aldurazyme® (Laronidase)
The purpose of this study is to see if treatment with an antigen-specific immunosuppressive can decrease or stop an antibody response to laronidase (Aldurazyme®) during enzyme replacement therapy with laronidase in severe…
- Trial name
- NCT00741338
- Testing
- Laronidase, Cyclosporine A (CsA), Azathioprine (Aza)
- Where
- 2 sites in Brazil, Russia
- Volunteers
- 7 people
- Sponsor
- Genzyme, a Sanofi Company
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 I, Hurler's Syndrome
Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
This study is being conducted to collect additional long-term efficacy and safety data of Aldurazyme® (laronidase) patients with MPS I disease.
- Conditions
- Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
- Trial name
- NCT00146770
- Testing
- Aldurazyme
- Where
- 25 sites in United States, Brazil and 5 more
- Volunteers
- 45 people
- Sponsor
- Genzyme, a Sanofi Company
Mucopolysaccharidosis IIIB
Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)
Study to evaluate the safety and tolerability of intravenous (IV) administration of SBC-103 in participants with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) with evaluable signs or symptoms of developmental delay.
- Trial name
- NCT02324049
- Testing
- SBC-103
- Where
- 4 sites in United States, Spain and 1 more
- Volunteers
- 11 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Mucopolysaccharidosis III, Type B (MPS IIIB), Sanfilippo B
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
This study evaluated the safety and tolerability of intravenous (IV) administration of SBC-103 in previously studied, SBC-103 treatment naïve patients with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) who…
- Trial name
- CL01-T (NCT02618512)
- Testing
- SBC-103
- Where
- United Kingdom
- Volunteers
- 3 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Mucopolysaccharidosis I, Hurler Syndrome
A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old
The main objectives of this study are to evaluate the safety and pharmacokinetics (PK) of enzyme replacement therapy with recombinant human alpha-L-iduronidase [Aldurazyme® (laronidase)] in mucopolysaccharidosis I (MPS I)…
- Conditions
- Mucopolysaccharidosis I, Hurler Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
- Trial name
- NCT00146757
- Testing
- Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
- Where
- 4 sites in France, Germany and 2 more
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
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
Mucopolysaccharidosis (MPS)
Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients
Sanfilippo syndrome, or Mucopolysaccharidosis (MPS) III, is a rare lysosomal storage disease (LSD) caused by loss in activity of 1 of 4 enzymes necessary for degradation of the glycosaminoglycan (GAG) heparan sulfate (HS) in…
- Trial name
- NCT01155778
- Testing
- Recombinant human heparan N-sulfatase (rhHNS)
- Where
- 2 sites in Netherlands, United Kingdom
- Volunteers
- 12 people
- Sponsor
- Shire