Mucopolysaccharidosis I Clinical Trial
Official title:
An Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Patients With Mucopolysaccharidosis I Who Were Previously Enrolled in Studies With AGT-181
Verified date | March 2023 |
Source | ArmaGen, Inc |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
AGT-181 is a fusion protein containing alpha-L-iduronidase that is intended to deliver the enzyme peripherally and to the brain, when administered intravenously. This is a long term safety and tolerability study of AGT-181 in patients with MPS I who completed the previous 26-week study, AGT-181-101. Information on the biological activity of the investigational drug will also be collected.
Status | Completed |
Enrollment | 19 |
Est. completion date | February 1, 2018 |
Est. primary completion date | February 1, 2018 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 2 Years and older |
Eligibility | Inclusion Criteria: - 2 years of age or older (and less than 18) - Must have been previously enrolled in study AGT-181-101 - Written consent and assent as required - Female patients must not be pregnant, willing to utilize appropriate birth control methods and undergo pregnancy testing during the study Exclusion Criteria: - Refusal to complete all assessments - Pregnant or Lactating - Received investigational drug within 1 year prior to study enrollment - Medical condition or extenuating circumstance that, in the opinion of the investigator, may interfere with study compliance - Clinically significant spinal cord compression or evidence of cervical instability (i.e. expected to require intervention during study participation) - Has developed clinically relevant hypersensitivity/anaphylactoid reactions to AGT-181 |
Country | Name | City | State |
---|---|---|---|
Brazil | HCPA - Hospital das Clinicas de Porto Alegre | Porto Alegre | RS |
Lead Sponsor | Collaborator |
---|---|
ArmaGen, Inc |
Brazil,
Giugliani R, Giugliani L, de Oliveira Poswar F, Donis KC, Corte AD, Schmidt M, Boado RJ, Nestrasil I, Nguyen C, Chen S, Pardridge WM. Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | number of patients with adverse events as a measure of safety and tolerability | Incidence and prevalence of adverse events | 24 months | |
Secondary | Total urinary glycosaminoglycans (GAGs) | Change in total urinary glycosaminoglycans (GAGs) | 24 months | |
Secondary | Urinary heparan sulfate and dermatan sulfate | Change in urinary heparan sulfate and dermatan sulfate | 24 months | |
Secondary | Plasma heparan sulfate and dermatan sulfate | Change in plasma heparan sulfate and dermatan sulfate | 24 months | |
Secondary | CSF heparan sulfate and dermatan sulfate | Change in CSF heparan sulfate and dermatan sulfate | 24 months | |
Secondary | liver and/or spleen volume | Change in liver and/or spleen volume measured by MRI | 24 months |
Status | Clinical Trial | Phase | |
---|---|---|---|
Terminated |
NCT01675674 -
Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics
|
N/A | |
Completed |
NCT00741338 -
Immune Tolerance Study With Aldurazyme® (Laronidase)
|
Phase 1/Phase 2 | |
Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
Completed |
NCT04227600 -
A Study of JR-171 in Patients With Mucopolysaccharidosis I
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT04453085 -
An Extension Study of JR-171-101 Study in Patients With MPS I
|
Phase 1/Phase 2 | |
Completed |
NCT05134571 -
China Post-marketing Surveillance (PMS) Study of Aldurazyme®
|
Phase 4 | |
Terminated |
NCT00418821 -
A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
|
Phase 4 | |
Completed |
NCT03053089 -
Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS I
|
Phase 1/Phase 2 | |
Completed |
NCT00146757 -
A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old
|
Phase 2 | |
Active, not recruiting |
NCT04628871 -
Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
|
||
Completed |
NCT02597114 -
Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181
|
Phase 1 | |
Terminated |
NCT00748969 -
Clinical Trial of Growth Hormone in MPS I, II, and VI
|
Phase 2/Phase 3 | |
Terminated |
NCT00215527 -
Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
|
Phase 1 | |
Recruiting |
NCT05619900 -
Registry of Patients Diagnosed With Lysosomal Storage Diseases
|
||
Completed |
NCT00146770 -
Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
|
Phase 3 | |
Completed |
NCT00176917 -
Stem Cell Transplantation for Hurler
|
Phase 2 | |
Completed |
NCT00176891 -
Stem Cell Transplant w/Laronidase for Hurler
|
Phase 2 | |
Active, not recruiting |
NCT03153319 -
Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI
|
Phase 1/Phase 2 | |
Completed |
NCT00852358 -
A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
|
N/A | |
Enrolling by invitation |
NCT06103487 -
Long Term Follow-Up for RGX-111
|