Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03071341 |
Recruitment Status :
Completed
First Posted : March 6, 2017
Last Update Posted : March 15, 2023
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
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.
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Mucopolysaccharidosis I | Drug: AGT-181 | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 19 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Intervention Model Description: | Patients will be assigned to 1.0, 3.0 or 6.0 mg/kg (same dose taken during the previous study, AGT-181-101) |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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 |
Actual Study Start Date : | October 2016 |
Actual Primary Completion Date : | February 1, 2018 |
Actual Study Completion Date : | February 1, 2018 |
Arm | Intervention/treatment |
---|---|
Experimental: AGT-181
Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) fusion protein
|
Drug: AGT-181
Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein |
- number of patients with adverse events as a measure of safety and tolerability [ Time Frame: 24 months ]Incidence and prevalence of adverse events
- Total urinary glycosaminoglycans (GAGs) [ Time Frame: 24 months ]Change in total urinary glycosaminoglycans (GAGs)
- Urinary heparan sulfate and dermatan sulfate [ Time Frame: 24 months ]Change in urinary heparan sulfate and dermatan sulfate
- Plasma heparan sulfate and dermatan sulfate [ Time Frame: 24 months ]Change in plasma heparan sulfate and dermatan sulfate
- CSF heparan sulfate and dermatan sulfate [ Time Frame: 24 months ]Change in CSF heparan sulfate and dermatan sulfate
- liver and/or spleen volume [ Time Frame: 24 months ]Change in liver and/or spleen volume measured by MRI
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 2 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03071341
Brazil | |
HCPA - Hospital das Clinicas de Porto Alegre | |
Porto Alegre, RS, Brazil, 90035-903 |
Study Director: | Patrice P Rioux, MD PhD | ArmaGen, Inc |
Responsible Party: | ArmaGen, Inc |
ClinicalTrials.gov Identifier: | NCT03071341 |
Other Study ID Numbers: |
AGT-181-101E |
First Posted: | March 6, 2017 Key Record Dates |
Last Update Posted: | March 15, 2023 |
Last Verified: | March 2023 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
MPS I; Hurler Syndrome |
Mucopolysaccharidoses Mucopolysaccharidosis I Carbohydrate Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn |
Lysosomal Storage Diseases Mucinoses Connective Tissue Diseases Metabolic Diseases |