Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I

• ClinicalTrials.gov Identifier: NCT03071341
• Recruitment Status: Completed
• First Posted: March 6, 2017
• Last Update Posted: March 15, 2023
• Sponsor: ArmaGen, Inc
• Information provided by (Responsible Party): ArmaGen, Inc

Tracking Information

• First Submitted Date: February 27, 2017
• First Posted Date: March 6, 2017
• Last Update Posted Date: March 15, 2023
• Actual Study Start Date: October 2016
• Actual Primary Completion Date: February 1, 2018 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: February 19, 2023)

number of patients with adverse events as a measure of safety and tolerability [ Time Frame: 24 months ]

Incidence and prevalence of adverse events

• Original Primary Outcome Measures (submitted: March 1, 2017): number of patients with adverse events as a measure of safety and tolerability [ Time Frame: 24 months ]

Current Secondary Outcome Measures (submitted: February 19, 2023)

• 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

Original Secondary Outcome Measures (submitted: March 1, 2017)

• change in total urinary glycosaminoglycans (GAGs) [ Time Frame: 24 months ]
• change in urinary heparan sulfate and dermatan sulfate [ Time Frame: 24 months ]
• change in plasma heparan sulfate and dermatan sulfate [ Time Frame: 24 months ]
• change in CSF heparan sulfate and dermatan sulfate [ Time Frame: 24 months ]
• change in liver and/or spleen volume (measured by MRI) [ Time Frame: 24 months ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I
• 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

Brief Summary

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.

• Detailed Description: AGT-181-101E is a 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 be collected as well as longitudinal safety information.
• Study Type: Interventional
• Study Phase: Phase 1; Phase 2

Study Design

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

• Condition: Mucopolysaccharidosis I

Intervention

Drug: AGT-181

Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein

Study Arms

Experimental: AGT-181

Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) fusion protein

Intervention: Drug: AGT-181

• Publications *: 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 intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial. Orphanet J Rare Dis. 2018 Jul 5;13(1):110. doi: 10.1186/s13023-018-0849-8.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: September 16, 2018): 19
• Original Estimated Enrollment (submitted: March 1, 2017): 11
• Actual Study Completion Date: February 1, 2018
• Actual Primary Completion Date: February 1, 2018 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 2 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Brazil

Administrative Information

• NCT Number: NCT03071341
• Other Study ID Numbers: AGT-181-101E
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: ArmaGen, Inc
• Original Responsible Party: Same as current
• Current Study Sponsor: ArmaGen, Inc
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Patrice P Rioux, MD PhD; ArmaGen, Inc

• PRS Account: ArmaGen, Inc
• Verification Date: March 2023