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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 : September 18, 2018
Sponsor:
Information provided by (Responsible Party):
ArmaGen, Inc

Tracking Information
First Submitted Date  ICMJE February 27, 2017
First Posted Date  ICMJE March 6, 2017
Last Update Posted Date September 18, 2018
Actual Study Start Date  ICMJE October 2016
Actual Primary Completion Date February 1, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 1, 2017)
number of patients with adverse events as a measure of safety and tolerability [ Time Frame: 24 months ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (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 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I
Official Title  ICMJE 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 Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE 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  ICMJE Mucopolysaccharidosis I
Intervention  ICMJE Drug: AGT-181
Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein
Study Arms  ICMJE 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 16, 2018)
19
Original Estimated Enrollment  ICMJE
 (submitted: March 1, 2017)
11
Actual Study Completion Date  ICMJE February 1, 2018
Actual Primary Completion Date February 1, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Brazil
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03071341
Other Study ID Numbers  ICMJE 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  ICMJE
Plan to Share IPD: No
Responsible Party ArmaGen, Inc
Study Sponsor  ICMJE ArmaGen, Inc
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Patrice P Rioux, MD PhD ArmaGen, Inc
PRS Account ArmaGen, Inc
Verification Date September 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP