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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

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.


Condition or disease Intervention/treatment Phase
Mucopolysaccharidosis I Drug: AGT-181 Phase 1 Phase 2

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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




Primary Outcome Measures :
  1. number of patients with adverse events as a measure of safety and tolerability [ Time Frame: 24 months ]

Secondary Outcome Measures :
  1. change in total urinary glycosaminoglycans (GAGs) [ Time Frame: 24 months ]
  2. change in urinary heparan sulfate and dermatan sulfate [ Time Frame: 24 months ]
  3. change in plasma heparan sulfate and dermatan sulfate [ Time Frame: 24 months ]
  4. change in CSF heparan sulfate and dermatan sulfate [ Time Frame: 24 months ]
  5. change in liver and/or spleen volume (measured by MRI) [ Time Frame: 24 months ]


Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

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


Locations
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Brazil
HCPA - Hospital das Clinicas de Porto Alegre
Porto Alegre, RS, Brazil, 90035-903
Sponsors and Collaborators
ArmaGen, Inc
Investigators
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Study Director: Patrice P Rioux, MD PhD ArmaGen, Inc
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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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: September 18, 2018
Last Verified: September 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by ArmaGen, Inc:
MPS I; Hurler Syndrome
Additional relevant MeSH terms:
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Mucopolysaccharidoses
Mucopolysaccharidosis I
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases