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

• Register: ClinicalTrials.gov
• Last refreshed on: 27 March 2023
• Main ID: NCT03071341
• Date of registration: 27/02/2017
• Prospective Registration: No
• Primary sponsor: ArmaGen, Inc
• Public title: Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I
• Scientific 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
• Date of first enrolment: October 2016
• Target sample size: 19
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03071341
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

Brazil

Contacts

• Name: Patrice P Rioux, MD PhD
• Affiliation: ArmaGen, Inc

Key inclusion & exclusion 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



Age minimum: 2 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Mucopolysaccharidosis I

Intervention(s)

Drug: AGT-181

Primary Outcome(s)

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

Secondary Outcome(s)

Total urinary glycosaminoglycans (GAGs) [Time Frame: 24 months]

Urinary heparan sulfate and dermatan sulfate [Time Frame: 24 months]

liver and/or spleen volume [Time Frame: 24 months]

CSF heparan sulfate and dermatan sulfate [Time Frame: 24 months]

Plasma heparan sulfate and dermatan sulfate [Time Frame: 24 months]

Secondary ID(s)

AGT-181-101E

Source(s) of Monetary Support

Please refer to primary and secondary sponsors