Clinical trial NCT03071341: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Mucopolysaccharidosis I
Keywords: MPS I; Hurler Syndrome
Lead sponsor: ArmaGen, Inc
Sponsor class: INDUSTRY
First posted: 2017-03-06
Enrollment: 19
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
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)
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: number of patients with adverse events as a measure of safety and tolerability
timeFrame: 24 months
Secondary outcomes:
measure: Total urinary glycosaminoglycans (GAGs)
timeFrame: 24 months
measure: Urinary heparan sulfate and dermatan sulfate
timeFrame: 24 months
measure: Plasma heparan sulfate and dermatan sulfate
timeFrame: 24 months
measure: CSF heparan sulfate and dermatan sulfate
timeFrame: 24 months
measure: liver and/or spleen volume
timeFrame: 24 months
Other outcomes:
Locations:
city: Porto Alegre
country: Brazil
facility: HCPA - Hospital das Clinicas de Porto Alegre
state: RS
Study officials:
affiliation: ArmaGen, Inc
name: Patrice P Rioux, MD PhD
role: STUDY_DIRECTOR
References:
citation: 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.