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

Mucopolysaccharidosis I Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03071341
• Other study ID #: AGT-181-101E
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: October 2016
• Est. completion date: February 1, 2018

Study information

• Verified date: March 2023
• Source: ArmaGen, Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 19
• Est. completion date: February 1, 2018
• Est. primary completion date: February 1, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis I

Intervention

Drug:
• AGT-181
Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein

Locations

Country	Name	City	State
Brazil	HCPA - Hospital das Clinicas de Porto Alegre	Porto Alegre	RS

Sponsors (1)

Lead Sponsor	Collaborator
ArmaGen, Inc

Country where clinical trial is conducted

Brazil, 

References & Publications (1)

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 — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	number of patients with adverse events as a measure of safety and tolerability	Incidence and prevalence of adverse events	24 months
Secondary	Total urinary glycosaminoglycans (GAGs)	Change in total urinary glycosaminoglycans (GAGs)	24 months
Secondary	Urinary heparan sulfate and dermatan sulfate	Change in urinary heparan sulfate and dermatan sulfate	24 months
Secondary	Plasma heparan sulfate and dermatan sulfate	Change in plasma heparan sulfate and dermatan sulfate	24 months
Secondary	CSF heparan sulfate and dermatan sulfate	Change in CSF heparan sulfate and dermatan sulfate	24 months
Secondary	liver and/or spleen volume	Change in liver and/or spleen volume measured by MRI	24 months