Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181

Mucopolysaccharidosis I Clinical Trial

Official title:

An Extension Study of AGT-181-102 Evaluating Safety and Glycosaminoglycans (GAGs) in Adult Patients With Hurler-Scheie or Scheie Syndrome Who Have Completed 8-Weeks of Dosing With AGT-181 in Study AGT-181-102

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02597114
• Other study ID #: AGT-181-103
• Status: Completed
• Phase: Phase 1
• Start date: November 2015
• Est. completion date: August 2, 2018

Study information

• Verified date: September 2018
• 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 study is an extension of a safety and dose ranging study to obtain long term safety and exposure data, as well as information on the biological activity of the investigational drug

Recruitment information / eligibility

• Status: Completed
• Enrollment: 3
• Est. completion date: August 2, 2018
• Est. primary completion date: March 19, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Must have completed clinical trial AGT-181-102

• Voluntary written consent by patient or legally responsible representative

• All women of childbearing potential and sexually mature males must be advised to use a medically accepted method of contraception throughout the study.

• Negative pregnancy test (females)

Exclusion Criteria:

• Refusal to complete screening evaluations.

• Any medical condition or other circumstances that may significantly interfere with study compliance

• Patient is pregnant or lactating

• Clinically significant spinal cord compression, evidence of cervical instability.

• Subject developed clinically relevant hypersensitivity to AGT-181

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis I

Intervention

Drug:
• AGT-181
intravenous infusion over 3-4 hours

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
ArmaGen, Inc

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with adverse events as a measure of safety and tolerability		104 weeks (2 years)
Secondary	changes in urinary or plasma glycosaminoglycans (GAGs)		104 weeks (2 years)
Secondary	change in liver size		104 weeks (2 years)
Secondary	change in spleen size		104 weeks (2 years)
Secondary	change in levels of heparan sulfate and dermatan sulfate in the cerebrospinal fluid		104 weeks (2 years)