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

ArmaGen

Status and phase: Completed; Phase 1

Conditions: Mucopolysaccharidosis I

Treatments: Drug: AGT-181

Study type: Interventional

Funder types: Industry

Identifiers

NCT02597114

AGT-181-103

Details and patient eligibility

About

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

Enrollment: 3 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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3 participants in 1 patient group

AGT-181

Experimental group

Description:

AGT-181 (fusion protein of anti-human insulin receptor monoclonal antibody and alpha-L-iduronidase) administered once weekly x 26 weeks at same dose level as subject received in core study

Treatment:

Drug: AGT-181