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

ArmaGen

Status and phase

Completed

Phase 2

Phase 1

Conditions

Mucopolysaccharidosis I

Treatments

Drug: AGT-181

Study type

Interventional

Funder types

Industry

Identifiers

NCT03071341

AGT-181-101E

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 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.

Full 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.

Enrollment

19 patients

Sex

All

Ages

2+ years old

Volunteers

No Healthy Volunteers

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