Principal Investigator: Dave Viskochil
Keywords: MPS I , Hurler Syndrome , Hurler-Scheie Syndrome , Scheie Syndrome Department: Pediatric Genetics
IRB Number: 00095859 Co Investigator: Nicola Longo
Specialty: Pediatric Genetics
Sub Specialties: Medical Genetics
Recruitment Status: Enrolling by invitation

Contact Information

Carrie Bailey

Brief Summary

This is an open-label, multi-dose safety extension study of AGT-181 for treatment of adult patients
with MPS I.
This study will enroll the patients who have completed protocol AGT-181-102 where the patient, sponsor and investigator believe the patient may potentially benefit by continuing to receive AGT-181. Patients who enter the trial from early cohorts (for example 1 mg/kg) will continue on their assigned dose from the prior study until safety from the highest dose cohort is assessed. After safety and tolerability of the highest dose is known, patients may have their dose changed.
Primary Objective:
To obtain additional safety and tolerability information by continuing weekly treatment of AGT-181 in adults with MPS I who have completed study AGT-181-102.
Secondary Objectives:
1. To determine longer term effects on urinary glycosaminoglycans (GAGs).
2. To determine longer term effects of AGT-181 on urinary and plasma heparan sulfate and dermatan sulfate.
3. To determine the longer term effects of AGT-181 treatment on liver and spleen size.
4. To determine the longer term effects on levels of heparan sulfate and dermatan sulfate in the cerebrospinal fluid.

Inclusion Criteria

1. Patients must be 18 years of age or older.
2. The patient must have completed clinical trial AGT-181-102
3. Patient must have signed and dated the informed consent document indicating that the patient
has been informed of all pertinent aspects of this extension trial.
4. A female patient of childbearing potential must have a negative pregnancy test (urine B–human
chorionic gonadotropin) at entry (prior to the first infusion). All women of childbearing potential and sexually mature men must agree use a highly reliable method of birth control by using two forms of contraception (for example oral contraceptive plus condom, two barrier method, etc.). throughout the study (Refer to sections 8.9-8.10). Women of childbearing potential must be willing to undergo periodic pregnancy tests during the course of the study.

Exclusion Criteria

1. The patient refuses to complete all screening evaluations.
2. The patient developed a medical condition, serious intercurrent illness, or other extenuating
circumstance that, in the opinion of the investigator, may significantly interfere with study
compliance, including all prescribed evaluations and follow-up activities.
3. The patient is pregnant or lactating.
4. The patient has known clinically significant spinal cord compression or evidence of such on
neurological exam, or evidence of cervical instability.
5. The patient developed clinically relevant hypersentivity/anaphylactoid reactions to AGT-181.