Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

3 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-07-31

Study Completion Date

2017-02-01

Brief Summary

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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 a safety and dose ranging study to obtain safety and exposure data, as well as information on the biological activity of the investigational drug.

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Detailed Description

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Conditions

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Mucopolysaccharidosis I

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Open Label

Study Groups

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Cohort 1

1 mg/kg AGT-181 (fusion protein of anti-human insulin receptor monoclonal antibody and alpha-L-iduronidase; HIRMAb-IDUA) administered once weekly x 8 weeks

Group Type EXPERIMENTAL

AGT-181 (HIRMAb-IDUA)

Intervention Type DRUG

intravenous infusion over 3-4 hours

Cohort 2

3 mg/kg AGT-181 (HIRMAb-IDUA) administered once weekly x 8 weeks

Group Type EXPERIMENTAL

AGT-181 (HIRMAb-IDUA)

Intervention Type DRUG

intravenous infusion over 3-4 hours

Cohort 3

6-9 mg/kg AGT-181 (HIRMAb-IDUA) administered once weekly x 8 weeks

Group Type EXPERIMENTAL

AGT-181 (HIRMAb-IDUA)

Intervention Type DRUG

intravenous infusion over 3-4 hours

Interventions

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AGT-181 (HIRMAb-IDUA)

intravenous infusion over 3-4 hours

Intervention Type DRUG

Other Intervention Names

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fusion protein of monoclonal antibody to human insulin receptor fused to alpha-L-iduronidase

Eligibility Criteria

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Inclusion Criteria

* Male age 18 years or older
* Diagnosis of MPS I (documented fibroblast or leukocyte IDUA enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory)
* 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)
* Must not have received ERT for at least 6 weeks prior to AGT-181 treatment
* Must have elevated urinary GAGs if no ERT has been received in the prior 3 months

Exclusion Criteria

* Refusal to complete baseline evaluations.
* Any medical condition or other circumstances that may significantly interfere with study compliance
* Receipt of an investigational drug within the prior 90 days
* History of diabetes mellitus or hypoglycemia
* Clinically significant spinal cord compression, evidence of cervical instability.
* Known hypersensitivity to alpha-L-iduronidase or any of the components of AGT-181.
* Known to be nonresponsive to standard ERT treatment.
* Previously successful (engrafted) hematopoietic stem cell transplantation that resulted in normalization of urinary GAGs.
* Contraindication for lumbar puncture

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No