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

• Register: ClinicalTrials.gov
• Last refreshed on: 27 February 2023
• Main ID: NCT02371226
• Date of registration: 19/02/2015
• Prospective Registration: Yes
• Primary sponsor: ArmaGen, Inc
• Public title: Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I
• Scientific title: A Phase 1 Safety and Dose-Finding Study of a Human Insulin Receptor Monoclonal Antibody-Human Alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein, AGT-181 in Adult Patients With Mucopolysaccharidosis I (MPS I, Hurler Syndrome)
• Date of first enrolment: July 2015
• Target sample size: 3
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02371226
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Patrice Rioux, MD PhD
• Affiliation: ArmaGen, Inc

Key inclusion & exclusion criteria

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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Mucopolysaccharidosis I

Intervention(s)

Drug: AGT-181 (HIRMAb-IDUA)

Primary Outcome(s)

Number of participants with adverse events as a measure of safety and tolerability [Time Frame: eight weeks]

Secondary Outcome(s)

change in levels of heparan sulfate and dermatan sulfate in the cerebrospinal fluid [Time Frame: 8 weeks]

change in urinary or plasma glycosaminoglycans (GAGs) [Time Frame: 8 weeks]

change in liver size [Time Frame: 8 weeks]

change in spleen size [Time Frame: 8 weeks]

plasma pharmacokinetic parameters (maximal concentration, half-life, area under the curve, mean residence time, volume of distribution and clearance of AGT-181) [Time Frame: 8 weeks]

Secondary ID(s)

AGT-181-102

Source(s) of Monetary Support

Please refer to primary and secondary sponsors