A Study of the Safety and Tolerability of BMN 701 in Patients with Late-onset Pompe Disease

Conditions: Pompe disease
MedDRA version: 16.0Level: LLTClassification code 10036143Term: Pompe's diseaseSystem Organ Class: 100000004850
Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Registration Number: EUCTR2010-023561-22-DE

Lead Sponsor: BioMarin Pharmaceutical Inc

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 30

Inclusion Criteria

•Patient has provided written informed consent after the nature of the study has been explained, and prior to any study-related procedures;
• Patient has been diagnosed with Pompe disease, either prior to, or during the screening period based on 2 GAA gene mutations and either o endogenous GAA activity <75% of the lower limit of the normal adult range reported by the testing laboratory, as assessed in cultured skin fibroblasts,
o or,
o endogenous GAA activity <75% of the lower limit of the normal adult range reported by the testing laboratory, as assessed by dried blood spot or whole blood assay;
• Patient is male or female and 18 years of age or older at the time of enrollment in the study;
• The patient, if sexually active, must be willing to use an acceptable method of contraception while participating in the study and for at least 4 months following the last dose of BMN 701;
• The patient, if female, is not considered to be of childbearing potential: that is she is at least 2 years post-menopausal or had tubal ligation at least 1 year prior to screening, or has had a total hysterectomy;
• The patient, if female of childbearing potential, has negative urine pregnancy tests during the Screening Period and at the Baseline visit and is willing to have additional pregnancy tests during the study;
• If patient is female, she is not lactating;
• Patient has =30% predicted upright FVC and either <80% predicted upright FVC or > 10% reduction in supine FVC compared to upright FVC during the Screening Period;
• Patient is naïve to ERT with rhGAA;
• Patient must be able to ambulate at least 40 meters (131.2 feet) on the 6MWT conducted at the Screening visit (use of assistive devices such as walker, cane, or crutches, is permitted); and
• Patient has the ability to comply with the protocol requirements, in the opinion of the Investigator.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 28
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion Criteria

•Patient has a history of diabetes or other disease known to cause hypoglycemia and is currently receiving, or might anticipate receiving, hypoglycemic agents during the course of the study;
• Patient has been on any immunosuppressive medication other than glucocorticosteroids within 1 year prior to enrollment into this study;
• Patient requires invasive ventilatory assistance at the time of enrollment into the study;
• Patient has received any investigational medication within 30 days prior to the first dose of study drug or is scheduled to receive any investigational drug other than BMN 701 during the course of the study;
• Patient has previously been admitted to the study;
• Patient is breastfeeding at screening or planning to become pregnant (self or partner) at any time during the study;
• Patient has a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the patient’s ability to comply with the protocol requirements or compromise the patient’s well being or safety;
• Patient has any condition that, in the view of the Investigator, places the patient at high risk of poor treatment compliance or of not completing the study.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objectives of the study are:<br>• To evaluate the safety and tolerability of BMN 701<br>• To determine the anti-BMN 701 antibody response to BMN 701; and<br>• To determine the anti-IGF-I and anti-IGF-II antibody response to BMN 701.<br>;Secondary Objective: Secondary objectives of the study are,:<br>• To determine the single-dose and multiple-dose PK of BMN 701; and<br>• To determine mobility and functional exercise capacity, as measured by the Six-Minute Walk Test (6MWT).<br>;Primary end point(s): The primary objectives of the study are:<br>• To evaluate the safety and tolerability of BMN 701;<br>• To determine the anti-BMN 701 antibody response to BMN 701<br>;Timepoint(s) of evaluation of this end point: Screening <br>Baseline <br>Day 1<br>Week 2-24 qow<br>30 Days after Week 24

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Secondary objectives of the study are:<br>• To determine the single-dose and multiple-dose PK of BMN 701; and<br>• To determine mobility and functional exercise capacity, as measured by the Six-Minute Walk Test (6MWT).<br>;Timepoint(s) of evaluation of this end point: Screening<br>Baseline <br>Day 1<br>Week 2-24 qow<br>30 Days after Week 24

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