Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

NCT ID: NCT01230801

Last Updated: 2018-06-11

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 22 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-01-17
• Study Completion Date: 2013-03-06

Brief Summary

A Phase 1/2, open-label, multicenter, multiple dose escalation study of BMN 701 administered by intravenous infusion every 2 weeks over a 24-week treatment period to patients with late-onset Pompe disease.

Conditions

Pompe Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

BMN 701

IV infusion

Group Type: EXPERIMENTAL

BMN 701

Intervention Type: BIOLOGICAL

GILT-tagged recombinant human GAA

Interventions

BMN 701

GILT-tagged recombinant human GAA

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

• Patient has been diagnosed with Pompe Disease prior to or during the screening period based on 2 GAA gene mutations and either: endogenous GAA activity <75% of the lower limit of the normal adult range reported by the testing laboratory, as assessed in cultured skin fibroblasts -or- 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 13 years of age or older at the time of enrollment in the study;
• Sexually active patients 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;
• If patient is female and not considered to be of childbearing potential, she is at least 2 years post-menopausal or had tubal ligation at least 1 year prior to screening, or who have had total hysterectomy;
• If patient is female and of childbearing potential, she has negative urine pregnancy tests during the Screening Period and at the Baseline visit and be willing to have additional pregnancy tests during the study;
• 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 Enzyme Replacement Therapy (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
• If subject was female, she was not lactating

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.

• Minimum Eligible Age: 13 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

BioMarin Pharmaceutical

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Monitor

Role: STUDY_DIRECTOR

BioMarin Pharmaceutical

Locations

Univ of California San Diego School of Medicine

La Jolla, California, United States

University of Florida College of Medicine

Gainesville, Florida, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

Royal Adelaide Hospital, SA Pathology

Adelaide, Adelaide, SA, Australia

Hôpital de I´Archet- Centre Hospitalier Universitaire Nice

Nice, , France

Hôpital Pitié-Salpêtrière

Paris, , France

Zentrum für Kinder- und Jugenmedizin

Mainz, Rhineland-Palatinate, Germany

Old Queen Elizabeth Hospital, Department of Medicine

Birmingham, , United Kingdom

Royal Free Hospital

London, , United Kingdom

Salford Royal Hospital NHS Trust

Salford, , United Kingdom

Countries

United States; Australia; France; Germany; United Kingdom

Other Identifiers

2010-023561-22

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

POM-001

Identifier Type: -

Identifier Source: org_study_id