Extension Study for Patients Who Have Participated in a BMN 701 Study

NCT ID: NCT01435772

Last Updated: 2018-05-22

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 21 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-08-15
• Study Completion Date: 2016-09-09

Brief Summary

This is a Phase 2 open-label, multiple dose study of BMN 701 administered by IV infusion every 2 weeks (qow) to patients with late-onset Pompe disease.

Conditions

Pompe Disease

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

BMN 701 20mg/kg

BMN 701 20mg/kg IV every other week

Group Type: EXPERIMENTAL

BMN 701

Intervention Type: BIOLOGICAL

GILT-tagged recombinant human GAA

BMN 701 10mg/kg

BMN 701 10mg/kg IV every other week

Group Type: EXPERIMENTAL

BMN 701

Intervention Type: BIOLOGICAL

GILT-tagged recombinant human GAA

BMN 701 5mg/kg

BMN 701 5mg/kg IV every other week

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

• Have completed a prior BMN 701 clinical development study;
• Have provided written informed consent after the nature of the study has been explained prior to performance of any study-related procedures. Minors may participate as long as they provide written assent after the nature of the study has been explained to them and after their parent, or legal guardian has provided written informed consent, prior to the performance of any study-related procedures;
• Have been diagnosed with late-onset Pompe Disease, based on the entry criteria of a prior BMN 701 study;
• If sexually active, be willing to use 2 known effective methods of contraception from Screening until 4 months after the last dose of study-drug;
• If female, and not considered to be of childbearing potential, be at least 2 years post-menopausal, or have had tubal ligation at least 1 year prior to screening, or have had a total hysterectomy;
• If female, and of childbearing potential, have a negative pregnancy test during the Screening Period and at the Baseline visit, and be willing to have additional pregnancy tests during the study;
• Have the ability to comply with the protocol requirements, in the opinion of the Investigator.

Exclusion Criteria

• Have received any experimental or approved therapy for Pompe disease, other than BMN 701, subsequent to completion of a BMN 701 study and prior to entry into POM-002;
• Have received, or are anticipated to receive, any investigational medication, other than BMN 701, within 30 days prior to the first dose of study-drug;
• Are breastfeeding at screening or planning to become pregnant (self or partner) at any time during the study;
• Have 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.

• 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

San Diego, California, United States

University of Florida College of Medicine

Gainesville, Florida, United States

Tampa General Hospital

Tampa, Florida, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

Royal Brisbane and Women's Hospital

Herston, Queensland, Australia

Royal Adelaide Hospital

North Adelaide, South Australia, Australia

Hôpital Pitié-Salpêtrière

Paris, , France

Villa Metabolica, ZKJM MC University Mainz

Mainz, , Germany

Auckland City and Starship Children's Hospital

Auckland, , New Zealand

University Hospitals Birmingham NHS Foundation Trust

Birmingham, , United Kingdom

Royal Free Hospital

London, , United Kingdom

Salford Royal NHS Foundation Trust

Salford, , United Kingdom

Countries

United States; Australia; France; Germany; New Zealand; United Kingdom

Other Identifiers

2011-001805-28

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

POM-002

Identifier Type: -

Identifier Source: org_study_id