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

Stopped early · Phase 2

Conditions studied: Pompe Disease

In brief

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.

Key facts

Study ID

NCT01435772

Run by

BioMarin Pharmaceutical

People needed

21

Starts

2011-08-15

Expected to finish

2016-09-09

Last updated by the study team

2018-05-22

Who can join

Age: 13 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• 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

Full record on ClinicalTrials.gov

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