Back to Results

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

Interested in this Trial?

Contact Us

Trial Status Terminated

Trial Identifier

NCT01435772

Condition

Pompe Disease

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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.

Eligibility Criteria

Birth Sex

All

Age

From 13 Years

Healthy Volunteers

No

Drug/Treatment:

BMN 701

Phase:

Phase 2

Study Type:

Interventional

Number of Participants:

21

Study Started:

2011-08-15

Study Updated:

2018-05-22

Trial 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

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.

Contact BioMarin Clinical Trials

Please complete and submit the following form to express interest in participating and request more information about one of our active clinical trials

*required fields

"*" indicates required fields

Please select the trial that you are interested in from the dropdown below. Note that this list only includes currently active trials. If you cannot find the trial you are seeking, please enter the study identifier and title in the comments box below, along with any other details you would like us to know to help address your inquiry.
Consent*
Consent*
For more Information about BioMarin's privacy obligations or to exercise your privacy rights, please visit BioMarin's Data Privacy Center.
This field is hidden when viewing the form
This field is hidden when viewing the form
This field is hidden when viewing the form
This field is hidden when viewing the form

What’s Next?

Visit the following links to learn more about clinical trials.

Discuss this trial with your care team.

You may wish to print this page to share the details with your healthcare providers and discuss whether you may be eligible.

Print

Get in touch.

Contact Us