A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study

Phase 1

• Conditions: Pompe disease; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]; MedDRA version: 18.0Level: LLTClassification code 10036143Term: Pompe's diseaseSystem Organ Class: 100000004850

• Registration Number: EUCTR2011-001805-28-GB
• Lead Sponsor: BioMarin Pharmaceutical Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2012-04-26
• Study Completion: 2016-09-09
• Last Update Posted: 27 July 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 21

Inclusion Criteria

• Participated in 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.

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 22
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 0

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified