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

BioMarin Pharmaceutical

Status and phase: Terminated; Phase 2

Conditions: Pompe Disease

Treatments: Biological: BMN 701

Study type: Interventional

Funder types: Industry

Identifiers

NCT01435772

2011-001805-28 (EudraCT Number)

POM-002

Details and patient eligibility

About

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.

Enrollment: 21 patients

Sex: All

Ages: 13+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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21 participants in 3 patient groups

BMN 701 20mg/kg

Experimental group

Description:

BMN 701 20mg/kg IV every other week

Treatment:

Biological: BMN 701

BMN 701 10mg/kg

Experimental group

Description:

BMN 701 10mg/kg IV every other week

Treatment:

Biological: BMN 701

BMN 701 5mg/kg

Experimental group

Description:

BMN 701 5mg/kg IV every other week

Treatment:

Biological: BMN 701