Clinical trial NCT01435772: Extension Study for Patients Who Have Participated in a BMN 701 Study
Official title: A Long-Term Study for Extended BMN 701 Treatment of Patients With Pompe Disease Who Have Participated in a BMN 701 Study
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Pompe Disease
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
First posted: 2011-09-19
Enrollment: 21
Sex: ALL
Minimum age (years): 13
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Number of Participants With a Positive Anti-BMN 701 Antibody
timeFrame: Baseline, Week 144
measure: Number of Participants With a Positive Anti-BMN 701 Antibody Response
timeFrame: Baseline, Week 144
measure: Number of Participants With a Positive Anti-BMN 701 Antibody Response
timeFrame: Baseline, Week 144
Secondary outcomes:
measure: Percent Predicted Maximal Inspiratory Pressure (MIP)
timeFrame: Baseline, Week 144
measure: Percent Predicted Maximum Expiratory Pressure (MEP)
timeFrame: Baseline, Week 144
measure: 6 Minutes Walk Test (Meters)
timeFrame: Baseline, Week 144
measure: Maximum Voluntary Ventilation (MVV)
timeFrame: Baseline, Week 144
measure: Percent Predicted Upright Forced Vital Capacity (FVC)
timeFrame: Baseline, Week 144
measure: Change From Baseline in Urine Tetrasaccharide Concentration at Week 144
timeFrame: Baseline, Week 144
measure: Plasma IGF-I Concentration
timeFrame: Baseline, Week 144
measure: Plasma IGF-II Concentration
timeFrame: Baseline, Week 144
measure: Insulin-like Growth Factor Binding Protein 3 (IGFBP3)
timeFrame: Baseline, Week 144
Other outcomes:
Locations:
city: San Diego
country: United States
facility: Univ of California San Diego School of Medicine
state: California
city: Gainesville
country: United States
facility: University of Florida College of Medicine
state: Florida
city: Tampa
country: United States
facility: Tampa General Hospital
state: Florida
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Herston
country: Australia
facility: Royal Brisbane and Women's Hospital
state: Queensland
city: North Adelaide
country: Australia
facility: Royal Adelaide Hospital
state: South Australia
city: Paris
country: France
facility: Hôpital Pitié-Salpêtrière
city: Mainz
country: Germany
facility: Villa Metabolica, ZKJM MC University Mainz
city: Auckland
country: New Zealand
facility: Auckland City and Starship Children's Hospital
city: Birmingham
country: United Kingdom
facility: University Hospitals Birmingham NHS Foundation Trust
city: London
country: United Kingdom
facility: Royal Free Hospital
city: Salford
country: United Kingdom
facility: Salford Royal NHS Foundation Trust
Study officials:
affiliation: BioMarin Pharmaceutical
name: Medical Monitor
role: STUDY_DIRECTOR
References: