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

Pompe Disease Clinical Trial

Official title:

A Long-Term Study for Extended BMN 701 Treatment of Patients With Pompe Disease Who Have Participated in a BMN 701 Study

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01435772
• Other study ID #: POM-002
• Secondary ID: 2011-001805-28
• Status: Terminated
• Phase: Phase 2
• Start date: August 15, 2011
• Est. completion date: September 9, 2016

Study information

• Verified date: April 2018
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 21
• Est. completion date: September 9, 2016
• Est. primary completion date: September 9, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 13 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Glycogen Storage Disease Type II
• Pompe Disease

Intervention

Biological:
• BMN 701
GILT-tagged recombinant human GAA

Locations

Country	Name	City	State
Australia	Royal Brisbane and Women's Hospital	Herston	Queensland
Australia	Royal Adelaide Hospital	North Adelaide	South Australia
France	Hôpital Pitié-Salpêtrière	Paris
Germany	Villa Metabolica, ZKJM MC University Mainz	Mainz
New Zealand	Auckland City and Starship Children's Hospital	Auckland
United Kingdom	University Hospitals Birmingham NHS Foundation Trust	Birmingham
United Kingdom	Royal Free Hospital	London
United Kingdom	Salford Royal NHS Foundation Trust	Salford
United States	University of Florida College of Medicine	Gainesville	Florida
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	Univ of California San Diego School of Medicine	San Diego	California
United States	Tampa General Hospital	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Countries where clinical trial is conducted

United States,  Australia,  France,  Germany,  New Zealand,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With a Positive Anti-BMN 701 Antibody	Status of Anti-BMN 701 antibody is corresponding to the test results of blood samples.	Baseline, Week 144
Primary	Number of Participants With a Positive Anti-BMN 701 Antibody Response	Status of Anti-IGF-I antibody is corresponding to the test results of blood samples	Baseline, Week 144
Primary	Number of Participants With a Positive Anti-BMN 701 Antibody Response	Status of Anti-IGF-II antibody is corresponding to the test results of blood samples	Baseline, Week 144
Secondary	Percent Predicted Maximal Inspiratory Pressure (MIP)	Pulmonary Function Test: Percent Predicted Maximal Inspiratory Pressure	Baseline, Week 144
Secondary	Percent Predicted Maximum Expiratory Pressure (MEP)	Pulmonary Function Test: Percent Predicted Maximum Expiratory Pressure	Baseline, Week 144
Secondary	6 Minutes Walk Test (Meters)	Distance walked within 6 minutes	Baseline, Week 144
Secondary	Maximum Voluntary Ventilation (MVV)	Pulmonary function test: Maximum Voluntary Ventilation (MVV)	Baseline, Week 144
Secondary	Percent Predicted Upright Forced Vital Capacity (FVC)	Pulmonary function test: Percent Predicted Upright Forced Vital Capacity	Baseline, Week 144
Secondary	Change From Baseline in Urine Tetrasaccharide Concentration at Week 144	Change from Baseline in Urine Tetrasaccharide Concentration at Week 144	Baseline, Week 144
Secondary	Plasma IGF-I Concentration	Plasma IGF-I concentration from lab	Baseline, Week 144
Secondary	Plasma IGF-II Concentration	Plasma IGF-II concentration from lab	Baseline, Week 144
Secondary	Insulin-like Growth Factor Binding Protein 3 (IGFBP3)	insulin-like growth factor binding protein 3 from lab	Baseline, Week 144