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

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

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.

Study Overview

• Status: Terminated
• Conditions: Pompe Disease
• Intervention / Treatment: Biological: BMN 701

• Study Type: Interventional
• Enrollment (Actual): 21
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • Queensland
    • Herston, Queensland, Australia, 4029
      • Royal Brisbane and Women's Hospital
  • South Australia
    • North Adelaide, South Australia, Australia, 5006
      • Royal Adelaide Hospital
• France
  • Paris, France, 75013
    • Hopital Pitie-Salpetriere
• Germany
  • Mainz, Germany, 55131
    • Villa Metabolica, ZKJM MC University Mainz
• New Zealand
  • Auckland, New Zealand, 1142
    • Auckland City and Starship Children's Hospital
• United Kingdom
  • Birmingham, United Kingdom, B15 2TH
    • University Hospitals Birmingham NHS Foundation Trust
  • London, United Kingdom, NW3 2QG
    • Royal Free Hospital
  • Salford, United Kingdom, M5 5AP
    • Salford Royal NHS Foundation Trust
• United States
  • California
    • San Diego, California, United States, 92103
      • Univ of California San Diego School of Medicine
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida College of Medicine
    • Tampa, Florida, United States, 33606
      • Tampa General Hospital
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • University of Kansas Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 13 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: BMN 701 20mg/kg; BMN 701 20mg/kg IV every other week	Biological: BMN 701; GILT-tagged recombinant human GAA
Experimental: BMN 701 10mg/kg; BMN 701 10mg/kg IV every other week	Biological: BMN 701; GILT-tagged recombinant human GAA
Experimental: BMN 701 5mg/kg; BMN 701 5mg/kg IV every other week	Biological: BMN 701; GILT-tagged recombinant human GAA

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percent Predicted Maximal Inspiratory Pressure (MIP)	Pulmonary Function Test: Percent Predicted Maximal Inspiratory Pressure	Baseline, Week 144
Percent Predicted Maximum Expiratory Pressure (MEP)	Pulmonary Function Test: Percent Predicted Maximum Expiratory Pressure	Baseline, Week 144
6 Minutes Walk Test (Meters)	Distance walked within 6 minutes	Baseline, Week 144
Maximum Voluntary Ventilation (MVV)	Pulmonary function test: Maximum Voluntary Ventilation (MVV)	Baseline, Week 144
Percent Predicted Upright Forced Vital Capacity (FVC)	Pulmonary function test: Percent Predicted Upright Forced Vital Capacity	Baseline, Week 144
Change From Baseline in Urine Tetrasaccharide Concentration at Week 144	Change from Baseline in Urine Tetrasaccharide Concentration at Week 144	Baseline, Week 144
Plasma IGF-I Concentration	Plasma IGF-I concentration from lab	Baseline, Week 144
Plasma IGF-II Concentration	Plasma IGF-II concentration from lab	Baseline, Week 144
Insulin-like Growth Factor Binding Protein 3 (IGFBP3)	insulin-like growth factor binding protein 3 from lab	Baseline, Week 144

Collaborators and Investigators

• Sponsor: BioMarin Pharmaceutical
• Investigators: Study Director: Medical Monitor, BioMarin Pharmaceutical

Study record dates

Study Major Dates

• Study Start (Actual): August 15, 2011
• Primary Completion (Actual): September 9, 2016
• Study Completion (Actual): September 9, 2016

Study Registration Dates

• First Submitted: September 8, 2011
• First Submitted That Met QC Criteria: September 15, 2011
• First Posted (Estimate): September 19, 2011

Study Record Updates

• Last Update Posted (Actual): May 22, 2018
• Last Update Submitted That Met QC Criteria: April 23, 2018
• Last Verified: April 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Lysosomal Storage Diseases, Nervous System; Glycogen Storage Disease; Glycogen Storage Disease Type II
• Other Study ID Numbers: POM-002; 2011-001805-28 (EudraCT Number)