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

NCT01435772 | Terminated Phase 2 Results DMC

• 2 other identifiers: POM-0022011-001805-28
• Study Type: interventional
• Target: 21
• Locations: 6 countries
• Sites: 12

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.

Conditions

Pompe Disease

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (9)

• Percent Predicted Maximal Inspiratory Pressure (MIP)

  Baseline, Week 144

• Percent Predicted Maximum Expiratory Pressure (MEP)

  Baseline, Week 144

• 6 Minutes Walk Test (Meters)

  Baseline, Week 144

• Maximum Voluntary Ventilation (MVV)

  Baseline, Week 144

• Percent Predicted Upright Forced Vital Capacity (FVC)

  Baseline, Week 144

Study Arms (3)

BMN 701 20mg/kg

EXPERIMENTAL

BMN 701 20mg/kg IV every other week

Biological: BMN 701

BMN 701 10mg/kg

EXPERIMENTAL

BMN 701 10mg/kg IV every other week

Biological: BMN 701

BMN 701 5mg/kg

EXPERIMENTAL

BMN 701 5mg/kg IV every other week

Biological: BMN 701

Interventions

BMN 701 BIOLOGICAL

GILT-tagged recombinant human GAA

BMN 701 10mg/kg; BMN 701 20mg/kg; BMN 701 5mg/kg

Eligibility Criteria

• Age: 13 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (12)

Univ of California San Diego School of Medicine

San Diego, California, 92103, United States

Location

University of Florida College of Medicine

Gainesville, Florida, 32610, United States

Location

Tampa General Hospital

Tampa, Florida, 33606, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Royal Brisbane and Women's Hospital

Herston, Queensland, 4029, Australia

Location

Royal Adelaide Hospital

North Adelaide, South Australia, 5006, Australia

Location

Hôpital Pitié-Salpêtrière

Paris, 75013, France

Location

Villa Metabolica, ZKJM MC University Mainz

Mainz, 55131, Germany

Location

Auckland City and Starship Children's Hospital

Auckland, 1142, New Zealand

Location

University Hospitals Birmingham NHS Foundation Trust

Birmingham, B15 2TH, United Kingdom

Location

Royal Free Hospital

London, NW3 2QG, United Kingdom

Location

Salford Royal NHS Foundation Trust

Salford, M5 5AP, United Kingdom

Location

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Debra Lounsbury, Principal Scientist, Clinical Sciences
• Organization: BioMarin Pharmaceutical Inc.

DLounsbury@bmrn.com

415-506-6348

Study Officials

• Medical Monitor

  BioMarin Pharmaceutical

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 8, 2011
• First Posted: September 19, 2011
• Study Start: August 15, 2011
• Primary Completion: September 9, 2016
• Study Completion: September 9, 2016
• Last Updated: May 22, 2018
• Results First Posted: May 22, 2018

Record last verified: 2018-04

Locations

GB (3); US (4); DE (1); NZ (1); FR (1); AU (2)