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

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ClinicalTrials.gov Identifier: NCT01435772
Recruitment Status : Terminated (The study was terminated because BioMarin decided to end the overall development program based on competing corporate priorities.)
First Posted : September 19, 2011
Results First Posted : May 22, 2018
Last Update Posted : May 22, 2018
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Tracking Information
First Submitted Date  ICMJE September 8, 2011
First Posted Date  ICMJE September 19, 2011
Results First Submitted Date  ICMJE September 12, 2017
Results First Posted Date  ICMJE May 22, 2018
Last Update Posted Date May 22, 2018
Actual Study Start Date  ICMJE August 15, 2011
Actual Primary Completion Date September 9, 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 23, 2018)
  • Number of Participants With a Positive Anti-BMN 701 Antibody [ Time Frame: Baseline, Week 144 ]
    Status of Anti-BMN 701 antibody is corresponding to the test results of blood samples.
  • Number of Participants With a Positive Anti-BMN 701 Antibody Response [ Time Frame: Baseline, Week 144 ]
    Status of Anti-IGF-I antibody is corresponding to the test results of blood samples
  • Number of Participants With a Positive Anti-BMN 701 Antibody Response [ Time Frame: Baseline, Week 144 ]
    Status of Anti-IGF-II antibody is corresponding to the test results of blood samples
Original Primary Outcome Measures  ICMJE
 (submitted: September 15, 2011)
  • Number of Treatment-Emergent Adverse Events [ Time Frame: 24 weeks ]
  • Anti-BMN 701 antibody titer [ Time Frame: 24 weeks ]
  • Anti-Insulin-like-growth-factor antibody titer [ Time Frame: 24 weeks ]
Change History Complete list of historical versions of study NCT01435772 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 23, 2018)
  • Percent Predicted Maximal Inspiratory Pressure (MIP) [ Time Frame: Baseline, Week 144 ]
    Pulmonary Function Test: Percent Predicted Maximal Inspiratory Pressure
  • Percent Predicted Maximum Expiratory Pressure (MEP) [ Time Frame: Baseline, Week 144 ]
    Pulmonary Function Test: Percent Predicted Maximum Expiratory Pressure
  • 6 Minutes Walk Test (Meters) [ Time Frame: Baseline, Week 144 ]
    Distance walked within 6 minutes
  • Maximum Voluntary Ventilation (MVV) [ Time Frame: Baseline, Week 144 ]
    Pulmonary function test: Maximum Voluntary Ventilation (MVV)
  • Percent Predicted Upright Forced Vital Capacity (FVC) [ Time Frame: Baseline, Week 144 ]
    Pulmonary function test: Percent Predicted Upright Forced Vital Capacity
  • Change From Baseline in Urine Tetrasaccharide Concentration at Week 144 [ Time Frame: Baseline, Week 144 ]
    Change from Baseline in Urine Tetrasaccharide Concentration at Week 144
  • Plasma IGF-I Concentration [ Time Frame: Baseline, Week 144 ]
    Plasma IGF-I concentration from lab
  • Plasma IGF-II Concentration [ Time Frame: Baseline, Week 144 ]
    Plasma IGF-II concentration from lab
  • Insulin-like Growth Factor Binding Protein 3 (IGFBP3) [ Time Frame: Baseline, Week 144 ]
    insulin-like growth factor binding protein 3 from lab
Original Secondary Outcome Measures  ICMJE
 (submitted: September 15, 2011)
Mean distance walked as measured by the Six-minute Walk Test (6MWT) [ Time Frame: 24 weeks ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Extension Study for Patients Who Have Participated in a BMN 701 Study
Official Title  ICMJE A Long-Term Study for Extended BMN 701 Treatment of Patients With Pompe Disease Who Have Participated in a BMN 701 Study
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Pompe Disease
Intervention  ICMJE Biological: BMN 701
GILT-tagged recombinant human GAA
Study Arms  ICMJE
  • Experimental: BMN 701 20mg/kg
    BMN 701 20mg/kg IV every other week
    Intervention: Biological: BMN 701
  • Experimental: BMN 701 10mg/kg
    BMN 701 10mg/kg IV every other week
    Intervention: Biological: BMN 701
  • Experimental: BMN 701 5mg/kg
    BMN 701 5mg/kg IV every other week
    Intervention: Biological: BMN 701
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: April 23, 2018)
21
Original Estimated Enrollment  ICMJE
 (submitted: September 15, 2011)
45
Actual Study Completion Date  ICMJE September 9, 2016
Actual Primary Completion Date September 9, 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 13 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   France,   Germany,   New Zealand,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01435772
Other Study ID Numbers  ICMJE POM-002
2011-001805-28 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party BioMarin Pharmaceutical
Study Sponsor  ICMJE BioMarin Pharmaceutical
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Monitor BioMarin Pharmaceutical
PRS Account BioMarin Pharmaceutical
Verification Date April 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP