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

Primary Purpose

Pompe Disease

Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
BMN 701
Sponsored by
BioMarin Pharmaceutical
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pompe Disease

Eligibility Criteria

13 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Univ of California San Diego School of Medicine
  • University of Florida College of Medicine
  • Tampa General Hospital
  • University of Kansas Medical Center
  • Royal Brisbane and Women's Hospital
  • Royal Adelaide Hospital
  • Hôpital Pitié-Salpêtrière
  • Villa Metabolica, ZKJM MC University Mainz
  • Auckland City and Starship Children's Hospital
  • University Hospitals Birmingham NHS Foundation Trust
  • Royal Free Hospital
  • Salford Royal NHS Foundation Trust

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

BMN 701 20mg/kg

BMN 701 10mg/kg

BMN 701 5mg/kg

Arm Description

BMN 701 20mg/kg IV every other week

BMN 701 10mg/kg IV every other week

BMN 701 5mg/kg IV every other week

Outcomes

Primary Outcome Measures

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.
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
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

Secondary Outcome Measures

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

Full Information

First Posted
September 8, 2011
Last Updated
April 23, 2018
Sponsor
BioMarin Pharmaceutical
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1. Study Identification

Unique Protocol Identification Number
NCT01435772
Brief Title
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

2. Study Status

Record Verification Date
April 2018
Overall Recruitment Status
Terminated
Why Stopped
The study was terminated because BioMarin decided to end the overall development program based on competing corporate priorities.
Study Start Date
August 15, 2011 (Actual)
Primary Completion Date
September 9, 2016 (Actual)
Study Completion Date
September 9, 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
BioMarin Pharmaceutical

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pompe Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
21 (Actual)

8. Arms, Groups, and Interventions

Arm Title
BMN 701 20mg/kg
Arm Type
Experimental
Arm Description
BMN 701 20mg/kg IV every other week
Arm Title
BMN 701 10mg/kg
Arm Type
Experimental
Arm Description
BMN 701 10mg/kg IV every other week
Arm Title
BMN 701 5mg/kg
Arm Type
Experimental
Arm Description
BMN 701 5mg/kg IV every other week
Intervention Type
Biological
Intervention Name(s)
BMN 701
Intervention Description
GILT-tagged recombinant human GAA
Primary Outcome Measure Information:
Title
Number of Participants With a Positive Anti-BMN 701 Antibody
Description
Status of Anti-BMN 701 antibody is corresponding to the test results of blood samples.
Time Frame
Baseline, Week 144
Title
Number of Participants With a Positive Anti-BMN 701 Antibody Response
Description
Status of Anti-IGF-I antibody is corresponding to the test results of blood samples
Time Frame
Baseline, Week 144
Title
Number of Participants With a Positive Anti-BMN 701 Antibody Response
Description
Status of Anti-IGF-II antibody is corresponding to the test results of blood samples
Time Frame
Baseline, Week 144
Secondary Outcome Measure Information:
Title
Percent Predicted Maximal Inspiratory Pressure (MIP)
Description
Pulmonary Function Test: Percent Predicted Maximal Inspiratory Pressure
Time Frame
Baseline, Week 144
Title
Percent Predicted Maximum Expiratory Pressure (MEP)
Description
Pulmonary Function Test: Percent Predicted Maximum Expiratory Pressure
Time Frame
Baseline, Week 144
Title
6 Minutes Walk Test (Meters)
Description
Distance walked within 6 minutes
Time Frame
Baseline, Week 144
Title
Maximum Voluntary Ventilation (MVV)
Description
Pulmonary function test: Maximum Voluntary Ventilation (MVV)
Time Frame
Baseline, Week 144
Title
Percent Predicted Upright Forced Vital Capacity (FVC)
Description
Pulmonary function test: Percent Predicted Upright Forced Vital Capacity
Time Frame
Baseline, Week 144
Title
Change From Baseline in Urine Tetrasaccharide Concentration at Week 144
Description
Change from Baseline in Urine Tetrasaccharide Concentration at Week 144
Time Frame
Baseline, Week 144
Title
Plasma IGF-I Concentration
Description
Plasma IGF-I concentration from lab
Time Frame
Baseline, Week 144
Title
Plasma IGF-II Concentration
Description
Plasma IGF-II concentration from lab
Time Frame
Baseline, Week 144
Title
Insulin-like Growth Factor Binding Protein 3 (IGFBP3)
Description
insulin-like growth factor binding protein 3 from lab
Time Frame
Baseline, Week 144

10. Eligibility

Sex
All
Minimum Age & Unit of Time
13 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Monitor
Organizational Affiliation
BioMarin Pharmaceutical
Official's Role
Study Director
Facility Information:
Facility Name
Univ of California San Diego School of Medicine
City
San Diego
State/Province
California
ZIP/Postal Code
92103
Country
United States
Facility Name
University of Florida College of Medicine
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
Tampa General Hospital
City
Tampa
State/Province
Florida
ZIP/Postal Code
33606
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Royal Brisbane and Women's Hospital
City
Herston
State/Province
Queensland
ZIP/Postal Code
4029
Country
Australia
Facility Name
Royal Adelaide Hospital
City
North Adelaide
State/Province
South Australia
ZIP/Postal Code
5006
Country
Australia
Facility Name
Hôpital Pitié-Salpêtrière
City
Paris
ZIP/Postal Code
75013
Country
France
Facility Name
Villa Metabolica, ZKJM MC University Mainz
City
Mainz
ZIP/Postal Code
55131
Country
Germany
Facility Name
Auckland City and Starship Children's Hospital
City
Auckland
ZIP/Postal Code
1142
Country
New Zealand
Facility Name
University Hospitals Birmingham NHS Foundation Trust
City
Birmingham
ZIP/Postal Code
B15 2TH
Country
United Kingdom
Facility Name
Royal Free Hospital
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom
Facility Name
Salford Royal NHS Foundation Trust
City
Salford
ZIP/Postal Code
M5 5AP
Country
United Kingdom

12. IPD Sharing Statement

Learn more about this trial

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

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