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

April 23, 2018 updated by: BioMarin Pharmaceutical

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

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
21

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

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
        • Hôpital Pitié-Salpêtrière
  • 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

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
Baseline, Week 144
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
Baseline, Week 144
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
Baseline, Week 144

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Percent Predicted Maximal Inspiratory Pressure (MIP)
Time Frame: Baseline, Week 144
Pulmonary Function Test: Percent Predicted Maximal Inspiratory Pressure
Baseline, Week 144
Percent Predicted Maximum Expiratory Pressure (MEP)
Time Frame: Baseline, Week 144
Pulmonary Function Test: Percent Predicted Maximum Expiratory Pressure
Baseline, Week 144
6 Minutes Walk Test (Meters)
Time Frame: Baseline, Week 144
Distance walked within 6 minutes
Baseline, Week 144
Maximum Voluntary Ventilation (MVV)
Time Frame: Baseline, Week 144
Pulmonary function test: Maximum Voluntary Ventilation (MVV)
Baseline, Week 144
Percent Predicted Upright Forced Vital Capacity (FVC)
Time Frame: Baseline, Week 144
Pulmonary function test: Percent Predicted Upright Forced Vital Capacity
Baseline, Week 144
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
Baseline, Week 144
Plasma IGF-I Concentration
Time Frame: Baseline, Week 144
Plasma IGF-I concentration from lab
Baseline, Week 144
Plasma IGF-II Concentration
Time Frame: Baseline, Week 144
Plasma IGF-II concentration from lab
Baseline, Week 144
Insulin-like Growth Factor Binding Protein 3 (IGFBP3)
Time Frame: Baseline, Week 144
insulin-like growth factor binding protein 3 from lab
Baseline, Week 144

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
BioMarin Pharmaceutical

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Medical Monitor, BioMarin Pharmaceutical

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 15, 2011

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 9, 2016

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 9, 2016

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 8, 2011

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 15, 2011

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 19, 2011

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 22, 2018

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 23, 2018

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • POM-002
  • 2011-001805-28 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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