Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01435772




Registration number
NCT01435772
Ethics application status
Date submitted
8/09/2011
Date registered
19/09/2011
Date last updated
22/05/2018

Titles & IDs
Public title
Extension Study for Patients Who Have Participated in a BMN 701 Study
Scientific title
A Long-Term Study for Extended BMN 701 Treatment of Patients With Pompe Disease Who Have Participated in a BMN 701 Study
Secondary ID [1] 0 0
2011-001805-28
Secondary ID [2] 0 0
POM-002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pompe Disease 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - BMN 701

Experimental: BMN 701 20mg/kg - BMN 701 20mg/kg IV every other week

Experimental: BMN 701 10mg/kg - BMN 701 10mg/kg IV every other week

Experimental: BMN 701 5mg/kg - BMN 701 5mg/kg IV every other week


Treatment: Other: BMN 701
GILT-tagged recombinant human GAA
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With a Positive Anti-BMN 701 Antibody
Timepoint [1] 0 0
Baseline, Week 144
Primary outcome [2] 0 0
Number of Participants With a Positive Anti-BMN 701 Antibody Response
Timepoint [2] 0 0
Baseline, Week 144
Primary outcome [3] 0 0
Number of Participants With a Positive Anti-BMN 701 Antibody Response
Timepoint [3] 0 0
Baseline, Week 144
Secondary outcome [1] 0 0
Percent Predicted Maximal Inspiratory Pressure (MIP)
Timepoint [1] 0 0
Baseline, Week 144
Secondary outcome [2] 0 0
Percent Predicted Maximum Expiratory Pressure (MEP)
Timepoint [2] 0 0
Baseline, Week 144
Secondary outcome [3] 0 0
6 Minutes Walk Test (Meters)
Timepoint [3] 0 0
Baseline, Week 144
Secondary outcome [4] 0 0
Maximum Voluntary Ventilation (MVV)
Timepoint [4] 0 0
Baseline, Week 144
Secondary outcome [5] 0 0
Percent Predicted Upright Forced Vital Capacity (FVC)
Timepoint [5] 0 0
Baseline, Week 144
Secondary outcome [6] 0 0
Change From Baseline in Urine Tetrasaccharide Concentration at Week 144
Timepoint [6] 0 0
Baseline, Week 144
Secondary outcome [7] 0 0
Plasma IGF-I Concentration
Timepoint [7] 0 0
Baseline, Week 144
Secondary outcome [8] 0 0
Plasma IGF-II Concentration
Timepoint [8] 0 0
Baseline, Week 144
Secondary outcome [9] 0 0
Insulin-like Growth Factor Binding Protein 3 (IGFBP3)
Timepoint [9] 0 0
Baseline, Week 144

Eligibility
Key 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.
Minimum age
13 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
15/08/2011
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
9/09/2016
Sample size
Target
Accrual to date
Final
21
Recruitment in Australia
Recruitment state(s)
QLD,SA
Recruitment hospital [1] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment hospital [2] 0 0
Royal Adelaide Hospital - North Adelaide
Recruitment postcode(s) [1] 0 0
4029 - Herston
Recruitment postcode(s) [2] 0 0
5006 - North Adelaide
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Kansas
Country [4] 0 0
France
State/province [4] 0 0
Paris
Country [5] 0 0
Germany
State/province [5] 0 0
Mainz
Country [6] 0 0
New Zealand
State/province [6] 0 0
Auckland
Country [7] 0 0
United Kingdom
State/province [7] 0 0
Birmingham
Country [8] 0 0
United Kingdom
State/province [8] 0 0
London
Country [9] 0 0
United Kingdom
State/province [9] 0 0
Salford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
BioMarin Pharmaceutical
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Monitor
Address 0 0
BioMarin Pharmaceutical
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT01435772