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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT01230801




Registration number
NCT01230801
Ethics application status
Date submitted
27/10/2010
Date registered
29/10/2010
Date last updated
11/06/2018

Titles & IDs
Public title
Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
Scientific title
A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe Disease
Secondary ID [1] 0 0
2010-023561-22
Secondary ID [2] 0 0
POM-001
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
Other interventions - BMN 701

Experimental: BMN 701 - IV infusion


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

Outcomes
Primary outcome [1] 0 0
Number of Participants With Adverse Events
Timepoint [1] 0 0
24 weeks
Secondary outcome [1] 0 0
Change From Baseline in Six Minutes Walk Test
Timepoint [1] 0 0
Baseline up to 24 weeks

Eligibility
Key inclusion criteria
Inclusion criteria:

- Patient has been diagnosed with Pompe Disease prior to or during the screening period
based on 2 GAA gene mutations and either: endogenous GAA activity <75% of the lower
limit of the normal adult range reported by the testing laboratory, as assessed in
cultured skin fibroblasts -or- endogenous GAA activity <75% of the lower limit of the
normal adult range reported by the testing laboratory, as assessed by dried blood spot
or whole blood assay;

- Patient is male or female and 13 years of age or older at the time of enrollment in
the study;

- Sexually active patients must be willing to use an acceptable method of contraception
while participating in the study and for at least 4 months following the last dose of
BMN 701;

- If patient is female and not considered to be of childbearing potential, she is at
least 2 years post-menopausal or had tubal ligation at least 1 year prior to
screening, or who have had total hysterectomy;

- If patient is female and of childbearing potential, she has negative urine pregnancy
tests during the Screening Period and at the Baseline visit and be willing to have
additional pregnancy tests during the study;

- Patient has =30% predicted upright FVC and either <80% predicted upright FVC, or >10%
reduction in supine FVC compared to upright FVC during the Screening Period;

- Patient is naïve to Enzyme Replacement Therapy (ERT) with rhGAA;

- Patient must be able to ambulate at least 40 meters (131.2 feet) on the 6MWT conducted
at the Screening visit (use of assistive devices such as walker, cane, or crutches, is
permitted); and

- If subject was female, she was not lactating
Minimum age
13 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion criteria:

- Patient has a history of diabetes or other disease known to cause hypoglycemia and is
currently receiving, or might anticipate receiving, hypoglycemic agents during the
course of the study;

- Patient has been on any immunosuppressive medication other than glucocorticosteroids
within 1 year prior to enrollment into this study;

- Patient requires invasive ventilatory assistance at the time of enrollment into the
study;

- Patient has received any investigational medication within 30 days prior to the first
dose of study drug or is scheduled to receive any investigational drug other than BMN
701 during the course of the study;

- Patient has previously been admitted to the study;

- Patient is breastfeeding at screening or planning to become pregnant (self or partner)
at any time during the study;

- Patient has 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;

- Patient has any condition that, in the view of the Investigator, places the patient at
high risk of poor treatment compliance or of not completing the study.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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
Single group
Other design features
Phase
Phase 1/Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
17/01/2011
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
6/03/2013
Sample size
Target
Accrual to date
Final
22
Recruitment in Australia
Recruitment state(s)
Adelaide, S
Recruitment hospital [1] 0 0
Royal Adelaide Hospital, SA Pathology - Adelaide
Recruitment postcode(s) [1] 0 0
5006 - 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
Nice
Country [5] 0 0
France
State/province [5] 0 0
Paris Cedex 13
Country [6] 0 0
Germany
State/province [6] 0 0
Rheinland-pfalz
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
A Phase 1/2, open-label, multicenter, multiple dose escalation study of BMN 701 administered
by intravenous infusion every 2 weeks over a 24-week treatment period to patients with
late-onset Pompe disease.
Trial website
https://clinicaltrials.gov/ct2/show/NCT01230801
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT01230801