Pompe DiseasePhase 2NCT01435772

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

A Phase 2 study testing BMN 701 in people with Pompe Disease, run by BioMarin Pharmaceutical at 12 sites in 6 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 13 years and older
Conditions the study is for: Pompe Disease
What is being tested?: BMN 701
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 21 participants
Where: 12 sitesin 6 countries
Study dates: 15 Aug 2011to 9 Sep 2016
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 13 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

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.

Who cannot join

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.

Trial locations and contacts

12 sites in 6 countries.

Country

City, postcode or hospital

12 sites in 6 countries

United States4 sites

University of Florida College of Medicine
Gainesville, Florida, 32610
University of Kansas Medical Center
Kansas City, Kansas, 66160
Univ of California San Diego School of Medicine
San Diego, California, 92103
Tampa General Hospital
Tampa, Florida, 33606

Other trials in United States

United Kingdom3 sites

University Hospitals Birmingham NHS Foundation Trust
Birmingham, B15 2TH
Royal Free Hospital
London, NW3 2QG
Salford Royal NHS Foundation Trust
Salford, M5 5AP

Other trials in United Kingdom

Australia2 sites

Royal Brisbane and Women's Hospital
Herston, Queensland, 4029
Royal Adelaide Hospital
North Adelaide, South Australia, 5006

Other trials in Australia

France1 site

Hôpital Pitié-Salpêtrière
Paris, 75013

Other trials in France

Germany1 site

Villa Metabolica, ZKJM MC University Mainz
Mainz, 55131

Other trials in Germany

New Zealand1 site

Auckland City and Starship Children's Hospital
Auckland, 1142

Other trials in New Zealand

About this 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

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.

Conditions: Pompe Disease
Being tested: BMN 701
Medical subject headings: Glycogen Storage Disease Type II

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

BMN 701Biological
GILT-tagged recombinant human GAA

Study groups in NCT01435772
Study group	Role	What they receive
BMN 701 20mg/kg	Experimental	BMN 701 20mg/kg IV every other week Receives: Biological: BMN 701
BMN 701 10mg/kg	Experimental	BMN 701 10mg/kg IV every other week Receives: Biological: BMN 701
BMN 701 5mg/kg	Experimental	BMN 701 5mg/kg IV every other week Receives: Biological: BMN 701

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: Baseline, Week 144
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
Measured: Baseline, Week 144
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
Measured: Baseline, Week 144

Secondary outcomes (9)

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

15 Aug 20119 Sep 2016

First posted19 Sep 2011 (estimated)
Study start15 Aug 2011
Primary completion9 Sep 2016
Study completion9 Sep 2016

Why it stopped: The study was terminated because BioMarin decided to end the overall development program based on competing corporate priorities.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Pompe DiseaseStudies of the same condition, open and under way.
More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 21 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01435772
Sponsor's protocol ID: POM-002
Other IDs: 2011-001805-28
Status last verified: Apr 2018
First submitted: 8 Sep 2011
Last updated: 22 May 2018
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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All Pompe Disease trials

Study at a glance

From the registry record, last updated by the sponsor on 22 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Long-Term Study for Extended BMN 701 Treatment of Patients With Pompe Disease Who Have Participated in a BMN 701 Study
ClinicalTrials.gov ID: NCT01435772
Acronym / sponsor ID: POM-002
Conditions: Pompe Disease
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: The study was terminated because BioMarin decided to end the overall development program based on competing corporate priorities.
Enrolment: 21 participants (actual)
Lead sponsor: BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
Study start: 15 Aug 2011 (actual)
Primary completion: 9 Sep 2016 (actual)
Study completion: 9 Sep 2016 (actual)
First posted: 19 Sep 2011 (estimated)
Results: Posted 22 May 2018

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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 design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 3

Arm	Type	Receives
BMN 701 20mg/kg	Experimental	Biological: BMN 701BMN 701 20mg/kg IV every other week
BMN 701 10mg/kg	Experimental	Biological: BMN 701BMN 701 10mg/kg IV every other week
BMN 701 5mg/kg	Experimental	Biological: BMN 701BMN 701 5mg/kg IV every other week

Who may qualify

Ages: 13 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (7 listed)

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;

Key exclusion criteria (4 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
BMN 701GILT-tagged recombinant human GAA	Biological	BMN 701 10mg/kg, BMN 701 20mg/kg, BMN 701 5mg/kg

Primary outcome

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

9 secondary outcomes

Percent Predicted Maximal Inspiratory Pressure (MIP)
Time frame: Baseline, Week 144
Percent Predicted Maximum Expiratory Pressure (MEP)
Time frame: Baseline, Week 144
6 Minutes Walk Test (Meters)
Time frame: Baseline, Week 144
Maximum Voluntary Ventilation (MVV)
Time frame: Baseline, Week 144
Percent Predicted Upright Forced Vital Capacity (FVC)
Time frame: Baseline, Week 144
Change From Baseline in Urine Tetrasaccharide Concentration at Week 144
Time frame: Baseline, Week 144
Plasma IGF-I Concentration
Time frame: Baseline, Week 144
Plasma IGF-II Concentration
Time frame: Baseline, Week 144
Insulin-like Growth Factor Binding Protein 3 (IGFBP3)
Time frame: Baseline, Week 144

Results and safety

Results posted to the registry on 22 May 2018, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
BMN 701 5 mg/kg	3	0
BMN 701 10 mg/kg	3	0
BMN 701 20 mg/kg	15	0

Primary outcome: Number of Participants With a Positive Anti-BMN 701 Antibody

Time frame: Baseline, Week 144. Count of participants. Unit: Participants

Measure	BMN 701 5 mg/kg n = 3	BMN 701 10 mg/kg n = 3	BMN 701 20 mg/kg n = 15
Baseline	2	1	11
Week 144	2	2	10

Population analysed: Full Analysis Set. Please note the overall number of participants reflects the number of patients in that arm, while the outcome measurement number of participants reflects the number of patients for which data is available and analyzed.

Primary outcome: Number of Participants With a Positive Anti-BMN 701 Antibody Response

Time frame: Baseline, Week 144. Count of participants. Unit: Participants

Measure	BMN 701 5 mg/kg n = 3	BMN 701 10 mg/kg n = 3	BMN 701 20 mg/kg n = 15
Baseline	0	2	0
Week 144	0	1	5

Population analysed: Full Analysis Set.

Primary outcome: Number of Participants With a Positive Anti-BMN 701 Antibody Response

Time frame: Baseline, Week 144. Count of participants. Unit: Participants

Measure	BMN 701 5 mg/kg n = 3	BMN 701 10 mg/kg n = 3	BMN 701 20 mg/kg n = 15
Baseline	1	2	2
Week 144	0	2	7

Population analysed: Full Analysis Set.

10 secondary outcomes are reported in the full results.

Adverse events

Time frame: 284.9 weeks (maximum exposure) +4 weeks follow up. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
BMN 701 5 mg/kg	2 of 3 (66.7%)	3 of 3 (100%)	0 of 3
BMN 701 10 mg/kg	1 of 3 (33.3%)	3 of 3 (100%)	0 of 3
BMN 701 20 mg/kg	7 of 15 (46.7%)	15 of 15 (100%)	0 of 15
Total	10 of 21 (47.6%)	21 of 21 (100%)	0 of 21

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2018.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Medical Monitor study director

BioMarin Pharmaceutical

12 sites in 6 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing BMN 701 in people with Pompe Disease, run by BioMarin Pharmaceutical at 12 sites in 6 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 22 May 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites in United States, United Kingdom, Australia, France, Germany and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 22 May 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With a Positive Anti-BMN 701 Antibody (time frame: Baseline, Week 144). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01435772.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 May 2018.