Late-onset Pompe DiseaseNCT02191917

A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)

An observational study of Late-onset Pompe Disease, run by BioMarin Pharmaceutical at 2 sites in 2 countries.

Terminated

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

Trial summary

Study 701-201 is a study in patients with late-onset Pompe disease (LOPD). The study will test respiratory muscle strength initially and again after 24 weeks in subjects treated or not treated with BMN 701 . Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Late-onset Pompe Disease
What is being tested?: Respiratory muscle strength measurements by different techniques.
Type of treatment: Surgery or procedure
How many people: 8 participants
Where: 2 sitesin 2 countries
Study dates: 21 Oct 2014to 22 Jun 2016
Phase: Observational
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 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Study population: Fifteen subjects were planned for enrollment; there were no screening failures; 8 subjects were enrolled. Two subjects were enrolled from 701-301 and the remaining 6 subjects enrolled directly into 701-201 (no parent study)

Who may be able to join

Willing and able to provide written informed consent, after the nature of the study has been explained, and prior to any study-related procedures
Documented diagnosis with late-onset Pompe disease - At least 18 years of age at study entry
Willing and able to comply with all study procedures

Who cannot join

Requires ventilatory support while awake and in the upright position
Concurrent disease, medical condition, or extenuating circumstance that, in the opinion of the investigator, might compromise patient well-being, study completion, or data collection
Allergy to tools or procedures used for respiratory muscle testing

Trial locations and contacts

2 sites in 2 countries.

Country

City, postcode or hospital

2 sites in 2 countries

United States1 site

University of Florida
Gainesville, Florida, 32610

Other trials in United States

United Kingdom1 site

Royal Brompton Hospital
London, SW36NP

Other trials in United Kingdom

About this study

Official title: A Study of Respiratory Muscle Strength, Including Effort-Independent Measures, in Subjects With Late-Onset Pompe Disease

Conditions: Late-onset Pompe Disease
Being tested: Respiratory muscle strength measurements by different techniques.

What participants receive

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

Respiratory muscle strength measurements by different techniques.Procedure or surgery

Study groups in NCT02191917
Study group	Role	What they receive
Measurement of Respiratory Muscle Strength		Not described Receives: Procedure: Respiratory muscle strength measurements by different techniques.

What is being measured

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

Maximal Inspiratory Pressure (MIP)
Pulmonary measure of change in maximal inspiratory pressure (MIP)
Measured: Baseline, Week 28

Secondary outcomes (1)

Maximal Expiratory Pressure (MEP)
Pulmonary measure of change in maximal expiratory pressure (MEP)
Measured: Baseline, Week 28

Timeline

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

21 Oct 201422 Jun 2016

First posted16 Jul 2014 (estimated)
Study start21 Oct 2014
Primary completion22 Jun 2016
Study completion22 Jun 2016

Why it stopped: Study was terminated early due to a company decision not based on safety concerns

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 Late-onset 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: Observational
Observational model: Cohort
Time perspective: Prospective
Enrolment: 8 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02191917
Sponsor's protocol ID: 701-201
Other IDs: 2014-002158-38, 2014
Status last verified: Apr 2022
First submitted: 15 Jul 2014
Last updated: 9 Jan 2023
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

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?

See locations

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A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)
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All Late-onset Pompe Disease trials

Study at a glance

From the registry record, last updated by the sponsor on 9 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Study of Respiratory Muscle Strength, Including Effort-Independent Measures, in Subjects With Late-Onset Pompe Disease
ClinicalTrials.gov ID: NCT02191917
Acronym / sponsor ID: 701-201
Conditions: Late-onset Pompe Disease
Study type: Observational
Recruitment status: TerminatedReason stopped: Study was terminated early due to a company decision not based on safety concerns
Enrolment: 8 participants (actual)
Lead sponsor: BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
Study start: 21 Oct 2014 (actual)
Primary completion: 22 Jun 2016 (actual)
Study completion: 22 Jun 2016 (actual)
First posted: 16 Jul 2014 (estimated)
Results: Posted 9 Jan 2023

Clinical summary

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

Study design

Observational model: Cohort
Time perspective: Prospective
Number of arms: 1

Arm	Type	Receives
Measurement of Respiratory Muscle Strength	–	Procedure: Respiratory muscle strength measurements by different techniques.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult
Study population: Fifteen subjects were planned for enrollment; there were no screening failures; 8 subjects were enrolled. Two subjects were enrolled from 701-301 and the remaining 6 subjects enrolled directly into 701-201 (no parent study)

Key inclusion criteria (3 listed)

Willing and able to provide written informed consent, after the nature of the study has been explained, and prior to any study-related procedures
Documented diagnosis with late-onset Pompe disease - At least 18 years of age at study entry
Willing and able to comply with all study procedures

Key exclusion criteria (3 listed)

Requires ventilatory support while awake and in the upright position
Concurrent disease, medical condition, or extenuating circumstance that, in the opinion of the investigator, might compromise patient well-being, study completion, or data collection
Allergy to tools or procedures used for respiratory muscle testing

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
Respiratory muscle strength measurements by different techniques.	Procedure or surgery	Measurement of Respiratory Muscle Strength

Primary outcome

Maximal Inspiratory Pressure (MIP)
Time frame: Baseline, Week 28
Pulmonary measure of change in maximal inspiratory pressure (MIP)

1 secondary outcome

Maximal Expiratory Pressure (MEP)
Time frame: Baseline, Week 28

Results and safety

Results posted to the registry on 9 Jan 2023, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Overall	8	7

Primary outcome: Maximal Inspiratory Pressure (MIP)

Time frame: Baseline, Week 28. Mean (Standard Deviation). Unit: cm H2O

Measure	Overall n = 8
Baseline	49.2 (21.7)
Last Visit	46.0 (20.93)
Change from Baseline	-5.5 (8.38)

Population analysed: Full Analysis Set (FAS) population includes all enrolled subjects who have at least one study assessment.

1 secondary outcome is reported in the full results.

Adverse events

Time frame: 28 weeks. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Overall	0 of 8 (0%)	1 of 8 (12.5%)	0 of 8

Limitations and caveats (from the sponsor): The study was planned to have 15 participants. However, the study was terminated prematurely. Only 8 participants were enrolled. Seven participants had last assessment at Days 146, 168, 172, 174, 179, 188, 196, translating to one participant at Week 20 and six participants Weeks 24-28.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 9 Jan 2023.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Late-onset Pompe Disease

Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
RecruitingObservational study
Pompe Disease Registry Protocol
Glycogen Storage Disease Type II, Pompe Disease
Not yet recruitingPhase 2
An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)
Pompe Disease
RecruitingPhase 1/2
A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)
Pompe Disease (Late-onset), Pompe Disease Late-Onset
Active, not recruiting
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
Pompe Disease (Late-onset)

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, MD study director

BioMarin Pharmaceutical

2 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

An observational study of Late-onset Pompe Disease, run by BioMarin Pharmaceutical at 2 sites in 2 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 3 inclusion and 3 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 9 Jan 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States, United Kingdom. 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.

Are results available for this study?

Yes. Results are posted in the registry (first posted 9 Jan 2023). 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?

Maximal Inspiratory Pressure (MIP) (time frame: Baseline, Week 28). 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 NCT02191917.

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 9 Jan 2023.