Late-onset Pompe Patients Untreated or Treated With rhGAANCT02221362
A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
An observational study of Late-onset Pompe Patients Untreated or Treated With rhGAA, run by BioMarin Pharmaceutical at 24 sites in 13 countries.
Trial summary
Study 701-901, a multicenter, multinational, longitudinal, non-interventional observational study in subjects, at least 18 years old, diagnosed with late-onset Pompe disease prospectively collects data to understand clinical progression in terms of respiratory function, symptomology, genotype, biochemistry, endurance… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Late-onset Pompe Patients Untreated or Treated With rhGAA
- How many people
- 30 participants
- Where
- 24 sitesin 13 countries
- Study dates
- Dec 2014to Sep 2016
- Phase
- Observational
- Are there results?
- None posted in the registry
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: Late-onset Pompe Disease patients untreated or treated with rhGAA
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 procedure
- Diagnosed with late-onset Pompe disease based on current or previous genomic testing and/or endogenous GAA activity
- At least 18 years of age at study entry
- Willing and able to comply with all study procedures
Who cannot join
- Requires non-invasive 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 study completion or data collection
- Unable to perform baseline efficacy assessments
Trial locations and contacts
24 sites in 13 countries.
24 sites in 13 countries
Greece4 sites
Study site
Alexandroupoli
Study site
Athens
Study site
Larissa
Study site
Thessaloniki
Australia3 sites
Women's and Children's Hospital
Adelaide, South Australia, 5006
Monash Medical Centre
Clayton, Victoria, 3168
Westmead Hospital, Dept. of Genetic Medicine
Westmead, New South Wales, 2145
Brazil3 sites
Study site
Porto Alegre
Study site
Recife
Study site
Ribeirão Preto
Poland3 sites
Copernicus, St. Adalbert Hospital
Gdansk, 80-462
Diamond Clinic
Krakow, 31-011
Insitute of Psychiatry and Neurology
Warsaw, 02-957
Spain3 sites
Hospital de la Santa Creu I Sant Pau
Barcelona, 08041
Hospital General Universitario Gregorio Maranon
Madrid, 28007
Hospital Universitari i Politecnic La Fe
Valencia, 46026
Romania1 site
Spitalul Clinic Judetean de Urgenta "Sf. Apostol Andrei"
Constanța, 900951
About this study
Study 701-901, a multicenter, multinational, longitudinal, non-interventional observational study in subjects, at least 18 years old, diagnosed with late-onset Pompe disease prospectively collects data to understand clinical progression in terms of respiratory function, symptomology, genotype, biochemistry, endurance and selected subject-reported measures for 24 weeks followed by a 240 week additional observation period for up to 100 subjects.
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Observational | No interventions |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the degree of change in respiratory and endurance endpoints over time in patients with Pompe disease
Measured: 264 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2014 (estimated)
- Study startDec 2014
- Primary completionSep 2016
- Study completionSep 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Late-onset Pompe Patients Untreated or Treated With rhGAAStudies 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
- Case-control
- Time perspective
- Prospective
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, MD, Study director, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02221362
- Sponsor's protocol ID
- 701-901
- Status last verified
- Sep 2015
- First submitted
- 6 Aug 2014
- Last updated
- 31 Oct 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Oct 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
- ClinicalTrials.gov ID
- NCT02221362
- Acronym / sponsor ID
- 701-901
- Study type
- Observational
- Recruitment status
- Terminated
- Enrolment
- 30 participants (actual)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- Dec 2014
- Primary completion
- Sep 2016 (actual)
- Study completion
- Sep 2016 (actual)
- First posted
- 20 Aug 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Study 701-901, a multicenter, multinational, longitudinal, non-interventional observational study in subjects, at least 18 years old, diagnosed with late-onset Pompe disease prospectively collects data to understand clinical progression in terms of respiratory function, symptomology, genotype, biochemistry, endurance and selected subject-reported measures for 24 weeks followed by a 240 week additional observation period for up to 100 subjects.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Observational | – | –No interventions |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Late-onset Pompe Disease patients untreated or treated with rhGAA
Key inclusion criteria (4 listed)
- Willing and able to provide written informed consent after the nature of the study has been explained and prior to any study-related procedure
- Diagnosed with late-onset Pompe disease based on current or previous genomic testing and/or endogenous GAA activity
- At least 18 years of age at study entry
- Willing and able to comply with all study procedures
Key exclusion criteria (3 listed)
- Requires non-invasive 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 study completion or data collection
- Unable to perform baseline efficacy assessments
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
To evaluate the degree of change in respiratory and endurance endpoints over time in patients with Pompe disease
Time frame: 264 Weeks
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Oct 2016.
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
24 sites in 13 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Late-onset Pompe Patients Untreated or Treated With rhGAA, run by BioMarin Pharmaceutical at 24 sites in 13 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 4 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 31 Oct 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 24 sites in Greece, Australia, Brazil, Poland, Spain and 8 more countries. 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?
The study was stopped early. Results are not available in the source record.
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?
To evaluate the degree of change in respiratory and endurance endpoints over time in patients with Pompe disease (time frame: 264 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 31 Oct 2016.