Alström SyndromePhase 2/3NCT03184584
Open-Label Rollover Study of PBI 4050 in Subjects With Alström Syndrome
A Phase 2/3 study testing PBI-4050 in people with Alström Syndrome, run by Liminal BioSciences Ltd. at 1 site in United Kingdom.
Trial summary
Phase 2, open-label, single-arm, multi-centre study evaluating the long term safety and tolerability of PBI-4050 in subjects with Alström Syndrome who have completed a preceding ProMetic-sponsored Alström Syndrome study with PBI-4050. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Alström Syndrome
- What is being tested?
- PBI-4050
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 9 Oct 2017to 8 May 2020
- Phase
- Phase 2/3
- 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 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject completed a preceding ProMetic-sponsored Alström syndrome study with PBI-4050.
- Subject has signed informed consent
- Subject has a documented diagnosis of Alström syndrome
- Subject receiving antidiabetic medications is able and willing to self-monitor blood glucose levels
- Subject must be willing to forego other forms of experimental drug treatment during the study.
- Female subjects of childbearing potential must have a negative serum pregnancy test at screening and agree to use adequate birth control from screening throughout the study and for 30 days after the last Investigational Medicinal Product (IMP) administration
- If a male subject has not been vasectomized at least 6 months before screening and partners with a woman of childbearing potential, he must be willing to use an acceptable contraceptive method throughout the study and for 30 days after the last IMP administration.
Who cannot join
- Subject discontinued PBI-4050 for safety reasons from any preceding ProMetic-sponsored Alström syndrome study with PBI-4050
- Subject has had a documented episode of severe hypoglycaemia within 12 months before screening and investigator judges that the subject is unable to adequately monitor their glucose levels.
- Subject has uncontrolled hypertension with BP > 170/100 mmHg as determined at screening.
- Woman who is pregnant, breast-feeding, or planning a pregnancy during the course of the study as determined at screening
- Subject has any condition that, in the investigator's opinion, is likely to interfere with study conduct and compliance
- Subject has a history of an allergic reaction to PBI-4050 or any of its excipients.
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
University Hospitals Birmingham NHS Foundation Trus
Birmingham, B15 2PR
About this study
Official title: An Open-Label Rollover Study of PBI 4050 in Subjects With Alström Syndrome
Phase 2, open-label, single-arm, multi-centre study evaluating the long term safety and tolerability of PBI-4050 in subjects with Alström Syndrome who have completed a preceding ProMetic-sponsored Alström Syndrome study with PBI-4050.
Read the detailed description
This is a Phase 2, open-label, single-arm, multi-centre study evaluating the long term safety and tolerability of PBI-4050 in subjects with Alström Syndrome who have completed the end-of-treatment (EoT) visit in a preceding ProMetic-sponsored Alström Syndrome study with PBI-4050. This study will also evaluate the efficacy and pharmacological effects of PBI 4050 in this multi faceted disorder. Approximately 20 to 30 subjects will be enrolled to receive 800 mg PBI-4050 once daily for 96 weeks or until product licensing or study termination by the Sponsor, whichever occurs first.
- Conditions
- Alström Syndrome
- Being tested
- PBI-4050
- Medical subject headings
- Alstrom Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PBI-4050Drug
Four 200 mg capsules (800 mg total) administered orally, once daily
| Study group | Role | What they receive |
|---|---|---|
| PBI-4050 | Experimental | Not described Receives: Drug: PBI-4050 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects with abnormal laboratory values and/or adverse events that are related to treatment
Measured: 96 weeks
Secondary outcomes (5)
Change from baseline in fasting plasma glucose over time
Measured: 96 weeks
Change from baseline in plasma insulin over time
Measured: 96 weeks
Change from baseline in glycated hemoglobin (HbA1c) over time
Measured: 96 weeks
Change from baseline in blood glucose as measured by weekly 4 point profile
Measured: 96 weeks
Change from baseline in the liver stiffness
Measured in kilopascal (kPa) correlated to fibrosis by using a FibroScan
Measured: 96 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jun 2017
- Study start9 Oct 2017
- Primary completion8 May 2020
- Study completion8 May 2020
Why it stopped: Terminated early due to redeployment of study site staff during Covid-19 pandemic
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alström SyndromeStudies of the same condition, open and under way.
- 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 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Liminal BioSciences Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03184584
- Sponsor's protocol ID
- PBI-4050-CT-9-10
- Status last verified
- Oct 2020
- First submitted
- 9 Jun 2017
- Last updated
- 3 Nov 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Rollover Study of PBI 4050 in Subjects With Alström Syndrome
- ClinicalTrials.gov ID
- NCT03184584
- Acronym / sponsor ID
- PBI-4050-CT-9-10
- Conditions
- Alström Syndrome
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Terminated early due to redeployment of study site staff during Covid-19 pandemic
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Liminal BioSciences Ltd.Industry (pharma, biotech, devices)
- Study start
- 9 Oct 2017 (actual)
- Primary completion
- 8 May 2020 (actual)
- Study completion
- 8 May 2020 (actual)
- First posted
- 12 Jun 2017 (actual)
- 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.
Phase 2, open-label, single-arm, multi-centre study evaluating the long term safety and tolerability of PBI-4050 in subjects with Alström Syndrome who have completed a preceding ProMetic-sponsored Alström Syndrome study with PBI-4050.
Detailed description
This is a Phase 2, open-label, single-arm, multi-centre study evaluating the long term safety and tolerability of PBI-4050 in subjects with Alström Syndrome who have completed the end-of-treatment (EoT) visit in a preceding ProMetic-sponsored Alström Syndrome study with PBI-4050. This study will also evaluate the efficacy and pharmacological effects of PBI 4050 in this multi faceted disorder. Approximately 20 to 30 subjects will be enrolled to receive 800 mg PBI-4050 once daily for 96 weeks or until product licensing or study termination by the Sponsor, whichever occurs first.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PBI-4050 | Experimental | Drug: PBI-4050 |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Subject completed a preceding ProMetic-sponsored Alström syndrome study with PBI-4050.
- Subject has signed informed consent
- Subject has a documented diagnosis of Alström syndrome
- Subject receiving antidiabetic medications is able and willing to self-monitor blood glucose levels
- Subject must be willing to forego other forms of experimental drug treatment during the study.
Key exclusion criteria (6 listed)
- Subject discontinued PBI-4050 for safety reasons from any preceding ProMetic-sponsored Alström syndrome study with PBI-4050
- Subject has had a documented episode of severe hypoglycaemia within 12 months before screening and investigator judges that the subject is unable to adequately monitor their glucose levels.
- Subject has uncontrolled hypertension with BP > 170/100 mmHg as determined at screening.
- Woman who is pregnant, breast-feeding, or planning a pregnancy during the course of the study as determined at screening
- Subject has any condition that, in the investigator's opinion, is likely to interfere with study conduct and compliance
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 |
|---|---|---|
| PBI-4050Four 200 mg capsules (800 mg total) administered orally, once daily | Drug | PBI-4050 |
Primary outcome
Number of subjects with abnormal laboratory values and/or adverse events that are related to treatment
Time frame: 96 weeks
5 secondary outcomes
Change from baseline in fasting plasma glucose over time
Time frame: 96 weeks
Change from baseline in plasma insulin over time
Time frame: 96 weeks
Change from baseline in glycated hemoglobin (HbA1c) over time
Time frame: 96 weeks
Change from baseline in blood glucose as measured by weekly 4 point profile
Time frame: 96 weeks
Change from baseline in the liver stiffness
Time frame: 96 weeks
Results and safety
The study was stopped early (reason given: Terminated early due to redeployment of study site staff during Covid-19 pandemic). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2020.
Related trials for Alström Syndrome
Recruiting
COhort for Bardet-Bield Syndrome and Alström Syndrome for Translational Research Monocentric Interventional Study
Bardet-Biedl Syndrome, Alström Syndrome
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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing PBI-4050 in people with Alström Syndrome, run by Liminal BioSciences Ltd. at 1 site in United Kingdom.
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 6 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 3 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in 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.
What does this combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Terminated early due to redeployment of study site staff during Covid-19 pandemic). 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?
Number of subjects with abnormal laboratory values and/or adverse events that are related to treatment (time frame: 96 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 3 Nov 2020.