Inflammation and FibrosisPhase 2NCT02739217
Safety and Tolerability of PBI-4050 and Its Effects on the Biomarkers in Subjects With Alström Syndrome
A Phase 2 study testing PBI-4050 in people with Inflammation and Fibrosis and Diabetes, run by Liminal BioSciences Ltd. at 1 site in United Kingdom.
Trial summary
This is a Phase 2, single-centre, single-arm, open-label study of the safety, tolerability, and effects on biomarkers of PBI-4050 in subjects with Alström syndrome for a treatment duration of 24 weeks. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Inflammation and Fibrosis, Diabetes
- 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
- 12 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 22 Feb 2016to 4 Jun 2018
- Phase
- Phase 2
- 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 is 16 years of age or older at screening.
- Subject has signed informed consent.
- Subject has a documented diagnosis of Alström syndrome
- Subject on diabetes treatment has been receiving the same antidiabetic agent(s) for a minimum of 1 month before screening.
- Subject is able and willing to self-monitor blood glucose level at home or can obtain adequate assistance from care givers.
- 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 has recent or on-going infection requiring systemic treatment with an anti-infective agent within 30 days before screening.
- Subject has had at least two documented episodes of severe hypoglycaemia within 12 months before screening
- Subject has uncontrolled hypertension with BP > 170/100 mmHg as determined at screening.
- Subject has alanine transaminase (ALT) or aspartate transaminase (AST) level ≥ 5 × upper limit of normal (ULN) at screening.
- Subject is currently using weight loss medications at screening. Subjects may be re-screened after stopping the weight loss medication for a period of at least 5 half-lives.
- Subject has used any moderate/potent inducer or inhibitor of CYP2C9 isozyme or strong inducer or inhibitor of cytochrome P450 (CYP) 3A isozyme within 30 days prior to the first study drug administration.
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- Subject has a history of chronic alcohol or other substance abuse 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 Trust
Birmingham, B15 2PR
About this study
Official title: A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Tolerability of PBI-4050 and of Its Effects on the Inflammatory, Fibrosis, Diabetes and Obesity Biomarkers in Subjects With Alström Syndrome
This is a Phase 2, single-centre, single-arm, open-label study of the safety, tolerability, and effects on biomarkers of PBI-4050 in subjects with Alström syndrome for a treatment duration of 24 weeks.
Subjects who complete the initial 24 weeks of treatment may continue treatment for an additional 36 or 48 weeks, provided the subject signs informed consent.
Read the detailed description
This is a Phase 2, single-centre, single-arm, open-label study of the safety, tolerability, and effects on biomarkers of PBI-4050 in subjects with Alström syndrome. Approximately 18 subjects will be enrolled. The duration of study participation is approximately 35 weeks for each subject and comprises of 9 on site visits and telephone contacts in between visits.
Subjects who complete the initial 24 weeks of treatment may continue treatment for an additional 36 or 48 weeks (Extension Period [EP]), provided the subject signs informed consent. The extension period includes a further 3 on site visits and telephone contacts in between visits. The total duration of the study participation is extended to approximately 71 or 83 weeks. The Data Safety Monitoring Board (DSMB) will determine if the safety data continue to support treatment for an additional 36 or 48 weeks.
At the completion of the EP End of Treatment, subjects will be allowed to enrol in the Alström Rollover Study PBI-4050-CT-9-10 and continue ongoing study medication without any break in treatment.
- Conditions
- Inflammation and Fibrosis, Diabetes
- Being tested
- PBI-4050
- Medical subject headings
- Inflammation, Fibrosis, Diabetes Mellitus
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 | Four 200 mg capsules (total 800 mg) administered orally, once daily. Receives: Drug: PBI-4050 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Description and number of abnormal laboratory values and adverse events that are related to treatment.
Measured: Primary on 24 weeks; Final on all data (including Extension Period)
Secondary outcomes (4)
Change from baseline in metabolic syndrome parameters over time.
Change from baseline in fasting plasma glucose over time. Change from baseline in fasting plasma insulin over time. Change from baseline in glycated hemoglobin (HbA1c) over time. Change from baseline in Homeostasis Model Assessment for steady state beta cell function (HOMA-B) and insulin sensitivity (HOMA-S) over time.
Measured: 24 weeks and end of Extension Period
Change from baseline in biomarkers in blood and urine over time
Percentage of reduction and/or increase of level of biomarkers
Measured: 24 weeks and end of Extension Phase
Change from baseline in cardiac function parameter: NT-proBNP
Measured: 24 weeks and end of Extension Phase
Change from baseline of antidiabetic treatment
Dosing change, new medication added or treatment discontinuation
Measured: 24 weeks and end of Extension Phase
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Apr 2016 (estimated)
- Study start22 Feb 2016
- Primary completion26 Sep 2017
- Study completion4 Jun 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Inflammation and FibrosisStudies 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Liminal BioSciences Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Tarekegn Hiwot, MD, Principal investigator, The Queen Elizabeth Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02739217
- Sponsor's protocol ID
- PBI-4050-ATX-9-05
- Status last verified
- Aug 2018
- First submitted
- 25 Feb 2016
- Last updated
- 29 Aug 2018
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Baig S, Veeranna V, Bolton S, Edwards N, Tomlinson JW, Manolopoulos K, Moran J, Steeds RP, Geberhiwot T. Treatment with PBI-4050 in patients with Alstrom syndrome: study protocol for a phase 2, single-Centre, single-arm, open-label trial. BMC Endocr Disord. 2018 Nov 26;18(1):88. doi: 10.1186/s12902-018-0315-6.
PubMed 30477455
Study at a glance
From the registry record, last updated by the sponsor on 29 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Tolerability of PBI-4050 and of Its Effects on the Inflammatory, Fibrosis, Diabetes and Obesity Biomarkers in Subjects With Alström Syndrome
- ClinicalTrials.gov ID
- NCT02739217
- Acronym / sponsor ID
- PBI-4050-ATX-9-05
- Conditions
- Inflammation and Fibrosis, Diabetes
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Liminal BioSciences Ltd.Industry (pharma, biotech, devices)
- Study start
- 22 Feb 2016 (actual)
- Primary completion
- 26 Sep 2017 (actual)
- Study completion
- 4 Jun 2018 (actual)
- First posted
- 15 Apr 2016 (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.
This is a Phase 2, single-centre, single-arm, open-label study of the safety, tolerability, and effects on biomarkers of PBI-4050 in subjects with Alström syndrome for a treatment duration of 24 weeks.
Subjects who complete the initial 24 weeks of treatment may continue treatment for an additional 36 or 48 weeks, provided the subject signs informed consent.
Detailed description
This is a Phase 2, single-centre, single-arm, open-label study of the safety, tolerability, and effects on biomarkers of PBI-4050 in subjects with Alström syndrome. Approximately 18 subjects will be enrolled. The duration of study participation is approximately 35 weeks for each subject and comprises of 9 on site visits and telephone contacts in between visits.
Subjects who complete the initial 24 weeks of treatment may continue treatment for an additional 36 or 48 weeks (Extension Period [EP]), provided the subject signs informed consent. The extension period includes a further 3 on site visits and telephone contacts in between visits. The total duration of the study participation is extended to approximately 71 or 83 weeks. The Data Safety Monitoring Board (DSMB) will determine if the safety data continue to support treatment for an additional 36 or 48 weeks.
At the completion of the EP End of Treatment, subjects will be allowed to enrol in the Alström Rollover Study PBI-4050-CT-9-10 and continue ongoing study medication without any break in treatment.
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-4050Four 200 mg capsules (total 800 mg) administered orally, once daily. |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Subject is 16 years of age or older at screening.
- Subject has signed informed consent.
- Subject has a documented diagnosis of Alström syndrome
- Subject on diabetes treatment has been receiving the same antidiabetic agent(s) for a minimum of 1 month before screening.
- Subject is able and willing to self-monitor blood glucose level at home or can obtain adequate assistance from care givers.
Key exclusion criteria (10 listed)
- Subject has recent or on-going infection requiring systemic treatment with an anti-infective agent within 30 days before screening.
- Subject has had at least two documented episodes of severe hypoglycaemia within 12 months before screening
- Subject has uncontrolled hypertension with BP > 170/100 mmHg as determined at screening.
- Subject has alanine transaminase (ALT) or aspartate transaminase (AST) level ≥ 5 × upper limit of normal (ULN) at screening.
- Subject is currently using weight loss medications at screening. Subjects may be re-screened after stopping the weight loss medication for a period of at least 5 half-lives.
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
Description and number of abnormal laboratory values and adverse events that are related to treatment.
Time frame: Primary on 24 weeks; Final on all data (including Extension Period)
4 secondary outcomes
Change from baseline in metabolic syndrome parameters over time.
Time frame: 24 weeks and end of Extension Period
Change from baseline in biomarkers in blood and urine over time
Time frame: 24 weeks and end of Extension Phase
Change from baseline in cardiac function parameter: NT-proBNP
Time frame: 24 weeks and end of Extension Phase
Change from baseline of antidiabetic treatment
Time frame: 24 weeks and end of Extension Phase
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Aug 2018.
- Derived from this study: PubMed 30477455Baig S, Veeranna V, Bolton S, Edwards N, Tomlinson JW, Manolopoulos K, Moran J, Steeds RP, Geberhiwot T. Treatment with PBI-4050 in patients with Alstrom syndrome: study protocol for a phase 2, single-Centre, single-arm, open-label trial. BMC Endocr Disord. 2018 Nov 26;18(1):88. doi: 10.1186/s12902-018-0315-6.
- Individual participant dataSharing planned: No
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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.
Tarekegn Hiwot, MD principal investigator
The Queen Elizabeth Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PBI-4050 in people with Inflammation and Fibrosis and Diabetes, 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 6 inclusion and 10 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: finished, as of the sponsor's last update on 29 Aug 2018. 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 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Description and number of abnormal laboratory values and adverse events that are related to treatment. (time frame: Primary on 24 weeks; Final on all data (including Extension Period)). 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 NCT02739217, which also lists 1 linked publication.
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 29 Aug 2018.