Cholangitis, SclerosingPhase 2NCT03359174
An Efficacy Trial of Low Dose All-trans Retinoic Acid in Patients With Primary Sclerosing Cholangitis
A Phase 2 study testing All-trans retinoic acid in people with Cholangitis, Sclerosing, run by Yale University at 1 site in United States.
Trial summary
The purpose of this research study is to determine whether a low dose of ATRA will improve laboratory tests of liver and bile duct inflammation in patients with PSC. The investigators will also look for changes to other blood tests which are related to inflammation, scarring, and the immune system. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Cholangitis, Sclerosing
- What is being tested?
- All-trans retinoic acid
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- 29 May 2018to 1 Jan 2021
- 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 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males and females ages 18-80
- Diagnosis of large-duct PSC based on ERCP or MRCP, or liver biopsy findings without alternative explanation for findings, for at least 6 months.
- Serum ALP levels persistently more than 1.5 x upper limit of normal over the past 6 months.
- Ursodeoxycholic acid therapy must be discontinued for at least 3 months.
- At least 2 forms of barrier protection for males and females of child-bearing age.
Who cannot join
- Small duct PSC, overlap with autoimmune hepatitis, IgG4 disease or secondary sclerosing cholangitis.
- Any malignancy, presently or within the past 5 years, except adequately treated non-melanoma skin cancer, carcinoma in situ of the cervix or in situ prostate cancer.
- Viral hepatitis including hepatitis A, B, C, D, E.
- Decompensated cirrhosis, or planned liver transplantation.
- Recent diagnostic or therapeutic biliary manipulation (endoscopic, radiologic) within the past 3 months.
- Ascending Cholangitis requiring antibiotics within the past 3 months.
Show all 9Show fewer
- Uncontrolled IBD, or IBD requiring the use of steroids.
- Acute or Chronic Kidney Disease with serum creatinine > 2 mg/dL.
- Allergy to ATRA or vitamin A compounds.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Efficacy Trial of Low Dose All-trans Retinoic Acid (ATRA) in Patients With Primary Sclerosing Cholangitis
The purpose of this research study is to determine whether a low dose of ATRA will improve laboratory tests of liver and bile duct inflammation in patients with PSC. The investigators will also look for changes to other blood tests which are related to inflammation, scarring, and the immune system.
- Conditions
- Cholangitis, Sclerosing
- Being tested
- All-trans retinoic acid
- Medical subject headings
- Cholangitis, Sclerosing
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
All-trans retinoic acidDrug
Fixed low dose of ATRA 10 mg twice daily for 24 weeks.
| Study group | Role | What they receive |
|---|---|---|
| All-trans retinoic acid (ATRA) therapy | Experimental | Fixed low dose of ATRA 10 mg twice daily for 24 weeks. Receives: Drug: All-trans retinoic acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change of Serum Alkaline Phosphatase (ALP)
Blood will be drawn at each time point to compare pre- and post-treatment values for each individual.
Measured: Baseline to week 24.
Secondary outcomes (6)
Change in Patients Who Have Normalization of Serum ALP or Reduction to Less Than 1.5 x Upper Limit of Normal (ULN)
Blood is drawn at each time point to assess the outcome.
Measured: Baseline to week 24.
The Percent of Patients Who Have Reduction of Serum C4 by 50%
Blood is drawn at each time point to assess the outcome.
Measured: Baseline to week 24.
The Percent of Patients Who Have Reduction of Serum Bile Acids by 50%
Blood is drawn at each time point to assess the outcome.
Measured: Baseline to week 24.
The Percent of Patients Who Have Reduction of Serum Alanine Aminotransferase (ALT) by 50%
Blood is drawn at each time point to assess the outcome.
Measured: Baseline to week 24.
The Percent of Patients Who Have Reduction of Serum Enhanced Liver Fibrosis Score (ELF) by 10%
Blood is drawn at each time point to assess the outcome.
Measured: Baseline to week 24.
The Percent of Patients Who Have Improvement in Fibrosis Per Transient Elastography by at Least 1 Stage
Transient Elastography will be performed at baseline and week 24 to assess the outcome.
Measured: Baseline to week 24.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2017
- Study start29 May 2018
- Primary completion1 Dec 2020
- Study completion1 Jan 2021
Why it stopped: low recruitment
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 Cholangitis, SclerosingStudies of the same condition, open and under way.
- More trials run by Yale UniversityThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Yale University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- James Boyer, MD, Principal investigator, Yale University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03359174
- Sponsor's protocol ID
- 2000021447
- Status last verified
- Aug 2021
- First submitted
- 27 Nov 2017
- Last updated
- 9 Aug 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Efficacy Trial of Low Dose All-trans Retinoic Acid (ATRA) in Patients With Primary Sclerosing Cholangitis
- ClinicalTrials.gov ID
- NCT03359174
- Acronym / sponsor ID
- 2000021447
- Conditions
- Cholangitis, Sclerosing
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: low recruitment
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Yale UniversityUniversity, hospital or other
- Study start
- 29 May 2018 (actual)
- Primary completion
- 1 Dec 2020 (actual)
- Study completion
- 1 Jan 2021 (actual)
- First posted
- 2 Dec 2017 (actual)
- Results
- Posted 9 Aug 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this research study is to determine whether a low dose of ATRA will improve laboratory tests of liver and bile duct inflammation in patients with PSC. The investigators will also look for changes to other blood tests which are related to inflammation, scarring, and the immune system.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All-trans retinoic acid (ATRA) therapy | Experimental | Drug: All-trans retinoic acidFixed low dose of ATRA 10 mg twice daily for 24 weeks. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Males and females ages 18-80
- Diagnosis of large-duct PSC based on ERCP or MRCP, or liver biopsy findings without alternative explanation for findings, for at least 6 months.
- Serum ALP levels persistently more than 1.5 x upper limit of normal over the past 6 months.
- Ursodeoxycholic acid therapy must be discontinued for at least 3 months.
- At least 2 forms of barrier protection for males and females of child-bearing age.
Key exclusion criteria (9 listed)
- Small duct PSC, overlap with autoimmune hepatitis, IgG4 disease or secondary sclerosing cholangitis.
- Any malignancy, presently or within the past 5 years, except adequately treated non-melanoma skin cancer, carcinoma in situ of the cervix or in situ prostate cancer.
- Viral hepatitis including hepatitis A, B, C, D, E.
- Decompensated cirrhosis, or planned liver transplantation.
- Recent diagnostic or therapeutic biliary manipulation (endoscopic, radiologic) within the past 3 months.
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 |
|---|---|---|
| All-trans retinoic acidFixed low dose of ATRA 10 mg twice daily for 24 weeks. | Drug | All-trans retinoic acid (ATRA) therapy |
Primary outcome
Change of Serum Alkaline Phosphatase (ALP)
Time frame: Baseline to week 24.
Blood will be drawn at each time point to compare pre- and post-treatment values for each individual.
6 secondary outcomes
Change in Patients Who Have Normalization of Serum ALP or Reduction to Less Than 1.5 x Upper Limit of Normal (ULN)
Time frame: Baseline to week 24.
The Percent of Patients Who Have Reduction of Serum C4 by 50%
Time frame: Baseline to week 24.
The Percent of Patients Who Have Reduction of Serum Bile Acids by 50%
Time frame: Baseline to week 24.
The Percent of Patients Who Have Reduction of Serum Alanine Aminotransferase (ALT) by 50%
Time frame: Baseline to week 24.
The Percent of Patients Who Have Reduction of Serum Enhanced Liver Fibrosis Score (ELF) by 10%
Time frame: Baseline to week 24.
The Percent of Patients Who Have Improvement in Fibrosis Per Transient Elastography by at Least 1 Stage
Time frame: Baseline to week 24.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Aug 2021.
- 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.
James Boyer, MD principal investigator
Yale University
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 All-trans retinoic acid in people with Cholangitis, Sclerosing, run by Yale University at 1 site in United States.
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 5 inclusion and 9 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 Aug 2021. 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 States. 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 9 Aug 2021). 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?
Change of Serum Alkaline Phosphatase (ALP) (time frame: Baseline to week 24.). 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 9 Aug 2021.