Primary Sclerosing CholangitisPhase 2HAAPSNCT05295680
Oral Hymecromone to Treat Adolescents and Adults With Primary Sclerosing Cholangitis.
A Phase 2 study testing Hymecromone in people with Primary Sclerosing Cholangitis, run by Aparna Goel at 2 sites in United States.
Trial summary
Primary objective: To evaluate the efficacy of hymecromone plus standard of care compared with standard of care alone in the treatment of adolescents and adults with primary sclerosing cholangitis (PSC). Read more about this study
- Who is it for?
- Ages 14 to 75 years
- Conditions the study is for
- Primary Sclerosing Cholangitis
- What is being tested?
- Hymecromone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 2 sitesUnited States
- Study dates
- 10 May 2023to Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 14 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of primary sclerosing cholangitis confirmed by liver biopsy and/or imaging study
- If history of endoscopically confirmed inflammatory bowel disease, currently stable based on Mayo Score / Disease Activity Index (DAI) for Ulcerative Colitis Score ≤ 1, normal inflammatory markers (ESR, CRP and fecal calprotectin) and stable non-excluded medical therapy for at least 6 months
Who cannot join
- Currently receiving biologic therapies
- Known allergy to hymecromone
- Cholangiocarcinoma
- Pregnancy
- Serious liver disease
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Recruiting
Stanford Clinic
Redwood City, California, 94063
Aparna Goel, MD, (650) 498-7878, goela21@stanford.edu
- Recruiting
Stanford Clinic
Stanford, California, 94305
Leina Alrabadi, MD, (650) 721-2250, alrabadi@stanford.edu
About this study
Official title: A Study of Oral Hymecromone to Treat Adolescents and Adults With Primary Sclerosing Cholangitis(HAAPS Study).
Primary objective: To evaluate the efficacy of hymecromone plus standard of care compared with standard of care alone in the treatment of adolescents and adults with primary sclerosing cholangitis (PSC).
Secondary objectives: To evaluate the change in Alkaline Phosphatase (ALP) from baseline to 6 months post-treatment following treatment with hymecromone plus standard of care compared with standard of care.
To evaluate changes in biomarkers of PSC disease during hymecromone treatment, namely: (a) fibrotic effect (FibroScan); (b) inflammatory biomarkers (serum Hyaluronan (HA)); and, (c) T-cell count.
- Conditions
- Primary Sclerosing Cholangitis
- Being tested
- Hymecromone
- Medical subject headings
- Cholangitis, Sclerosing
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HymecromoneDrug
Also called Isochol
Hymecromone 400 mg 3 times daily by mouth.
| Study group | Role | What they receive |
|---|---|---|
| Hymecromone | Experimental | Participants will receive Hymecromone for six months + Standard Of Care (SOC), and will be followed for an additional nine months. Receives: Drug: Hymecromone |
| Standard Of Care (SOC) | No intervention | Participants will receive Standard Of Care (SOC), and will be followed for 15 months. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in serum gamma-glutamyltransferase (GGT) levels
Measured: Baseline to Month 6
Secondary outcomes (10)
Change in serum Alkaline Phosphatase (ALP) levels
Measured: Baseline to Month 6
Change in serum hyaluronan levels
Measured: Baseline to Month 6
Change in T-cell count
Measured: Baseline to Month 6
Change in fibrotic effect based on FibroScan
Fibrotic effect is the amount of liver with fatty change
Measured: Baseline to Month 6
Change in biliary tree anatomy (e.g. strictures) based on FibroScan
Measured: Baseline to Month 6
Change in serum inflammatory cytokine profile
This outcome measure will assess pro-inflammatory cytokines previously associated with biliary inflammation and other autoimmune diseases including IFNg, IL-6, and TNF.
Measured: Baseline to Month 6
Change in lymphocyte immunophenotype
Single cell analysis technique will be used to assess the lymphocytes (B- and T-cells) present in serum samples, including FoxP3+ regulatory T-cells, a tolerogenic lymphocyte subset with important roles in immune tolerance.
Measured: Baseline to Month 6
Plasma drug levels of 4-MU
Measured: Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
Plasma drug levels of 4-MUG
Measured: Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
Plasma drug levels of 4-MUS
Measured: Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2022
- Study start10 May 2023
- Primary completionMay 2027 (estimated)
- Study completionDec 2027 (estimated)
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 Primary Sclerosing CholangitisStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Aparna Goel
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Aparna Goel, Stanford University
- Investigators
- Aparna Goel, MD, Principal investigator, Stanford UniversityLeina Alrabadi, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05295680
- Sponsor's protocol ID
- 64030
- Acronym
- HAAPS
- Status last verified
- Nov 2024
- First submitted
- 4 Mar 2022
- Last updated
- 13 Jun 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Salman L, Martinez L, Faddoul G, Manning C, Ali K, Salman M, Vazquez-Padron R. Hyaluronan Inhibition as a Therapeutic Target for Diabetic Kidney Disease: What Is Next? Kidney360. 2023 Jun 1;4(6):e851-e860. doi: 10.34067/KID.0000000000000126. Epub 2023 Apr 14.
PubMed 37055910
Study at a glance
From the registry record, last updated by the sponsor on 13 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of Oral Hymecromone to Treat Adolescents and Adults With Primary Sclerosing Cholangitis(HAAPS Study).
- ClinicalTrials.gov ID
- NCT05295680
- Acronym / sponsor ID
- HAAPS / 64030
- Conditions
- Primary Sclerosing Cholangitis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Aparna GoelUniversity, hospital or other
- Study start
- 10 May 2023 (actual)
- Primary completion
- May 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 25 Mar 2022 (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.
Primary objective: To evaluate the efficacy of hymecromone plus standard of care compared with standard of care alone in the treatment of adolescents and adults with primary sclerosing cholangitis (PSC).
Secondary objectives: To evaluate the change in Alkaline Phosphatase (ALP) from baseline to 6 months post-treatment following treatment with hymecromone plus standard of care compared with standard of care.
To evaluate changes in biomarkers of PSC disease during hymecromone treatment, namely: (a) fibrotic effect (FibroScan); (b) inflammatory biomarkers (serum Hyaluronan (HA)); and, (c) T-cell count.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Hymecromone | Experimental | Drug: HymecromoneParticipants will receive Hymecromone for six months + Standard Of Care (SOC), and will be followed for an additional nine months. |
| Standard Of Care (SOC) | No intervention | –Participants will receive Standard Of Care (SOC), and will be followed for 15 months. |
Who may qualify
- Ages
- 14 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Diagnosis of primary sclerosing cholangitis confirmed by liver biopsy and/or imaging study
- If history of endoscopically confirmed inflammatory bowel disease, currently stable based on Mayo Score / Disease Activity Index (DAI) for Ulcerative Colitis Score ≤ 1, normal inflammatory markers (ESR, CRP and fecal calprotectin) and stable non-excluded medical therapy for at least 6 months
Key exclusion criteria (5 listed)
- Currently receiving biologic therapies
- Known allergy to hymecromone
- Cholangiocarcinoma
- Pregnancy
- Serious liver disease
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 |
|---|---|---|
| HymecromoneAlso: IsocholHymecromone 400 mg 3 times daily by mouth. | Drug | Hymecromone |
Primary outcome
Change in serum gamma-glutamyltransferase (GGT) levels
Time frame: Baseline to Month 6
10 secondary outcomes
Change in serum Alkaline Phosphatase (ALP) levels
Time frame: Baseline to Month 6
Change in serum hyaluronan levels
Time frame: Baseline to Month 6
Change in T-cell count
Time frame: Baseline to Month 6
Change in fibrotic effect based on FibroScan
Time frame: Baseline to Month 6
Change in biliary tree anatomy (e.g. strictures) based on FibroScan
Time frame: Baseline to Month 6
Change in serum inflammatory cytokine profile
Time frame: Baseline to Month 6
Change in lymphocyte immunophenotype
Time frame: Baseline to Month 6
Plasma drug levels of 4-MU
Time frame: Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
Plasma drug levels of 4-MUG
Time frame: Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
Plasma drug levels of 4-MUS
Time frame: Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jun 2025.
- Derived from this study: PubMed 37055910Salman L, Martinez L, Faddoul G, Manning C, Ali K, Salman M, Vazquez-Padron R. Hyaluronan Inhibition as a Therapeutic Target for Diabetic Kidney Disease: What Is Next? Kidney360. 2023 Jun 1;4(6):e851-e860. doi: 10.34067/KID.0000000000000126. Epub 2023 Apr 14.
- 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.
Aparna Goel, MD principal investigator
Stanford University
Leina Alrabadi, MD principal investigator
Stanford University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Hymecromone in people with Primary Sclerosing Cholangitis, run by Aparna Goel at 2 sites 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 2 inclusion and 5 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: looking for participants now, as of the sponsor's last update on 13 Jun 2025. 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. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in serum gamma-glutamyltransferase (GGT) levels (time frame: Baseline to Month 6). 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 NCT05295680, 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 13 Jun 2025.