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Oral Hymecromone to Treat Adolescents and Adults With Primary Sclerosing Cholangitis. (HAAPS)

Primary Purpose

Primary Sclerosing Cholangitis

Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Hymecromone
Sponsored by
Aparna Goel
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Sclerosing Cholangitis

Eligibility Criteria

14 Years - 75 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • Currently receiving biologic therapies
  • Known allergy to hymecromone
  • Cholangiocarcinoma
  • Pregnancy
  • Serious liver disease

Sites / Locations

  • Stanford ClinicRecruiting
  • Stanford ClinicRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

No Intervention

Arm Label

Hymecromone

Standard Of Care (SOC)

Arm Description

Participants will receive Hymecromone for six months + Standard Of Care (SOC), and will be followed for an additional nine months.

Participants will receive Standard Of Care (SOC), and will be followed for 15 months.

Outcomes

Primary Outcome Measures

Change in serum gamma-glutamyltransferase (GGT) levels

Secondary Outcome Measures

Change in serum Alkaline Phosphatase (ALP) levels
Change in serum hyaluronan levels
Change in T-cell count
Change in fibrotic effect based on FibroScan
Fibrotic effect is the amount of liver with fatty change
Change in biliary tree anatomy (e.g. strictures) based on FibroScan
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.
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.
Plasma drug levels of 4-MU
Plasma drug levels of 4-MUG
Plasma drug levels of 4-MUS

Full Information

First Posted
March 4, 2022
Last Updated
October 4, 2023
Sponsor
Aparna Goel
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1. Study Identification

Unique Protocol Identification Number
NCT05295680
Brief Title
Oral Hymecromone to Treat Adolescents and Adults With Primary Sclerosing Cholangitis.
Acronym
HAAPS
Official Title
A Study of Oral Hymecromone to Treat Adolescents and Adults With Primary Sclerosing Cholangitis(HAAPS Study).
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
May 10, 2023 (Actual)
Primary Completion Date
May 2025 (Anticipated)
Study Completion Date
May 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Aparna Goel

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief 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). 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Sclerosing Cholangitis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
24 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Hymecromone
Arm Type
Experimental
Arm Description
Participants will receive Hymecromone for six months + Standard Of Care (SOC), and will be followed for an additional nine months.
Arm Title
Standard Of Care (SOC)
Arm Type
No Intervention
Arm Description
Participants will receive Standard Of Care (SOC), and will be followed for 15 months.
Intervention Type
Drug
Intervention Name(s)
Hymecromone
Other Intervention Name(s)
Isochol
Intervention Description
Hymecromone 400 mg 3 times daily by mouth.
Primary Outcome Measure Information:
Title
Change in serum gamma-glutamyltransferase (GGT) levels
Time Frame
Baseline to Month 6
Secondary Outcome Measure Information:
Title
Change in serum Alkaline Phosphatase (ALP) levels
Time Frame
Baseline to Month 6
Title
Change in serum hyaluronan levels
Time Frame
Baseline to Month 6
Title
Change in T-cell count
Time Frame
Baseline to Month 6
Title
Change in fibrotic effect based on FibroScan
Description
Fibrotic effect is the amount of liver with fatty change
Time Frame
Baseline to Month 6
Title
Change in biliary tree anatomy (e.g. strictures) based on FibroScan
Time Frame
Baseline to Month 6
Title
Change in serum inflammatory cytokine profile
Description
This outcome measure will assess pro-inflammatory cytokines previously associated with biliary inflammation and other autoimmune diseases including IFNg, IL-6, and TNF.
Time Frame
Baseline to Month 6
Title
Change in lymphocyte immunophenotype
Description
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.
Time Frame
Baseline to Month 6
Title
Plasma drug levels of 4-MU
Time Frame
Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
Title
Plasma drug levels of 4-MUG
Time Frame
Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
Title
Plasma drug levels of 4-MUS
Time Frame
Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits

10. Eligibility

Sex
All
Minimum Age & Unit of Time
14 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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 Exclusion Criteria: Currently receiving biologic therapies Known allergy to hymecromone Cholangiocarcinoma Pregnancy Serious liver disease
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Aparna Goel, MD
Organizational Affiliation
Stanford University
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Leina Alrabadi, MD
Organizational Affiliation
Stanford University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Stanford Clinic
City
Redwood City
State/Province
California
ZIP/Postal Code
94063
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Aparna Goel, MD
Phone
650-498-7878
Email
goela21@stanford.edu
Facility Name
Stanford Clinic
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Leina Alrabadi, MD
Phone
650-721-2250
Email
alrabadi@stanford.edu

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Oral Hymecromone to Treat Adolescents and Adults With Primary Sclerosing Cholangitis.

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