Not provided
Not provided
Not provided
Not provided
Not provided
low recruitment
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| All-trans retinoic acid (ATRA) therapy | Experimental | Fixed low dose of ATRA 10 mg twice daily for 24 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| All-trans retinoic acid | Drug | Fixed low dose of ATRA 10 mg twice daily for 24 weeks. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change of Serum Alkaline Phosphatase (ALP) | Blood will be drawn at each time point to compare pre- and post-treatment values for each individual. | Baseline to week 24. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline to week 24. |
| The Percent of Patients Who Have Reduction of Serum C4 by 50% |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| James Boyer, MD | Yale University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Yale University | New Haven | Connecticut | 06510 | United States |
Of the two patients who screened, both met inclusion and exclusion criterial and were enrolled
Patients were recruited from physicians case load at Yale University, Digestive Disease's Liver clinic between May 2018 and December 2018. Both participants were enrolled in May of 2018.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | All-trans Retinoic Acid (ATRA) Therapy | Fixed low dose of ATRA 10 mg twice daily for 24 weeks. All-trans retinoic acid: Fixed low dose of ATRA 10 mg twice daily for 24 weeks. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | All-trans Retinoic Acid (ATRA) Therapy | Fixed low dose of ATRA 10 mg twice daily for 24 weeks. All-trans retinoic acid: Fixed low dose of ATRA 10 mg twice daily for 24 weeks. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change of Serum Alkaline Phosphatase (ALP) | Blood will be drawn at each time point to compare pre- and post-treatment values for each individual. | Data were not collected | Posted | Baseline to week 24. |
|
|
Up to 28 weeks post treatment initiation.
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | All-trans Retinoic Acid (ATRA) Therapy | Fixed low dose of ATRA 10 mg twice daily for 24 weeks. All-trans retinoic acid: Fixed low dose of ATRA 10 mg twice daily for 24 weeks. |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| alkaline phosphatase increase | Hepatobiliary disorders | CTCAE (4.0) | Non-systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Research Coordinator | Yale Univeristy | 203 737 6839 | laura.cusack@yale.edu |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Sep 17, 2018 | Jun 4, 2021 | Prot_SAP_000.pdf |
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D015209 | Cholangitis, Sclerosing |
| ID | Term |
|---|---|
| D002761 | Cholangitis |
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
| D004066 | Digestive System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D014212 | Tretinoin |
| ID | Term |
|---|---|
| D014801 | Vitamin A |
| D012176 | Retinoids |
| D002338 | Carotenoids |
| D011090 | Polyenes |
| D000475 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Blood is drawn at each time point to assess the outcome. |
| 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. | 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. | 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. | 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. | Baseline to week 24. |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Secondary | 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. | Only 1 patient completed the study, and no trial end point was achieved so there was no data to record. . | Posted | Baseline to week 24. |
|
|
| Secondary | The Percent of Patients Who Have Reduction of Serum C4 by 50% | Blood is drawn at each time point to assess the outcome. | Only 1 patient completed the study, data not reported. | Posted | Baseline to week 24. |
|
|
| Secondary | The Percent of Patients Who Have Reduction of Serum Bile Acids by 50% | Blood is drawn at each time point to assess the outcome. | Only 1 patient completed the study, data not reported. | Posted | Baseline to week 24. |
|
|
| Secondary | 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. | Only 1 patient completed the study, data not reported. | Posted | Baseline to week 24. |
|
|
| Secondary | 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. | Only 1 patient completed the study, data not reported. | Posted | Baseline to week 24. |
|
|
| Secondary | 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. | Only 1 patient completed the study, data not reported. | Posted | Baseline to week 24. |
|
|
| 0 |
| 2 |
| 0 |
| 2 |
| 1 |
| 2 |
| Blood bilirubin increased | Hepatobiliary disorders | CTCAE (4.0) | Non-systematic Assessment |
|
Not provided
Not provided
Not provided
| Alkenes |
| D006839 | Hydrocarbons, Acyclic |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D053138 | Cyclohexenes |
| D003510 | Cyclohexanes |
| D003516 | Cycloparaffins |
| D006840 | Hydrocarbons, Alicyclic |
| D006844 | Hydrocarbons, Cyclic |
| D013729 | Terpenes |
| D004224 | Diterpenes |
| D010860 | Pigments, Biological |
| D001685 | Biological Factors |