A pilot study of vidofludimus calcium for treatment of primary sclerosing cholangitis
Abstract The purpose of this pilot study was to explore the efficacy, safety, and tolerability of vidofludimus calcium (VC) in the treatment of primary sclerosing cholangitis (PSC). This was a single‐arm open‐label pilot study with a cohort of 18 patients with PSC. Study patients received VC for a p...
| Main Authors: | , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Wolters Kluwer Health/LWW
2022-07-01
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| Series: | Hepatology Communications |
| Online Access: | https://doi.org/10.1002/hep4.1926 |
| _version_ | 1811174096751296512 |
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| author | Elizabeth J. Carey John Eaton Mitchell Clayton Andrea Gossard Sara Iqbal Hamid Ullah Nan Zhang Richard Butterfield Keith D. Lindor |
| author_facet | Elizabeth J. Carey John Eaton Mitchell Clayton Andrea Gossard Sara Iqbal Hamid Ullah Nan Zhang Richard Butterfield Keith D. Lindor |
| author_sort | Elizabeth J. Carey |
| collection | DOAJ |
| description | Abstract The purpose of this pilot study was to explore the efficacy, safety, and tolerability of vidofludimus calcium (VC) in the treatment of primary sclerosing cholangitis (PSC). This was a single‐arm open‐label pilot study with a cohort of 18 patients with PSC. Study patients received VC for a period of 6 months. The study was undertaken at two sites, Mayo Clinic, Rochester, MN, and Mayo Clinic, Phoenix, AZ. The primary endpoint of the study was improvement of serum alkaline phosphatase (ALP) at the end of the study. Secondary endpoints included assessment of other liver biomarkers (bilirubin, alanine aminotransferase, and aspartate aminotransferase). Of 18 patients enrolled, 11 completed the 6 months of study treatment. Patients who completed treatment versus those who did not were similar other than a significantly higher direct bilirubin at baseline in the group that completed treatment (mean ± SD, 0.4 ± 0.3 versus 0.1 ± 0.1, p = 0.04). By intent to treat analysis, the primary outcome was met in 16.7% (3/18) of patients. By per‐protocol analysis, including only patients who completed treatment, normalization of ALP occurred in 27.7% (3/11) at week 24 (95% confidence interval, 6.0% to 61.0%). VC was well tolerated with no drug‐related serious adverse events. Conclusion: This proof of concept study provides support for further exploration of VC in patients with PSC. |
| first_indexed | 2024-04-10T17:57:39Z |
| format | Article |
| id | doaj.art-be5638eb4bb74432ad93ac0f30b6a27d |
| institution | Directory Open Access Journal |
| issn | 2471-254X |
| language | English |
| last_indexed | 2024-04-10T17:57:39Z |
| publishDate | 2022-07-01 |
| publisher | Wolters Kluwer Health/LWW |
| record_format | Article |
| series | Hepatology Communications |
| spelling | doaj.art-be5638eb4bb74432ad93ac0f30b6a27d2023-02-02T17:27:39ZengWolters Kluwer Health/LWWHepatology Communications2471-254X2022-07-01671589159710.1002/hep4.1926A pilot study of vidofludimus calcium for treatment of primary sclerosing cholangitisElizabeth J. Carey0John Eaton1Mitchell Clayton2Andrea Gossard3Sara Iqbal4Hamid Ullah5Nan Zhang6Richard Butterfield7Keith D. Lindor8Division of Gastroenterology and Hepatology Mayo Clinic Phoenix Arizona USADivision of Gastroenterology and Hepatology Mayo Clinic Rochester Minnesota USADivision of Gastroenterology and Hepatology Mayo Clinic Rochester Minnesota USADivision of Gastroenterology and Hepatology Mayo Clinic Rochester Minnesota USAGastroenterology West Virginia University Morgantown West Virginia USAGastroenterology West Virginia University Morgantown West Virginia USADepartment of Quantitative Health Research Mayo Clinic Phoenix Arizona USADepartment of Quantitative Health Research Mayo Clinic Phoenix Arizona USADivision of Gastroenterology and Hepatology Mayo Clinic Phoenix Arizona USAAbstract The purpose of this pilot study was to explore the efficacy, safety, and tolerability of vidofludimus calcium (VC) in the treatment of primary sclerosing cholangitis (PSC). This was a single‐arm open‐label pilot study with a cohort of 18 patients with PSC. Study patients received VC for a period of 6 months. The study was undertaken at two sites, Mayo Clinic, Rochester, MN, and Mayo Clinic, Phoenix, AZ. The primary endpoint of the study was improvement of serum alkaline phosphatase (ALP) at the end of the study. Secondary endpoints included assessment of other liver biomarkers (bilirubin, alanine aminotransferase, and aspartate aminotransferase). Of 18 patients enrolled, 11 completed the 6 months of study treatment. Patients who completed treatment versus those who did not were similar other than a significantly higher direct bilirubin at baseline in the group that completed treatment (mean ± SD, 0.4 ± 0.3 versus 0.1 ± 0.1, p = 0.04). By intent to treat analysis, the primary outcome was met in 16.7% (3/18) of patients. By per‐protocol analysis, including only patients who completed treatment, normalization of ALP occurred in 27.7% (3/11) at week 24 (95% confidence interval, 6.0% to 61.0%). VC was well tolerated with no drug‐related serious adverse events. Conclusion: This proof of concept study provides support for further exploration of VC in patients with PSC.https://doi.org/10.1002/hep4.1926 |
| spellingShingle | Elizabeth J. Carey John Eaton Mitchell Clayton Andrea Gossard Sara Iqbal Hamid Ullah Nan Zhang Richard Butterfield Keith D. Lindor A pilot study of vidofludimus calcium for treatment of primary sclerosing cholangitis Hepatology Communications |
| title | A pilot study of vidofludimus calcium for treatment of primary sclerosing cholangitis |
| title_full | A pilot study of vidofludimus calcium for treatment of primary sclerosing cholangitis |
| title_fullStr | A pilot study of vidofludimus calcium for treatment of primary sclerosing cholangitis |
| title_full_unstemmed | A pilot study of vidofludimus calcium for treatment of primary sclerosing cholangitis |
| title_short | A pilot study of vidofludimus calcium for treatment of primary sclerosing cholangitis |
| title_sort | pilot study of vidofludimus calcium for treatment of primary sclerosing cholangitis |
| url | https://doi.org/10.1002/hep4.1926 |
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