# ROCKstar

Source: https://onco.cc/trials/rockstar/  
OnCo record `rockstar` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

ROCKstar showed that belumosudil, a daily pill, improved chronic graft-versus-host disease in about three in four people whose disease had not settled after at least two earlier treatments, which led to its US approval in 2021.

## Summary

ROCKstar (study KD025-213) was an open-label, randomised phase 2 registration study of belumosudil, an oral ROCK2 inhibitor, in patients with chronic graft-versus-host disease after a donor stem-cell transplant whose disease was still active after 2 to 5 previous lines of systemic therapy. Patients were randomised to 200 mg once daily (66) or 200 mg twice daily (66). There was no control arm: the primary endpoint was best overall response by the 2014 NIH consensus criteria.

At a median follow-up of 14 months the best response rate was 74 percent with once-daily and 77 percent with twice-daily dosing (95% CI 62 to 84 and 65 to 87), with high response rates in all subgroups and complete responses in every affected organ. The median duration of response was 54 weeks, 44 percent of patients stayed on treatment for at least a year, and symptoms improved in 59 and 62 percent. Sixteen patients (12 percent) stopped because of possibly drug-related adverse events (Blood 2021).

The FDA approved the once-daily dose on 16 July 2021 on a stricter analysis of 65 patients that counted responses only through cycle 7 day 1: 75 percent responded (6 percent complete, 69 percent partial), the median duration of response was 1.9 months on that definition, and 62 percent of responders were alive without a new systemic therapy at least 12 months after response (FDA approval summary, Clinical Cancer Research 2022; the Rezurock label Table 6 gives the same figures). The registry lists 159 participants in all, including adolescent arms, and records the study as terminated in December 2023 because adolescents were hard to recruit, with no safety concern.

## Fields

- Kind: Trial
- Status: positive
- Last checked: 2026-09-24
- Also known as: KD025-213; DRI17633; The ROCKstar Study
- Registry id: NCT03640481
- Phase: 2
- Setting: Chronic graft-versus-host disease after allogeneic stem-cell transplant, still active after 2 to 5 previous lines of systemic therapy: belumosudil 200 mg once daily against 200 mg twice daily, with no control arm
- Sponsor: Kadmon, a Sanofi Company
- Enrolled: 159
- Result: Best overall response 74% (200 mg once daily) and 77% (200 mg twice daily); the FDA analysis of the once-daily dose found 75% through cycle 7 day 1.
- Outcomes: Best overall response rate (2014 NIH criteria): Belumosudil 200 mg once daily 74% vs Belumosudil 200 mg twice daily 77%; Overall response rate through cycle 7 day 1 (FDA approval analysis): Belumosudil 200 mg once daily 75%; Symptom improvement (Lee Symptom Scale): Belumosudil 200 mg once daily 59% vs Belumosudil 200 mg twice daily 62%
- Replication: No randomised trial has compared belumosudil with another treatment for chronic graft-versus-host disease.

## Sources

- ClinicalTrials.gov NCT03640481: https://clinicaltrials.gov/study/NCT03640481
- Blood 2021: https://doi.org/10.1182/blood.2021012021
- FDA approval summary (Clinical Cancer Research 2022): https://doi.org/10.1158/1078-0432.CCR-21-4176
- Rezurock label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=102e4ef4-7f84-4e34-8df1-479c24d1575d

## Connected records

- technologies: [Small-molecule kinase inhibitors](https://onco.cc/technologies/kinase-inhibitors/)
- drugs: [Belumosudil](https://onco.cc/drugs/belumosudil/)
- companies: [Sanofi](https://onco.cc/companies/sanofi/)
- terms: [Allogeneic stem cell transplant (allo-SCT)](https://onco.cc/terms/allogeneic-transplant/), [Graft-versus-host disease (GVHD) and graft-versus-leukaemia](https://onco.cc/terms/gvhd/)
- key papers: [Belumosudil for chronic graft-versus-host disease after 2 or more prior lines of therapy: the ROCKstar Study](https://onco.cc/key-papers/paper-rockstar-blood-2021/)

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