ROCKstar
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.
Overview
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.
- 74 vs 77 out of 100 had their tumour shrink with Belumosudil 200 mg once daily compared with Belumosudil 200 mg twice daily; 3 fewer per 100.
- On this measure the first group did worse, not better.
- A response rate counts how many people had their tumours shrink by a set amount; shrinking is encouraging but does not by itself show that people live longer or feel better.
- 75 out of 100 people had their tumour shrink with Belumosudil 200 mg once daily.
- There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
- A response rate counts how many people had their tumours shrink by a set amount; shrinking is encouraging but does not by itself show that people live longer or feel better.
- 59 vs 62 out of 100 reached this endpoint with Belumosudil 200 mg once daily compared with Belumosudil 200 mg twice daily; 3 fewer per 100.
- On this measure the first group did worse, not better.
- This endpoint is not one of the standard survival or response measures; read it alongside the trial's primary result.
- These results apply to the people the trial enrolled: 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. People in a different situation may not see the same effect.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
159 enrolled.
95% CI 62 to 84; median follow-up 14 months · 95% CI 65 to 87
Source95% CI 63 to 85; complete response 6%, partial response 69%
Source| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Best overall response rate (2014 NIH criteria)primary | Belumosudil 200 mg once daily | 66 | 74% | - | - | link |
| Belumosudil 200 mg twice daily | 66 | 77% | ||||
| Overall response rate through cycle 7 day 1 (FDA approval analysis) | Belumosudil 200 mg once daily | 65 | 75% | - | - | link |
| Symptom improvement (Lee Symptom Scale) | Belumosudil 200 mg once daily | 66 | 59% | - | - | link |
| Belumosudil 200 mg twice daily | 66 | 62% |
Similar pages
not linked directly; found by shared links- PersonXiaojun Huang
Shares Graft-versus-host disease (GVHD) and graft-versus-leukaemia, Allogeneic stem cell transplant (allo-SCT).
- Key paperA phase 3, single-arm, prospective study of remestemcel-L, ex vivo culture-expanded adult human mesenchymal stromal cells for the treatment of pediatric patients who failed to respond to steroid treatment for acute graft-versus-host disease
Shares Graft-versus-host disease (GVHD) and graft-versus-leukaemia, Allogeneic stem cell transplant (allo-SCT).
- TrialMSB-GVHD001
Shares Graft-versus-host disease (GVHD) and graft-versus-leukaemia, Allogeneic stem cell transplant (allo-SCT).
- Trial(PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo
Shares Sanofi, Small-molecule kinase inhibitors.
- TermConditioning regimen (myeloablative, reduced-intensity)
Shares Graft-versus-host disease (GVHD) and graft-versus-leukaemia, Allogeneic stem cell transplant (allo-SCT).
- TrialZETA
Shares Sanofi, Small-molecule kinase inhibitors.
- CompanyBlueprint Medicines
Shares Sanofi, Small-molecule kinase inhibitors.
- TreatmentVandetanib
Shares Sanofi, Small-molecule kinase inhibitors.