ABC
Anti-PD 1 Brain Collaboration for Patients With Melanoma Brain Metastases
Phase 2N = 79NCT02374242
- Population
- Immunotherapy-naive adults with melanoma brain metastases (79 enrolled; 76 received study drug)
- Design
- Open-label multicentre phase 2 study at four Australian sites in immunotherapy-naive patients with melanoma brain metastases. Cohorts A and B were randomised (30:24 after a safety run-in of six patients) in patients with asymptomatic brain metastases and no previous local brain therapy; cohort C (non-randomised) enrolled patients with failed local therapy, neurological symptoms or leptomeningeal disease. Primary end point intracranial response from week 12. The registry record lists 76 enrolled; the papers report 79 enrolled (36, 27 and 16) of whom 76 received study drug (3 ineligible, excluded before treatment). Cohorts A and B are the randomised comparison; cohort B is labelled the control arm only because it is the comparator group of the 2018 and 2025 report titles; the abstracts report no formal between-cohort test.
- Primary endpoint
- Intracranial response rate
- Arms
- Cohort A: nivolumab plus ipilimumab (experimental, n = 36): Nivolumab 1 mg per kilogram plus ipilimumab 3 mg per kilogram every 3 weeks for four doses, then nivolumab 3 mg per kilogram every 2 weeks
- Cohort B: nivolumab (control, n = 27): Nivolumab 3 mg per kilogram every 2 weeks
- Cohort C (non-randomised): nivolumab (experimental, n = 16): Nivolumab 3 mg per kilogram every 2 weeks
- People
- Long GV, senior author McArthur GA
Δ Intracranial response rate+26 pointsΔ Intracranial response rate+31 pointsΔ 84-month PFS+27 pointsΔ 84-month OS+22 points
Show the numbers
| Endpoint | Analysis | Cohort A: nivolumab plus ipilimumab / Cohort C (non-randomised): nivolumab | Cohort B: nivolumab | Effect |
|---|---|---|---|---|
| Intracranial response ratePatients who received at least one dose of study drug (35, 25 and 16) | primarymedian follow-up 17 monthsLong 2018 | 46% (29–63%) | 20% (7–41%) | Median follow-up 17 months at the data cutoff of 28 August 2017. Intracranial complete responses in 6 patients (17%) in cohort A, 3 (12%) in cohort B and none in cohort C. Grade 3 or 4 treatment-related adverse events in 54%, 16% and 13%. |
| Intracranial response ratePatients who received at least one dose of study drug | long term-updateLong 2025 | 51% (34–69%) | 20% (7–41%) | Median follow-up 7.6 years at the data cutoff of 26 March 2024. |
| PFSPatients who received at least one dose of study drugIntracranial progression-free survival | long term-updateLong 2025 | 42% at 7 y (29–63%) | 15% at 7 y (6–39%) | Seven-year intracranial progression-free survival. |
| OSPatients who received at least one dose of study drug | long term-updateLong 2025 | 48% at 7 y (34–68%) | 26% at 7 y (13–51%) | Seven-year overall survival; secondary end point. Cohort C was not randomised. |
| Endpoint | Analysis | Cohort A: nivolumab plus ipilimumab / Cohort C (non-randomised): nivolumab | Cohort B: nivolumab | Effect |
|---|---|---|---|---|
| Intracranial response ratePatients who received at least one dose of study drug (35, 25 and 16) | primarymedian follow-up 17 monthsLong 2018 | 46% (29–63%) | 20% (7–41%) | Median follow-up 17 months at the data cutoff of 28 August 2017. Intracranial complete responses in 6 patients (17%) in cohort A, 3 (12%) in cohort B and none in cohort C. Grade 3 or 4 treatment-related adverse events in 54%, 16% and 13%. |
| Intracranial response ratePatients who received at least one dose of study drug | long term-updateLong 2025 | 51% (34–69%) | 20% (7–41%) | Median follow-up 7.6 years at the data cutoff of 26 March 2024. |
| PFSPatients who received at least one dose of study drugIntracranial progression-free survival | long term-updateLong 2025 | 42% at 7 y (29–63%) | 15% at 7 y (6–39%) | Seven-year intracranial progression-free survival. |
| OSPatients who received at least one dose of study drug | long term-updateLong 2025 | 48% at 7 y (34–68%) | 26% at 7 y (13–51%) | Seven-year overall survival; secondary end point. Cohort C was not randomised. |
Appears in
Sources
Long GV, Atkinson V, Lo S, Sandhu S, Guminski AD, Brown MP, et al. Combination nivolumab and ipilimumab or nivolumab alone in melanoma brain metastases: a multicentre randomised phase 2 study. Lancet Oncol. 2018;19(5):672-681.
Long GV, Atkinson V, Lo SN, Guminski AD, Sandhu SK, Brown MP, et al. Ipilimumab plus nivolumab versus nivolumab alone in patients with melanoma brain metastases (ABC): 7-year follow-up of a multicentre, open-label, randomised, phase 2 study. Lancet Oncol. 2025;26(3):320-330.