Table 2.
Outcomes of the initial 27 substudies (of 38 total) in NCI-MATCH.
| Arm | Molecular Aberration | Treatment | N Enrolled | N Evaluable† | Number of Responses (%) | 6-month PFS | Ref | Met Endpoint?* |
|---|---|---|---|---|---|---|---|---|
| A | EGFR activating mutations | afatinib | 19 | 14 | 1 (7.1%) | 8.9% | 18 | No |
| B | HER2 activating mutations | afatinib | 40 | 37 | 1 (2.7%) | 12.0% | 19 | No |
| F | ALK fusions | crizotinib | 5 | 4 | 2 (50.0%) | 25% | 20 | Yes |
| G | ROS1 fusions | crizotinib | 4 | 4 | 1 (25.0%) | 50% | 20 | No |
| H | BRAF V600E or V600K mutations | Dabrafenib/trametinib | 35 | 29 | 11 (37.9%) | 68.4% | 21 | Yes |
| I | PIK3CA mutation without RAS mutation or PTEN loss | taselisib | 70 | 61 | 0.0% | 19.9% | 22 | No |
| J | HER2 amplification | Trastuzumab/pertuzumab | 35 | 25 | 3 (12%) | 25.3% | 23 | No |
| K2 | FGFR mutation/fusion | erdafitinib | 35 | 21 | 3 (14.3%) | 36.8% | 24 | Yes |
| M | TSC1 or TSC2 Mutations | TAK-228 | 49 | 34 | 5 (14.7%) | 28.7% | 25 | No |
| N | PTEN aberration, with + expression on IHC | GSK2636771 | 24 | 22 | 0.0% | 4.8% | 26 | No |
| P | PTEN loss by IHC | GSK2636771 | 35 | 32 | 0.0% | 3.3% | 26 | No |
| Q | HER2 amplification | ado-trastuzumab emtansine | 38 | 36 | 2 (5.6%) | 23.6% | 27 | No |
| R | BRAF fusions/non-V600 mutations | trametinib | 35 | 32 | 1 (3.0%) | 17% | 28 | No |
| S1 | NF1 mutation | trametinib | 50 | 46 | 2 (4.3%) | 20.5% | 29 | No |
| S2 | GNAQ or GNA11 mutation | trametinib | 4 | 4 | 1 (25%) | 50% | 29 | No |
| T | SMO or PTCH1 mutations | vismodegib | 34 | 22 | 2 (9.1%) | 22.4% | 30 | No |
| U | NF2 mutation | Defactinib | 35 | 30 | 1 (3.3%) | 22.8% | 31 | No |
| V | C-kit mutations | Sunitinib | 10 | 8 | 2 (25%) | 25% | 32 | No |
| W | FGFR pathway aberrations | AZD4547 | 52 | 48 | 4 (8.3%) | 15.0% | 33 | No |
| Y | AKT mutations | capivasertib | 35 | 35 | 10 (28.6%) | 50.0% | 34 | Yes |
| Z1A | NRAS mutations | binimetinib | 53 | 47 | 1 (2.1%) | 29.2% | 35 | No |
| Z1B | CCND1/2/3 amp and Rb + | palbociclib | 40 | 32 | 0.0% | 16.0% | 36 | No |
| Z1D | dMMR status | nivolumab | 47 | 42 | 15 (35.7%) | 51.3% | 37 | Yes |
| Z1F | PIK3CA | copanlisib | 35 | 25 | 4 (16.0%) | 38% | 38 | Yes |
| Z1H | PTEN mut without PTEN protein loss | copanlisib | 35 | 23 | 1 (4.3%) | 14.3% | 39 | No |
| Z1K | AKT mutation | Ipatasertib | 35 | 26 | 6 (23.1%) | 52.4% | 40 | Yes |
| Z1L | BRAF Fusions or Non-V600E, Non-V600K BRAF Mutations | Ulixertinib | 35 | 26 | 0.0% | 5% | 41 | No |
†
Eligible, treated and variant confirmed by central laboratory testing.
*
A substudy with 31 or more analyzable patients was to be called positive if the null hypothesis of objective response rate (ORR) ≤ 5% could be rejected at the one-sided type 1 error rate of 1.8%; if there were fewer than 31 analyzable patients, a type I error of 5.0% was used. This requires five or more responses (partial or complete) for a substudy with 31 analyzable patients, i.e., ORR > 5/31 (16%).