Table 3.

Individual patient treatment duration and clinical responses (NC cohort)

Patient*	Age, y	Sex	Fusion protein	Primary site of origin	Dose, QD	Best response by RECIST	No. of prior treatments	Duration of treatment, d
1	23	Male	BRD4-NUT	Head and neck	2	NE†	2	11
2	36	Male	BRD4-NUT	Thoracic	2	NE	2	9
3	43	Female	BRD4-NUT	Other (left kidney)	4	PD	0	17
4	17	Male	BRD4-NUT	Head and neck	4	SD	1	255
5	48	Female	BRD4-NUT	Head and neck	16	SD	0	43
6	19	Male	BRD4-NUT	Head and neck	30	PD‡	1	42
7	20	Female	BRD4-NUT	Thoracic	60	PD‡	1	8
8	23	Male	NA	Thoracic	60	PR§	1	82
9	27	Male	BRD3-NUT	Thoracic	80	PR‖	1	69
10	39	Female	NSD3-NUT	Thoracic	80	SD	0	48
11	36	Female	BRD3-NUT	Head and neck	80	PR¶	1	196
12	51	Female	BRD4-NUT	Head and neck	80	non-CR/non-PD#	1	55
13	37	Female	BRD4-NUT	Head and neck	80	SD	0	199
14	39	Female	BRD4-NUT	Head and neck	80	SD	2	75
15	23	Male	NA	Thoracic	80	NE	1	8
16	22	Male	BRD3-NUT	Thoracic	80	SD	1	114
17	17	Female	BRD4-NUT	Thoracic	80	PD	1	28
18	61	Female	BRD4-NUT	Thoracic	100	SD	1	113
19	18	Male	BRD3-NUT	Other (right scapula)	100	PR¶	2	186

^*All patients came off study treatment for PD except patient 10, who withdrew consent because of a disease-related pericardial effusion requiring drainage, and patient 13, who withdrew consent for multiple AEs, including periorbital edema around the resected lesion. Patient 4 underwent dose escalation from 4 to 8 to 16 mg once daily. Patients 9 and 13 had the dose increased from 80 mg to 100 mg once daily. Two patients had dose reduction due to thrombocytopenia, including patient 18 (from 100 mg to 80 mg in week 5 to 60 mg in week 13) and patient 19 (from 100 mg to 80 mg in week 12). BRD = bromodomain; CR = complete response; CT = computed tomography; FDG-PET = ¹⁸F-fluorodeoxyglucose positron emission tomography; NA = not available; NE = not evaluable due to insufficient or unavailable pre- or postbaseline data; NC = NUT carcinoma; NUT = nuclear protein in testis; PD = progressive disease; PR = partial response; QD = once daily; RECIST = Response Evaluation Criteria in Solid Tumors; SD = stable disease.

^†Considered to have clinical progression but was NE by RECIST 1.1.

^‡PD defined by presence of new lesion(s) without reassessment of baseline target lesions.

^§Unconfirmed PR with subsequent scans demonstrating PD by RECIST 1.1.

^‖Unconfirmed PR with subsequent FDG-PET scans demonstrating worsening bone disease without CT correlate; best response scored as stable disease.

^¶Confirmed response of PR by RECIST 1.1.

^#Nonmeasurable disease at baseline.