Table 1.
Summary of Treatment-emergent Adverse Events
| n (%) | BG00011 Dose Cohort |
Overall |
|||||
|---|---|---|---|---|---|---|---|
| 0.015
mg/kg n = 6 |
0.1
mg/kg n = 6 |
0.3
mg/kg n = 6 |
1.0
mg/kg n = 7 |
3.0
mg/kg n = 6 |
BG00011 n = 31 |
Placebo n = 10 |
|
| Completed study (8 doses and follow-up) | 4 (66.7) | 6 (100) | 6 (100) | 5 (71.4) | 2 (33.3)* | 23 (74.2) | 7 (70.0) |
| Any TEAE | 6 (100) | 5 (83.3) | 6 (100) | 4 (57.1) | 6 (100) | 27 (87.1) | 7 (70.0) |
| TEAE occurring in ⩾4 patients | |||||||
| Cough | 1 (16.7) | 0 | 1 (16.7) | 1 (14.3) | 2 (33.3) | 5 (16.1) | 1 (10.0) |
| Dyspnea | 1 (16.7) | 2 (33.3) | 0 | 1 (14.3) | 1 (16.7) | 5 (16.1) | 1 (10.0) |
| IPF exacerbation | 0 | 0 | 0 | 2 (28.6) | 3 (50.0) | 5 (16.1) | 0 |
| Hypoxia | 2 (33.3) | 0 | 0 | 2 (28.6) | 0 | 4 (12.9) | 0 |
| Upper RTI | 2 (33.3) | 0 | 1 (16.7) | 0 | 1 (16.7) | 4 (12.9) | 1 (10.0) |
| Dizziness | 0 | 1 (16.7) | 1 (16.7) | 1 (14.3) | 1 (16.7) | 4 (12.9) | 1 (10.0) |
| Rash | 0 | 2 (33.3) | 2 (33.3) | 0 | 0 | 4 (12.9) | 0 |
| Serious TEAE | 1 (16.7)† | 0 | 0 | 2 (28.6)‡ | 1 (16.7)§ | 4 (12.9) | 0 |
Definition of abbreviations: IPF = idiopathic pulmonary fibrosis; RTI = respiratory tract infection; TEAE = treatment-emergent adverse event.
Adverse events were coded using the Medical Dictionary for Regulatory Activities version 20.0. Safety was assessed in all patients who received at least one dose of the study drug.
The 3.0 mg/kg dose cohort was terminated early because of a clinically significant decline in respiratory function at Week 16 in four of six patients.
One patient experienced two serious TEAEs. A complete atrioventricular block was observed on Day 11, requiring pacemaker placement. The patient restarted treatment and completed dosing. On Day 105, during the follow-up period (approximately 49 days after dosing was completed), the patient experienced a serious adverse event of hypoxemia, which lasted for 2 days and resolved.
One patient had IPF exacerbation on Day 60. Lung transplantation was required, and the patient was ultimately withdrawn from the study. The second patient had type II second-degree atrioventricular block requiring pacemaker implantation, syncope, and jaw fracture on Day 26 and had an IPF exacerbation on Day 38 that resulted in death after 4 days.
Respiratory failure after viral respiratory infection on Day 69, which lasted for 2 days and resolved.