Table 2.
Summary of clinical trials: lumacaftor monotherapy
| Measure, Test, and Assay |
||||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| Study Name | Trial Phase No. | Mutations | Age Group, yr | Sample Size | ppFEV1 | BMI | CFQ-R | Sweat Chloride | Conclusion (Failed or Approved) | References |
| Lumacaftor Monotherapy, 25 mg | 2 | Homozygous F508de1 | ≥18 | 18 | −2.53% (P > 0.05) [4] | −9.7 points (P > 0.05) [4] | +0.10 mmol/l (P > 0.05) [4] | Failed | (49) | |
| Lumacaftor Monotherapy, 50 mg | 2 | Homozygous F508de1 | ≥18 | 18 | −2.22% (P > 0.05) [4] | −10.8 points (P > 0.05) [4] | −4.61 mmol/l (P > 0.05) [4] | |||
| Lumacaftor Monotherapy, 100 mg | 2 | Homozygous F508de1 | ≥18 | 17 | +0.25% (P > 0.05) [4] | −5.8 points (P > 0.05) [4] | −6.13mmol/l (P < 0.05) [4] | |||
| Lumacaftor Monotherapy, 200 mg | 2 | Homozygous F508de1 | ≥18 | 19 | +0.40% (P > 0.05) [4] | −2.3 points (P > 0.05) [4] | −8.21 mmol/l (P < 0.01) [4] | |||
Numbers within brackets indicate n.