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Table 4 Summary of clinical studies investigating the efficacy of ataluren in patients with nonsense cystic fibrosis mutations

From: Targeted therapies to improve CFTR function in cystic fibrosis

Study name and reference

Kerem et al. 2008 [64]

Sermet-Gaudelus et al. 2010 [46]

Wilschanski et al. 2011 [65]

Kerem et al. 2014 [66]

Type of study

Phase II randomized crossover trial

Phase II randomized crossover trial

Extension of trial by Kerem et al. 2008 [45]

Phase III RCT

Number of participants

n = 23

n = 30

n = 19

n = 238

Duration

Cycle 1: 16 mg/kg/day for 14 days; no treatment for 14 days

2 × 28 days

12 weeks

48 weeks

Cycle 2: 40 mg/kg/day for 14 days; no treatment for 14 days

Cycle 1: 16 mg/kg/day for 14 days; no treatment for 14 days

Cycle 2: 40 mg/kg/day for 14 days; no treatment for 14 days

Group 1: 16 mg/kg/day

Group 2 : 40 mg/kg/day

 

Inclusion criteria

≥18 years

6–18 years

≥18 years

≥6 years

2 disease mutations, >1 nonsense

2 disease mutations, >1 nonsense

2 disease mutations, >1 nonsense

Nonsense mutations

Sweat chloride >40 mmol/L

Sweat chloride >40 mmol/L

Sweat chloride >40 mmol/L

Sweat chloride >40 mmol/L

Abnormal nasal potential difference

Abnormal nasal potential difference

Abnormal nasal potential difference

Abnormal nasal potential difference

FEV1 > 40 %

FEV1 > 40 %

FEV1 > 40 %

FEV1 40–90 %

O2 saturation ≥92 % room air

O2 saturation ≥92 % room air

O2 saturation ≥92 % room air

O2 saturation ≥92 % room air

Weight ≥25 kg

Weight ≥16 kg

Outcome measure

Treatment effect

Treatment effect

Treatment effect

Treatment effect

Mean FEV1 (percentage predicted)

Small increase (P = 0.037)

No significant difference

No significant difference

+3 % (P = 0.12)

Sweat chloride levels (mmol/L)

No significant difference

–

–

–

Chloride transport

Cycle 1: −7.1 (P < 0.0001)

Cycle 1: −4.6 mV (P = 0.037)

Group 1: −6.8 (P < 0.004)

–

Cycle 2: −3.7 (P = 0.032)

Cycle 2: −3.9 mV (P = 0.046)

Group 2: −3.4 (P = 0.025)

Nasal potential difference (mV)

(Change in basal nasal potential difference)

–

–

–

Cycle 1: +3.3 (P = 0.04)

Cycle 2: +3.1 (P = 0.13)

Weight (kg)

+0.6 kg (P < 0.0001)

No significant difference

–

–

Pulmonary exacerbations

–

–

–

Rate ratio 0.77 (P = 0.0992)

  1. FEV 1 percentage predicted forced expiratory volume in 1 second for age, sex and height, RCT randomized controlled trial