Please select the arm or intervention descriptions that belong to the clinical trial below.
Title: A Phase 2b Extension Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis
Brief Summary: In some patients with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein. PTC124 has been shown to partially restore CFTR production in animals with CF due to a nonsense mutation. In an ongoing Phase 2a study being performed at the Hadassah University Hospital - Mount Scopus in Israel, patients with nonsense-mutation-mediated CF have received PTC124 in two 14-day treatment courses. Preliminary results from that study indicate that PTC124 has pharmacodynamic activity of PTC124 in CF and that PTC124 can be safely administered in this patient population. This Phase 2b extension study is designed to evaluate the activity and safety of 3 months (approximately 84 days) of continuous treatment with PTC124 in the same patients who were enrolled to the Phase 2a study. The main purpose of this study is to understand whether PTC124 can be safely administered and achieve pharmacodynamic activity in patients with CF due to a nonsense mutation.


Options:
A: Arm Label: VX-661/Ivacaftor combination; Type: EXPERIMENTAL; Interventions: Drug: VX-661/Ivacaftor, Drug: Ivacaftor, Drug: Placebo matched to Ivacaftor | Arm Label: Ivacaftor monotherapy; Type: EXPERIMENTAL; Interventions: Drug: Ivacaftor, Drug: Placebo matched to VX-661/ ivacaftor | Arm Label: Placebo; Type: PLACEBO_COMPARATOR; Interventions: Drug: Placebo matched to VX-661/ ivacaftor, Drug: Placebo matched to Ivacaftor | Intervention Type: DRUG; Name: VX-661/Ivacaftor; Assigned to Arm(s): VX-661/Ivacaftor combination | Intervention Type: DRUG; Name: Ivacaftor; Assigned to Arm(s): Ivacaftor monotherapy, VX-661/Ivacaftor combination | Intervention Type: DRUG; Name: Placebo matched to VX-661/ ivacaftor; Assigned to Arm(s): Ivacaftor monotherapy, Placebo | Intervention Type: DRUG; Name: Placebo matched to Ivacaftor; Assigned to Arm(s): Placebo, VX-661/Ivacaftor combination
B: Arm Label: Placebo (pooled two times a day [BID]/three times a day [TID]); Type: PLACEBO_COMPARATOR; Interventions: Drug: Placebo two times a day (BID)/three times a day (TID) | Arm Label: AZLI (pooled two times a day [BID]/three times a day [TID]); Type: EXPERIMENTAL; Interventions: Drug: AZLI 75 mg two times a day (BID)/three times a day (TID) | Intervention Type: DRUG; Name: AZLI 75 mg two times a day (BID)/three times a day (TID); Assigned to Arm(s): AZLI (pooled two times a day [BID]/three times a day [TID]) | Intervention Type: DRUG; Name: Placebo two times a day (BID)/three times a day (TID); Assigned to Arm(s): Placebo (pooled two times a day [BID]/three times a day [TID])
C: Arm Label: TEZ/IVA; Type: ACTIVE_COMPARATOR; Interventions: Drug: TEZ/IVA, Drug: IVA | Arm Label: ELX/TEZ/IVA; Type: EXPERIMENTAL; Interventions: Drug: ELX/TEZ/IVA, Drug: IVA | Intervention Type: DRUG; Name: ELX/TEZ/IVA; Assigned to Arm(s): ELX/TEZ/IVA | Intervention Type: DRUG; Name: TEZ/IVA; Assigned to Arm(s): TEZ/IVA | Intervention Type: DRUG; Name: IVA; Assigned to Arm(s): ELX/TEZ/IVA, TEZ/IVA
D: Arm Label: PTC124 PO; Type: EXPERIMENTAL; Interventions: Drug: PTC124 | Intervention Type: DRUG; Name: PTC124; Assigned to Arm(s): PTC124 PO

Answer: D