Please select the arm or intervention descriptions that belong to the clinical trial below.
Title: A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis
Brief Summary: Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A specific type of mutation called a nonsense (premature stop codon) mutation is the cause of CF in approximately 10% of patients with the disease. Ataluren is an orally delivered investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 3 trial that will evaluate the clinical benefit of ataluren in adult and pediatric participants with CF due to a nonsense mutation. The main goals of the study are to understand whether ataluren can improve pulmonary function and whether the drug can safely be given for a long period of time. The study will also assess the effects of ataluren on CF pulmonary exacerbation frequency, cough frequency, health-related quality of life, antibiotic use for CF-related infections, CF-related disruptions to daily living, body weight, and CF pathophysiology.


Options:
A: Arm Label: 1; Type: EXPERIMENTAL; Interventions: Drug: miglustat | Arm Label: 2; Type: PLACEBO_COMPARATOR; Interventions: Drug: placebo | Intervention Type: DRUG; Name: miglustat; Assigned to Arm(s): 1 | Intervention Type: DRUG; Name: placebo; Assigned to Arm(s): 2
B: Arm Label: Part 1: Placebo; Type: PLACEBO_COMPARATOR; Interventions: Drug: Placebo (matched to VX-659/TEZ/IVA) | Arm Label: Part 1: VX-659/TEZ/IVA TC - Low Dose; Type: EXPERIMENTAL; Interventions: Drug: VX-659, Drug: TEZ/IVA, Drug: IVA | Arm Label: Part 1: VX-659/TEZ/IVA TC - Medium Dose; Type: EXPERIMENTAL; Interventions: Drug: VX-659, Drug: TEZ/IVA, Drug: IVA | Arm Label: Part 1: VX-659/TEZ/IVA TC - High Dose; Type: EXPERIMENTAL; Interventions: Drug: VX-659, Drug: TEZ/IVA, Drug: IVA | Arm Label: Part 2: TEZ/IVA; Type: ACTIVE_COMPARATOR; Interventions: Drug: TEZ/IVA, Drug: IVA | Arm Label: Part 2: VX-659/TEZ/IVA TC; Type: EXPERIMENTAL; Interventions: Drug: VX-659, Drug: TEZ/IVA, Drug: IVA | Arm Label: Part 3: Placebo; Type: PLACEBO_COMPARATOR; Interventions: Drug: Placebo (matched to VX-659/TEZ/VX-561) | Arm Label: Part 3: VX-659/TEZ/VX-561 TC; Type: EXPERIMENTAL; Interventions: Drug: VX-659, Drug: TEZ, Drug: VX-561 | Intervention Type: DRUG; Name: VX-659; Assigned to Arm(s): Part 1: VX-659/TEZ/IVA TC - High Dose, Part 1: VX-659/TEZ/IVA TC - Low Dose, Part 1: VX-659/TEZ/IVA TC - Medium Dose, Part 2: VX-659/TEZ/IVA TC, Part 3: VX-659/TEZ/VX-561 TC | Intervention Type: DRUG; Name: TEZ/IVA; Assigned to Arm(s): Part 1: VX-659/TEZ/IVA TC - High Dose, Part 1: VX-659/TEZ/IVA TC - Low Dose, Part 1: VX-659/TEZ/IVA TC - Medium Dose, Part 2: TEZ/IVA, Part 2: VX-659/TEZ/IVA TC | Intervention Type: DRUG; Name: IVA; Assigned to Arm(s): Part 1: VX-659/TEZ/IVA TC - High Dose, Part 1: VX-659/TEZ/IVA TC - Low Dose, Part 1: VX-659/TEZ/IVA TC - Medium Dose, Part 2: TEZ/IVA, Part 2: VX-659/TEZ/IVA TC | Intervention Type: DRUG; Name: Placebo (matched to VX-659/TEZ/IVA); Assigned to Arm(s): Part 1: Placebo | Intervention Type: DRUG; Name: TEZ; Assigned to Arm(s): Part 3: VX-659/TEZ/VX-561 TC | Intervention Type: DRUG; Name: VX-561; Assigned to Arm(s): Part 3: VX-659/TEZ/VX-561 TC | Intervention Type: DRUG; Name: Placebo (matched to VX-659/TEZ/VX-561); Assigned to Arm(s): Part 3: Placebo
C: Arm Label: Part A; Type: EXPERIMENTAL; Interventions: Drug: lumacaftor, Drug: ivacaftor | Arm Label: Part B; Type: EXPERIMENTAL; Interventions: Drug: lumacaftor, Drug: ivacaftor | Intervention Type: DRUG; Name: lumacaftor; Assigned to Arm(s): Part A, Part B | Intervention Type: DRUG; Name: ivacaftor; Assigned to Arm(s): Part A, Part B
D: Arm Label: Ataluren; Type: EXPERIMENTAL; Interventions: Drug: Ataluren | Arm Label: Placebo; Type: PLACEBO_COMPARATOR; Interventions: Drug: Placebo | Intervention Type: DRUG; Name: Ataluren; Assigned to Arm(s): Ataluren | Intervention Type: DRUG; Name: Placebo; Assigned to Arm(s): Placebo

Answer: D