Please select the arm or intervention descriptions that belong to the clinical trial below.
Title: A Pilot Trial of Phenylbutyrate/Genistein Duotherapy in Delta F508-Heterozygous Cystic Fibrosis Patients
Brief Summary: The purpose of this research study is to test a new combination of medicines, Phenylbutyrate and Genistein, to determine if they could be used to treat cystic fibrosis (CF). The most common genetic mutation found in patients with CF is called Delta F508. Due to this mutation, there is a lack of salt (chloride) movement in your nose, sinuses, lungs, intestines, pancreas and sweat glands. This lack of movement causes the clinical manifestations of the disease.

Although Phenylbutyrate has been extensively used to treat patients with rare metabolic diseases, Phenylbutyrate is an investigational drug for the purpose of this study. Genistein is a naturally occurring substance that is found in food products such as soy and tofu, but is also an investigational drug for this study. When used together, both drugs may be able to restore normal chloride and salt (water) movements in body organs and glands in people with CF.

We will be studying salt and water movement in the nose by a technique called nasal transepithelial potential difference (NPD).


Options:
A: Arm Label: Phenylbutyrate; Type: ACTIVE_COMPARATOR; Interventions: Drug: Sodium 4-Phenylbutyrate, Drug: Genistein (Unconjugated Isoflavones 100) | Arm Label: Placebo; Type: PLACEBO_COMPARATOR; Interventions: Drug: Genistein (Unconjugated Isoflavones 100), Drug: Placebo | Intervention Type: DRUG; Name: Sodium 4-Phenylbutyrate; Assigned to Arm(s): Phenylbutyrate | Intervention Type: DRUG; Name: Genistein (Unconjugated Isoflavones 100); Assigned to Arm(s): Phenylbutyrate, Placebo | Intervention Type: DRUG; Name: Placebo; Assigned to Arm(s): Placebo
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: Part A: VX-659 or Matching Placebo; Type: EXPERIMENTAL; Interventions: Drug: VX-659, Drug: VX-659 Matching Placebo | Arm Label: Part B: VX-659 or Matching Placebo; Type: EXPERIMENTAL; Interventions: Drug: VX-659, Drug: VX-659 Matching Placebo | Arm Label: Part C: VX-659 in TC with TEZ/IVA or Matching Triple Placebo; Type: EXPERIMENTAL; Interventions: Drug: VX-659, Drug: Tezacaftor, Drug: Ivacaftor, Drug: Triple Combination (TC) Matching Placebos | Arm Label: Part D: VX-659 in TC with TEZ/IVA or Matching Triple Placebo; Type: EXPERIMENTAL; Interventions: Drug: VX-659, Drug: Tezacaftor, Drug: Ivacaftor, Drug: Triple Combination (TC) Matching Placebos | Intervention Type: DRUG; Name: VX-659; Assigned to Arm(s): Part A: VX-659 or Matching Placebo, Part B: VX-659 or Matching Placebo, Part C: VX-659 in TC with TEZ/IVA or Matching Triple Placebo, Part D: VX-659 in TC with TEZ/IVA or Matching Triple Placebo | Intervention Type: DRUG; Name: Tezacaftor; Assigned to Arm(s): Part C: VX-659 in TC with TEZ/IVA or Matching Triple Placebo, Part D: VX-659 in TC with TEZ/IVA or Matching Triple Placebo | Intervention Type: DRUG; Name: Ivacaftor; Assigned to Arm(s): Part C: VX-659 in TC with TEZ/IVA or Matching Triple Placebo, Part D: VX-659 in TC with TEZ/IVA or Matching Triple Placebo | Intervention Type: DRUG; Name: VX-659 Matching Placebo; Assigned to Arm(s): Part A: VX-659 or Matching Placebo, Part B: VX-659 or Matching Placebo | Intervention Type: DRUG; Name: Triple Combination (TC) Matching Placebos; Assigned to Arm(s): Part C: VX-659 in TC with TEZ/IVA or Matching Triple Placebo, Part D: VX-659 in TC with TEZ/IVA or Matching Triple Placebo
D: Arm Label: GLPG2222 Dose 1; Type: EXPERIMENTAL; Interventions: Drug: GLPG2222 150 mg q.d. | Arm Label: GLPG2222 Dose 2; Type: EXPERIMENTAL; Interventions: Drug: GLPG2222 300 mg q.d. | Arm Label: Placebo; Type: PLACEBO_COMPARATOR; Interventions: Drug: Placebo | Intervention Type: DRUG; Name: GLPG2222 150 mg q.d.; Assigned to Arm(s): GLPG2222 Dose 1 | Intervention Type: DRUG; Name: GLPG2222 300 mg q.d.; Assigned to Arm(s): GLPG2222 Dose 2 | Intervention Type: DRUG; Name: Placebo; Assigned to Arm(s): Placebo

Answer: A