A Phase 2 Study of Galicaftor/Navocaftor/ABBV-119 Combination Therapy in Subjects with Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation.

A Phase 2 Study of Galicaftor/Navocaftor/ABBV-119 Combination Therapy in Subjects with Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation. - M19-771

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON49886

Enrollment

Registered

2021-09-23

Start date

2021-10-28

Completion date

Unknown

Last updated

2025-08-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

CF mucoviscidosis

Interventions

Participants in arm 1 will receive oral capsules of galicaftor/navocaftor dual combination for 28 days followed by galicaftor/navocaftor/ABBV-119 triple combination for 28 days. All other participan

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

Inclusion criteria: 1. Confirmed clinical diagnosis of CF, and genotype homozygous for the F508del CFTR mutation for Cohort 1 and Cohort 3, heterozygous for F508del CFTR mutation and a minimal function mutation for Cohort 2 and Cohort 3. 2. ppFEV1 >= 40% and = 60 mmol/L for Cohort 1 and Cohort 2, and this criteria does not apply to Cohort 3. 7. No history of diseases aggravated or triggered by ultraviolet radiation and no history of abnormal reaction photosensitivity or photoallergy to sunlight, or artificial source of intense light, especially ultraviolet light.

Exclusion criteria

Exclusion criteria: 1. Cirrhosis with or without portal hypertension (e.g., splenomegaly, esophageal varices) or history of clinically significant liver disease. 3. History of malignancy within past 5 years (except for excised basal cell carcinoma of the skin with no recurrence, or treated carcinoma in situ of the cervix with no recurrence). 4. Recent (within the past 6 months) history of drug or alcohol abuse that might preclude adherence to the protocol, in the opinion of the investigator. 5. Smoking or vaping tobacco or cannabis products within 6 months before Screening. 6. History of solid organ or hematopoietic transplantation. 7. History of known sensitivity to any component of the study drug. 8. Need for supplemental oxygen while awake, or >2 L/minute while sleeping. 10. Evidence of active SARS-CoV-2 infection. If a subject has signs/symptoms suggestive of SARS CoV-2 infection, they should undergo molecular (e.g., polymerase chain reaction [PCR]) testing to rule out SARS-CoV-2 infection. Subjects who do not meet SARS-CoV-2 infection eligibility criteria must be screen failed and may only rescreen after they meet the SARS-CoV-2 infection viral clearance criteria listed in the protocol.

Design outcomes

Primary

Measure	Time frame
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1).	—

Secondary

Measure	Time frame
1. Absolute change from Baseline in Sweat Chloride (SwCl). 2. Absolute change from Baseline in forced vital capacity [FVC]. 3. Absolute change from Baseline in forced expiratory flow at mid-lung capacity [FEF25-75]. 4. Relative changes from Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1). 5. Relative changes from Baseline in forced vital capacity [FVC]. 6. Relative changes from Baseline in Forced Expiratory Flow Between 25% and 75% of Exhaled Volume (FEF25-75). 7. Absolute change in CF Questionnaire-Revised (CFQ-R) respiratory domain score from Baseline.	—

Measure

Time frame

1. Absolute change from Baseline in Sweat Chloride (SwCl). 2. Absolute change from Baseline in forced vital capacity [FVC]. 3. Absolute change from Baseline in forced expiratory flow at mid-lung capacity [FEF25-75]. 4. Relative changes from Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1). 5. Relative changes from Baseline in forced vital capacity [FVC]. 6. Relative changes from Baseline in Forced Expiratory Flow Between 25% and 75% of Exhaled Volume (FEF25-75). 7. Absolute change in CF Questionnaire-Revised (CFQ-R) respiratory domain score from Baseline.

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Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)