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Clinical Trial Alerts – August 2019

Cystic Fibrosis Foundation issued the following clinical trial alerts in the month of August.

August 7, 2019

Study to evaluate the effects of the triple-combination modulator, elexacaftor/tezacaftor/ivacaftor (PROMISE)

Status: Enrolling

Description: This observational study will measure the effects of the triple-combination therapy, elexacaftor/tezacaftor/ivacaftor, in people with CF. These drugs are intended to help CFTR protein function closer to normal. This study will look at how treatment with the triple-combination therapy affects people with CF across many different aspects of the disease.

Age: 12 Years and Older

Mutation: Two Copies F508del or One Copy F508del

Fev1% Predicted: No FEV1 Limit

Number of Visits: 6

Length of Participation: 2 years

ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT04038047

August 21, 2019

Study of BI 1265162, a mucus clearance drug, in teens and adults with CF

Status: Enrolling

Description: This study will look at the safety and effectiveness of BI 1265162, a drug intended to improve the clearance of mucus from the lungs. This study is for people with CF ages 12 and older.

Age: 12 Years and Older

Mutation: No Mutation Requirement

Fev1% Predicted: 40 to 90%

Number of Visits: 5

Length of Participation: 7 weeks

ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT04059094

Phase 2a study of Galapagos GLPG2222 in adults with CF

Status: Completed with results

Description: This study evaluated the safety and effectiveness of the CFTR modulator GLPG2222. This study was for people with CF who have two copies of the F508del mutation.

Age: 18 Years and Older

Mutation: Two Copies F508del

Fev1% Predicted: 40% or greater

Number of Visits: 5

Length of Participation: 10 weeks

ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT03119649

Phase 3 study of tezacaftor (VX-661) combination drug in children with cystic fibrosis

Status: Completed with results

Description: This study evaluated the safety and tolerability of the drug tezacaftor (VX-661) in combination with ivacaftor (Kalydeco®) in children with CF. This study was for children who have two copies of the F508del mutation or one copy of F508del and a second mutation that is either residual function or results in a gating defect and responds to ivacaftor.

Age: 6 Years to 11 Years

Mutation: Two Copies F508del or One Copy F508del

Fev1% Predicted: 40% or greater

Number of Visits: 9

Length of Participation: 28 weeks

ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT02953314

Phase 1 exploratory study on the effect of the drug QR-010 on nasal cells

Status: Completed with results

Description: This study evaluated the effectiveness of the drug eluforsen (when sprayed in the nose) to improve nasal cell chloride transport. This study was for people with CF who have at least one copy of the F508del CFTR mutation.

Age: 18 Years and Older

Mutation: Two Copies F508del or One Copy F508del

Fev1% Predicted: 40% or greater

Number of Visits: 8

Length of Participation: 7 weeks

ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT02564354

SNO6: Phase 2 study of N91115 in adults with CF who are currently taking lumacaftor/ivacaftor

Status: Completed with results

Description: This study evaluated the safety and effectiveness of the CFTR modulator drug N91115 (cavosonstat). This study was for adults with cystic fibrosis who have two copies of the F508del CFTR mutation and were also taking lumacaftor/ivacaftor (Orkambi®).

Age: 18 Years and Older

Mutation: Two Copies F508del

Fev1% Predicted: 40 to 85%

Number of Visits: 10

Length of Participation: 16 weeks

ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT02589236

Phase 1b safety study of QR-010 in adults with CF

Status: Completed with results

Description: This study evaluated the safety and tolerability of the inhaled drug eluforsen in people with CF who have two copies of the F508del CFTR mutation.

Age: 18 Years to 60 Years

Mutation: Two Copies F508del

Fev1% Predicted: 70% or greater

Number of Visits: 10

Length of Participation: 8 weeks

ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT02532764

Study of Lung Clearance Index (LCI) in children 3 to 5 years old before and after taking ivacaftor (GOAL e2)

Status: Completed with results

Description: This study evaluated lung function in young children with CF before and after taking ivacaftor. This study was for children ages 3 to 5 years old who have at least one CFTR gating mutation.

Age: 3 Years to 5 Years

Mutation: One Copy F508del or No Copies F508del

Fev1% Predicted: No FEV1 Limit

Number of Visits: 5

Length of Participation: 6 months

ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT01521338

SHIP: Study of hypertonic saline in preschoolers

Status: Completed with results

Description: The SHIP study evaluated the safety and effectiveness of hypertonic saline (7%) in pre-school children with CF. 

Age: 3 Years to 5 Years

Mutation: No Mutation Requirement

Fev1% Predicted: Less than 99%

Number of Visits: 6

Length of Participation: 54 weeks

ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT02378467

Study of VX-661 combined with ivacaftor in adults with cystic fibrosis

Status: Completed with results

Description: This study evaluated the safety and the best way to dose VX-661 in combination with ivacaftor. This study was for adults with CF who have two copies of the F508del CFTR mutation.

Age: 18 Years and Older

Mutation: Two Copies F508del

Fev1% Predicted: 40 to 90%

Number of Visits: 8

Length of Participation: 20 weeks

ClinicalTrials.gov link: https://www.clinicaltrials.gov/ct2/show/NCT02070744

Arikace compared to TOBI® in people with CF with chronic Pseudomonas aeruginosa infections

Status: Completed with results

Description: This study looked at the effectiveness, safety and tolerability of Arikayce compared to a currently available antibiotic, TOBI (tobramycin inhalation solution). This study was for people with CF with chronic lung infections.

Age: 6 Years and Older

Mutation: No Mutation Requirement

Fev1% Predicted: 25% or greater

Number of Visits: 11

Length of Participation: 6 months

ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT01315678

August 27, 2019

Roll-over study of ivacaftor in children with CF who have a CFTR gating mutation

Status: Completed with results

Description: This study evaluated the long-term safety of ivacaftor (Kalydeco®) in children with CF. This study was for children who have a CFTR gating mutation and received at least one dose of ivacaftor in the VX11-770-108 study.

Age: 2 Years to 5 Years

Mutation: One Copy F508del or No Copies F508del

Fev1% Predicted: No FEV1 Limit

Number of Visits: 7

Length of Participation: 88 weeks

ClinicalTrials.gov link: https://www.clinicaltrials.gov/ct2/show/NCT01946412

Open-label study of ivacaftor in people with CF who have a non-G551D CFTR gating mutation

Status: Completed with results

Description: This study evaluated the long-term safety of ivacaftor (Kalydeco®) in participants who have a non-G551D CFTR gating mutation. This study was for people who had participated in one of the VX11-770-110, VX12-770-111, or VX12-770-113 studies.

Age: 6 Years and Older

Mutation: One Copy F508del or No Copies F508del

Fev1% Predicted: No FEV1 Limit

Number of Visits: 7

Length of Participation: 2 years

ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT01707290

August 28, 2019

Study of VX-445 triple combination in children 6-11 years old with cystic fibrosis

Status: Enrolling

Description: This study will look at the safety and effectiveness of three CFTR modulators when taken in combination: VX-445 (elexacaftor), tezacaftor, and ivacaftor. CFTR modulators are drugs intended to help CFTR protein function closer to normal.

Age: 6 Years to 11 Years

Mutation: Two Copies F508del or One Copy F508del

Fev1% Predicted: 40% or greater

Number of Visits: 9

Length of Participation: 32 weeks

ClinicalTrials.gov link: https://clinicaltrials.gov/ct2/show/NCT03691779


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