A Study to Evaluate Safety, PK and PD of FDL169 in Cystic Fibrosis Subjects

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03093714
• Date of registration: 16/03/2017
• Prospective Registration: Yes
• Primary sponsor: Flatley Discovery Lab LLC
• Public title: A Study to Evaluate Safety, PK and PD of FDL169 in Cystic Fibrosis Subjects
• Scientific title: A Randomized, Double-Blind, Placebo-Controlled, Parallel Study to Evaluate Safety, Pharmacokinetics (PK) and Pharmacodynamics(PD) of FDL169 in Cystic Fibrosis (CF) Subjects Homozygous for the F508del-CFTR Mutation
• Date of first enrolment: August 23, 2017
• Target sample size: 27
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03093714
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 1

Countries of recruitment

Australia, Czechia, Germany, United Kingdom

Contacts

• Name: Claudia Ordonez, MD
• Affiliation: Flatley Discovery Lab

Key inclusion & exclusion criteria

Inclusion Criteria:

- Male or female subjects with a confirmed diagnosis of CF defined as a sweat chloride
value =60 mmol/L by quantitative pilocarpine iontophoresis or two CF-causing
mutations,documented in the subject's medical record or confirmed at screening.

- Age 18 and above on the date of informed consent.

- Weight =40 kg.

- Homozygous for the F508del-CFTR mutation. Genotyping to be confirmed at screening.

- Ability to perform a valid, reproducible spirometry test with demonstration of a
forced expiratory volume in 1 sec (FEV1) >40% of predicted normal for age, sex and
height.

- Screening laboratory tests with no clinically significant abnormalities that would
interfere with the study assessments (as judged by the Investigator).

- Subjects who are sexually active must agree to follow the study's contraception
requirements.

Exclusion Criteria:

- An acute upper or lower respiratory tract infection, pulmonary exacerbation, or
changes in therapy for pulmonary disease within 4 weeks prior to Day 1.

- Major complications of lung disease (including massive hemoptysis, pneumothorax, or
pleural effusion) within 8 weeks prior to screening.

- Impaired renal function or known portal hypertension.

- History of prolonged QT and/or QTcF (Fridericia's correction) interval (>450 msec) or
QTcF >450 msec at Screening.

- History of solid organ or hematological transplantation.

- History of alcohol abuse or drug addiction (including cannabis, cocaine and opiates)
during the past year, (as judged by the Investigator).

- Use of ivacaftor or lumacaftor, within 4 weeks of Day 1

- Any change (initiation, change in type of drug, dose modification, schedule
modification, interruption, discontinuation, or re-initiation) in a chronic
treatment/prophylaxis regimen for CF or for CF-related conditions within 4 weeks prior
to Day 1.

- Ongoing immunosuppressive therapy (including systemic corticosteroids).

- Hemoglobin <10 g/dL.

- Abnormal liver function, at screening.

- Abnormal renal function at screening.

- Ongoing participation in another clinical study or prior participation without
appropriate washout (minimum of 10 half- lives or 30 days, whichever is longer) prior
to Screening visit.



Age minimum: 18 Years
Age maximum: 85 Years
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: FDL169

Drug: Placebo

Primary Outcome(s)

Incidence of Treatment-Emergent Adverse Events [Time Frame: 28 days]

Secondary Outcome(s)

Pharmacokinetic parameters, CL/F [Time Frame: 28 days]

Pharmacokinetic parameters, V/F [Time Frame: 28 days]

Pharmacokinetic parameters, AUC [Time Frame: 28 days]

Pharmacokinetic parameters, Tmax [Time Frame: 28 days]

Pharmacokinetic parameters, Cmax [Time Frame: 28 days]

Secondary ID(s)

FDL169-2015-04

Source(s) of Monetary Support

Please refer to primary and secondary sponsors