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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03093714




Registration number
NCT03093714
Ethics application status
Date submitted
16/03/2017
Date registered
28/03/2017
Date last updated
12/04/2018

Titles & IDs
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
Secondary ID [1] 0 0
FDL169-2015-04
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - FDL169
Treatment: Drugs - Placebo

Experimental: FDL 169 test formulation (Dose Level 1) - Multiple dose (Dose Level 1) FDL 169 test formulation administered as repeat doses in CF subjects

Experimental: FDL 169 test formulation (Dose Level 2) - Multiple dose (Dose Level 2) FDL 169 test formulation administered as repeat doses in CF subjects

Experimental: FDL 169 test formulation ( Dose Level 3) - Multiple dose (Dose Level 3) FDL 169 test formulation administered as repeat doses in CF subjects

Placebo Comparator: Placebo - Multiple dose placebo as repeat doses in CF subjects


Treatment: Drugs: FDL169
CFTR corrector

Treatment: Drugs: Placebo
Placebo for FDL169

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of Treatment-Emergent Adverse Events - Safety and tolerability of FDL169 as determined by the incidence of adverse events (AEs) and serious adverse events (SAEs).
Timepoint [1] 0 0
28 days
Secondary outcome [1] 0 0
Pharmacokinetic parameters, Cmax - The pharmacokinetic parameters of FDL169: maximal plasma concentration (Cmax).
Timepoint [1] 0 0
28 days
Secondary outcome [2] 0 0
Pharmacokinetic parameters, Tmax - The pharmacokinetic parameters of FDL169: maximal concentration (Tmax).
Timepoint [2] 0 0
28 days
Secondary outcome [3] 0 0
Pharmacokinetic parameters, AUC - The pharmacokinetic parameters of FDL169: area under the plasma concentration curve (AUC).
Timepoint [3] 0 0
28 days
Secondary outcome [4] 0 0
Pharmacokinetic parameters, CL/F - The pharmacokinetic parameters of FDL169: clearance (CL/F).
Timepoint [4] 0 0
28 days
Secondary outcome [5] 0 0
Pharmacokinetic parameters, V/F - The pharmacokinetic parameters of FDL169: apparent volume of distribution (V/F).
Timepoint [5] 0 0
28 days

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
85 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QueenlandQLD
Recruitment hospital [1] 0 0
Mater Misericordiae Ltd - South Brisbane
Recruitment hospital [2] 0 0
The Prince Charles Hospital - Chermside
Recruitment hospital [3] 0 0
The Alfred Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
4101 - South Brisbane
Recruitment postcode(s) [2] 0 0
4032 - Chermside
Recruitment postcode(s) [3] 0 0
3004 - Melbourne
Recruitment outside Australia
Country [1] 0 0
Czechia
State/province [1] 0 0
Brno
Country [2] 0 0
Czechia
State/province [2] 0 0
Praha
Country [3] 0 0
Germany
State/province [3] 0 0
Berlin
Country [4] 0 0
Germany
State/province [4] 0 0
Donaustauf
Country [5] 0 0
Germany
State/province [5] 0 0
Essen
Country [6] 0 0
Germany
State/province [6] 0 0
Frankfurt
Country [7] 0 0
United Kingdom
State/province [7] 0 0
Belfast
Country [8] 0 0
United Kingdom
State/province [8] 0 0
Liverpool
Country [9] 0 0
United Kingdom
State/province [9] 0 0
London
Country [10] 0 0
United Kingdom
State/province [10] 0 0
Manchester
Country [11] 0 0
United Kingdom
State/province [11] 0 0
Southampton

Funding & Sponsors
Primary sponsor type
Other
Name
Flatley Discovery Lab LLC
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This is a multicenter, randomized, placebo-controlled, dose-escalation study. Enrollment is
planned to occur at approximately 14 global sites. Approximately 24 subjects with CF.
Trial website
https://clinicaltrials.gov/show/NCT03093714
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Claudia Ordonez, MD
Address 0 0
Flatley Discovery Lab
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications