Cystic FibrosisPhase 1NCT03093714
A Study to Evaluate Safety, PK and PD of FDL169 in Cystic Fibrosis Subjects
A Phase 1 study testing FDL169 in people with Cystic Fibrosis, run by Flatley Discovery Lab LLC at 14 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- FDL169
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 27 participants
- Where
- 14 sitesin 4 countries
- Study dates
- 23 Aug 2017to 3 Apr 2018
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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).
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- 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.
Trial locations and contacts
14 sites in 4 countries.
14 sites in 4 countries
United Kingdom5 sites
NICRN Respiratory Research Office, Belfast City Hospital
Belfast, BT9 7AB
Research Dept., Liverpool Heart and Chest Hospital
Liverpool, L14 3PE
Royal Brompton Hospital
London, SW3 6NP
The Medicines Evaluation Unit (MEU)
Manchester, M23 9QZ
NIHR Wellcome Trust Clinical Research Facility
Southampton, SO16 6YD
Germany4 sites
Charité - Universitätsmedizin Berlin CVK
Berlin
Klinik Donaustauf, Zentrum für Pneumologie
Donaustauf, 93093
Ruhrlandklinik
Essen
Universitätsklinikum Frankfurt
Frankfurt
Australia3 sites
The Prince Charles Hospital
Chermside, Queensland, 4032
The Alfred Hospital
Melbourne, 3004
Mater Misericordiae Ltd
South Brisbane, Queenland, 4101
Czechia2 sites
FN v Motole, Pediatrická klinika, Centrum cystické fibrózy
Brno
FN v Motole, Pediatrická klinika, Centrum cystické fibrózy
Prague
About this study
Official 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
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.
Read the detailed description
This is a multicenter, randomized double-blind, placebo-controlled dose-escalation and parallel-arm, dose-ranging study. Enrollment is planned to occur at approximately 14 global sites. Approximately 24 subjects with CF who are homozygous for the F508del-CFTR mutation will be enrolled in two cohorts.
- Conditions
- Cystic Fibrosis
- Being tested
- FDL169, Placebo
- Keywords
- Cystic Fibrosis
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FDL169Drug
CFTR corrector
PlaceboDrug
Placebo for FDL169
| Study group | Role | What they receive |
|---|---|---|
| FDL 169 test formulation (Dose Level 1) | Experimental | Multiple dose (Dose Level 1) FDL 169 test formulation administered as repeat doses in CF subjects Receives: Drug: FDL169 |
| FDL 169 test formulation (Dose Level 2) | Experimental | Multiple dose (Dose Level 2) FDL 169 test formulation administered as repeat doses in CF subjects Receives: Drug: FDL169 |
| FDL 169 test formulation ( Dose Level 3) | Experimental | Multiple dose (Dose Level 3) FDL 169 test formulation administered as repeat doses in CF subjects Receives: Drug: FDL169 |
| Placebo | Placebo comparator | Multiple dose placebo as repeat doses in CF subjects Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 28 days
Secondary outcomes (5)
Pharmacokinetic parameters, Cmax
The pharmacokinetic parameters of FDL169: maximal plasma concentration (Cmax).
Measured: 28 days
Pharmacokinetic parameters, Tmax
The pharmacokinetic parameters of FDL169: maximal concentration (Tmax).
Measured: 28 days
Pharmacokinetic parameters, AUC
The pharmacokinetic parameters of FDL169: area under the plasma concentration curve (AUC).
Measured: 28 days
Pharmacokinetic parameters, CL/F
The pharmacokinetic parameters of FDL169: clearance (CL/F).
Measured: 28 days
Pharmacokinetic parameters, V/F
The pharmacokinetic parameters of FDL169: apparent volume of distribution (V/F).
Measured: 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Mar 2017
- Study start23 Aug 2017
- Primary completion3 Apr 2018
- Study completion3 Apr 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Flatley Discovery Lab LLC
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Claudia Ordonez, MD, Study chair, Flatley Discovery Lab
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03093714
- Sponsor's protocol ID
- FDL169-2015-04
- Status last verified
- Apr 2018
- First submitted
- 16 Mar 2017
- Last updated
- 12 Apr 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
PubMed 37983082Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
PubMed 33331662
Study at a glance
From the registry record, last updated by the sponsor on 12 Apr 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT03093714
- Acronym / sponsor ID
- FDL169-2015-04
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Flatley Discovery Lab LLCUniversity, hospital or other
- Study start
- 23 Aug 2017 (actual)
- Primary completion
- 3 Apr 2018 (actual)
- Study completion
- 3 Apr 2018 (actual)
- First posted
- 28 Mar 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a multicenter, randomized double-blind, placebo-controlled dose-escalation and parallel-arm, dose-ranging study. Enrollment is planned to occur at approximately 14 global sites. Approximately 24 subjects with CF who are homozygous for the F508del-CFTR mutation will be enrolled in two cohorts.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| FDL 169 test formulation (Dose Level 1) | Experimental | Drug: FDL169Multiple dose (Dose Level 1) FDL 169 test formulation administered as repeat doses in CF subjects |
| FDL 169 test formulation (Dose Level 2) | Experimental | Drug: FDL169Multiple dose (Dose Level 2) FDL 169 test formulation administered as repeat doses in CF subjects |
| FDL 169 test formulation ( Dose Level 3) | Experimental | Drug: FDL169Multiple dose (Dose Level 3) FDL 169 test formulation administered as repeat doses in CF subjects |
| Placebo | Placebo comparator | Drug: PlaceboMultiple dose placebo as repeat doses in CF subjects |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (13 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FDL169CFTR corrector | Drug | FDL 169 test formulation ( Dose Level 3), FDL 169 test formulation (Dose Level 1), FDL 169 test formulation (Dose Level 2) |
| PlaceboPlacebo for FDL169 | Drug | Placebo |
Primary outcome
Incidence of Treatment-Emergent Adverse Events
Time frame: 28 days
Safety and tolerability of FDL169 as determined by the incidence of adverse events (AEs) and serious adverse events (SAEs).
5 secondary outcomes
Pharmacokinetic parameters, Cmax
Time frame: 28 days
Pharmacokinetic parameters, Tmax
Time frame: 28 days
Pharmacokinetic parameters, AUC
Time frame: 28 days
Pharmacokinetic parameters, CL/F
Time frame: 28 days
Pharmacokinetic parameters, V/F
Time frame: 28 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Apr 2018.
- Derived from this study: PubMed 37983082Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
- Derived from this study: PubMed 33331662Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Claudia Ordonez, MD study chair
Flatley Discovery Lab
14 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing FDL169 in people with Cystic Fibrosis, run by Flatley Discovery Lab LLC at 14 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Apr 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United Kingdom, Germany, Australia, Czechia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events (time frame: 28 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03093714, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Apr 2018.