NCT03093714

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 Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
27

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Aug 2017

Shorter than P25 for phase_1

Geographic Reach
4 countries

14 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 16, 2017

Completed
12 days until next milestone

First Posted

Study publicly available on registry

March 28, 2017

Completed
5 months until next milestone

Study Start

First participant enrolled

August 23, 2017

Completed
7 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 3, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 3, 2018

Completed
Last Updated

April 12, 2018

Status Verified

April 1, 2018

Enrollment Period

7 months

First QC Date

March 16, 2017

Last Update Submit

April 11, 2018

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

  • 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).

    28 days

Secondary Outcomes (5)

  • Pharmacokinetic parameters, Cmax

    28 days

  • Pharmacokinetic parameters, Tmax

    28 days

  • Pharmacokinetic parameters, AUC

    28 days

  • Pharmacokinetic parameters, CL/F

    28 days

  • Pharmacokinetic parameters, V/F

    28 days

Study Arms (4)

FDL 169 test formulation (Dose Level 1)

EXPERIMENTAL

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

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

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

Drug: FDL169

Placebo

PLACEBO COMPARATOR

Multiple dose placebo as repeat doses in CF subjects

Drug: Placebo

Interventions

FDL169DRUG

CFTR corrector

FDL 169 test formulation ( Dose Level 3)FDL 169 test formulation (Dose Level 1)FDL 169 test formulation (Dose Level 2)
PlaceboDRUG

Placebo for FDL169

Placebo

Eligibility Criteria

Age18 Years - 85 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (14)

Mater Misericordiae Ltd

South Brisbane, Queenland, 4101, Australia

Location

The Prince Charles Hospital

Chermside, Queensland, 4032, Australia

Location

The Alfred Hospital

Melbourne, 3004, Australia

Location

FN v Motole, Pediatrická klinika, Centrum cystické fibrózy

Brno, Czechia

Location

FN v Motole, Pediatrická klinika, Centrum cystické fibrózy

Prague, Czechia

Location

Charité - Universitätsmedizin Berlin CVK

Berlin, Germany

Location

Klinik Donaustauf, Zentrum für Pneumologie

Donaustauf, 93093, Germany

Location

Ruhrlandklinik

Essen, Germany

Location

Universitätsklinikum Frankfurt

Frankfurt, Germany

Location

NICRN Respiratory Research Office, Belfast City Hospital

Belfast, BT9 7AB, United Kingdom

Location

Research Dept., Liverpool Heart and Chest Hospital

Liverpool, L14 3PE, United Kingdom

Location

Royal Brompton Hospital

London, SW3 6NP, United Kingdom

Location

The Medicines Evaluation Unit (MEU)

Manchester, M23 9QZ, United Kingdom

Location

NIHR Wellcome Trust Clinical Research Facility

Southampton, SO16 6YD, United Kingdom

Location

Related Publications (2)

  • 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.

    PMID: 37983082DERIVED

  • 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. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.

    PMID: 33331662DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Study Officials

  • Claudia Ordonez, MD

    Flatley Discovery Lab

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 16, 2017

First Posted

March 28, 2017

Study Start

August 23, 2017

Primary Completion

April 3, 2018

Study Completion

April 3, 2018

Last Updated

April 12, 2018

Record last verified: 2018-04

Locations

AU(3)CZ(2)GB(5)DE(4)