Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 8 for:    FDL169 | Cystic Fibrosis
Previous Study | Return to List | Next Study

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03093714
Recruitment Status : Completed
First Posted : March 28, 2017
Last Update Posted : April 12, 2018
Sponsor:
Information provided by (Responsible Party):
Flatley Discovery Lab LLC

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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: FDL169 Drug: Placebo Phase 1

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 27 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
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
Actual Study Start Date : August 23, 2017
Actual Primary Completion Date : April 3, 2018
Actual Study Completion Date : April 3, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: FDL 169 test formulation (Dose Level 1)
Multiple dose (Dose Level 1) FDL 169 test formulation administered as repeat doses in CF subjects
Drug: FDL169
CFTR corrector

Experimental: FDL 169 test formulation (Dose Level 2)
Multiple dose (Dose Level 2) FDL 169 test formulation administered as repeat doses in CF subjects
Drug: FDL169
CFTR corrector

Experimental: FDL 169 test formulation ( Dose Level 3)
Multiple dose (Dose Level 3) FDL 169 test formulation administered as repeat doses in CF subjects
Drug: FDL169
CFTR corrector

Placebo Comparator: Placebo
Multiple dose placebo as repeat doses in CF subjects
Drug: Placebo
Placebo for FDL169




Primary Outcome Measures :
  1. 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).


Secondary Outcome Measures :
  1. Pharmacokinetic parameters, Cmax [ Time Frame: 28 days ]
    The pharmacokinetic parameters of FDL169: maximal plasma concentration (Cmax).

  2. Pharmacokinetic parameters, Tmax [ Time Frame: 28 days ]
    The pharmacokinetic parameters of FDL169: maximal concentration (Tmax).

  3. Pharmacokinetic parameters, AUC [ Time Frame: 28 days ]
    The pharmacokinetic parameters of FDL169: area under the plasma concentration curve (AUC).

  4. Pharmacokinetic parameters, CL/F [ Time Frame: 28 days ]
    The pharmacokinetic parameters of FDL169: clearance (CL/F).

  5. Pharmacokinetic parameters, V/F [ Time Frame: 28 days ]
    The pharmacokinetic parameters of FDL169: apparent volume of distribution (V/F).



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03093714


Locations
Layout table for location information
Australia, Queenland
Mater Misericordiae Ltd
South Brisbane, Queenland, Australia, 4101
Australia, Queensland
The Prince Charles Hospital
Chermside, Queensland, Australia, 4032
Australia
The Alfred Hospital
Melbourne, Australia, 3004
Czechia
FN v Motole, Pediatrická klinika, Centrum cystické fibrózy
Brno, Czechia
FN v Motole, Pediatrická klinika, Centrum cystické fibrózy
Praha, Czechia
Germany
Charité - Universitätsmedizin Berlin CVK
Berlin, Germany
Klinik Donaustauf, Zentrum für Pneumologie
Donaustauf, Germany, 93093
Ruhrlandklinik
Essen, Germany
Universitätsklinikum Frankfurt
Frankfurt, Germany
United Kingdom
NICRN Respiratory Research Office, Belfast City Hospital
Belfast, United Kingdom, BT9 7AB
Research Dept., Liverpool Heart and Chest Hospital
Liverpool, United Kingdom, L14 3PE
Royal Brompton Hospital
London, United Kingdom, SW3 6NP
The Medicines Evaluation Unit (MEU)
Manchester, United Kingdom, M23 9QZ
NIHR Wellcome Trust Clinical Research Facility
Southampton, United Kingdom, SO16 6YD
Sponsors and Collaborators
Flatley Discovery Lab LLC
Investigators
Layout table for investigator information
Study Chair: Claudia Ordonez, MD Flatley Discovery Lab

Layout table for additonal information
Responsible Party: Flatley Discovery Lab LLC
ClinicalTrials.gov Identifier: NCT03093714     History of Changes
Other Study ID Numbers: FDL169-2015-04
First Posted: March 28, 2017    Key Record Dates
Last Update Posted: April 12, 2018
Last Verified: April 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Flatley Discovery Lab LLC:
Cystic Fibrosis
Additional relevant MeSH terms:
Layout table for MeSH terms
Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases