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Trial record 1 of 2 for:    CTX-4430 | Cystic Fibrosis
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Phase 1 Study Assessing the Safety and Tolerability of CTX-4430 in Cystic Fibrosis Patients

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ClinicalTrials.gov Identifier: NCT01944735
Recruitment Status : Completed
First Posted : September 18, 2013
Last Update Posted : March 6, 2015
Sponsor:
Collaborator:
Celerion
Information provided by (Responsible Party):
Celtaxsys, Inc.

Tracking Information
First Submitted Date  ICMJE September 6, 2013
First Posted Date  ICMJE September 18, 2013
Last Update Posted Date March 6, 2015
Study Start Date  ICMJE September 2013
Actual Primary Completion Date October 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 13, 2013)
Assessment of the safety and tolerability of multiple oral doses of CTX-4430 when administered to cystic fibrosis (CF) patients once-daily (QD) [ Time Frame: 15 days ]
Changes in clinical signs and symptoms of safety data that include physical examinations, ECGs, vital signs, pulse oximetry, pulmonary function (spirometry), clinical laboratory results, and adverse events which will be summarized descriptively and reported in tabular form.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phase 1 Study Assessing the Safety and Tolerability of CTX-4430 in Cystic Fibrosis Patients
Official Title  ICMJE A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Ascending Multiple-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of CTX-4430 When Administered Orally to Cystic Fibrosis Patients for Fifteen Days
Brief Summary The purpose of this study is to assess the safety and tolerability of multiple oral doses of CTX-4430 when administered to cystic fibrosis (CF) patients once-daily (QD) for 15 days.This study will include two dose levels. For each dose level, blood samples will be collected for exploratory PK and PD assay validation. In addition, sputum will be collected for exploratory biomarker analysis. Following multiple dose administration, pulmonary function and exploratory lung clearance index (LCI) measurements will be taken.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE
  • Drug: CTX-4430
    Ascending repeat doses of CTX-4430 will be administered orally, once-daily, to patients in 2 cohorts on days 1-15.
  • Drug: Placebo
    Repeat doses of placebo will be administered orally, once-daily, to patients in 2 cohorts on days 1-15
    Other Name: Mannitol
Study Arms  ICMJE
  • Experimental: Active
    Once daily oral capsule containing 50 or 100 mg of CTX-4430
    Intervention: Drug: CTX-4430
  • Placebo Comparator: Placebo
    Once daily oral capsule containing mannitol, visibly identical to CTX-4430 capsules
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 17, 2015)
17
Original Estimated Enrollment  ICMJE
 (submitted: September 13, 2013)
36
Actual Study Completion Date  ICMJE November 2014
Actual Primary Completion Date October 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Adult male or female, 18 to 55 years of age (inclusive) at the time of screening.
  2. Confirmed diagnosis of CF based on the following: positive sweat chloride or sodium value ≥ 60 mEq/L, and/or genotype with two identifiable mutations consistent with CF, accompanied by one or more clinical features consistent with the CF phenotype or a diagnosis of CF made by a specialist in this condition.
  3. In the judgment of the Principal Investigator (PI), the patient is medically stable with no change in symptoms, medication, or with clinical laboratory results that in PI opinion are compatible with the diagnosis of either CF or a complication thereof and are judged acceptable for inclusion.
  4. Body mass index (BMI) ≥ 17.0 (kg/m2).
  5. Non-tobacco/nicotine-containing product user for a minimum of 6 months prior to the first study drug administration.
  6. Forced Expiratory Volume (FEV1) of >50% and <90% of predicted at screening.
  7. Must agree to use adequate method of contraception.

Exclusion Criteria:

  1. Patient has displayed any significant clinical/laboratory/radiological/spirometric sign of unstable or unexpectedly deteriorating respiratory disease within 30 days prior to the first study drug administration.
  2. History or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematological, gastrointestinal, endocrine, immunologic, dermatologic, neurological, or psychiatric disease that depart from the patient's usual baseline level of health as a patient with CF.
  3. Has a history of lung transplantation.
  4. History or presence of alcoholism or drug abuse within 2 years prior to the first study drug administration.
  5. Personal or family history of prolonged QT syndrome; or a QTc interval >430 msec (males) or >450 msec (females) using Bazett's formula (QTcB) or deemed clinically significant by the PI.
  6. Sitting blood pressure is less than 90/40 mmHg or greater than 140/90 mmHg, unless deemed clinically insignificant by the PI.
  7. Pulse is higher than 100 b.p.m or lower than 50 b.p.m. unless deemed clinically insignificant by the PI.
  8. Failure to satisfy the PI of fitness to participate for any other reason.
  9. In the judgment of the PI, clinically significant hemoptysis (>30 cc per episode) within the last 180 days.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 55 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01944735
Other Study ID Numbers  ICMJE CTX-4430-CF-001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Celtaxsys, Inc.
Study Sponsor  ICMJE Celtaxsys, Inc.
Collaborators  ICMJE Celerion
Investigators  ICMJE
Principal Investigator: J Stuart Elborn, MD, FRCP Queen's University, Belfast
PRS Account Celtaxsys, Inc.
Verification Date March 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP