Phase 1 Study Assessing the Safety and Tolerability of CTX-4430 in Cystic Fibrosis Patients

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2014 by Celtaxsys, Inc.
Sponsor:
Collaborators:
Celerion
Thermo Fisher Scientific
Appledown Clinical Research Limited
Information provided by (Responsible Party):
Celtaxsys, Inc.
ClinicalTrials.gov Identifier:
NCT01944735
First received: September 6, 2013
Last updated: March 26, 2014
Last verified: March 2014
  Purpose

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 four 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.


Condition Intervention Phase
Cystic Fibrosis
Drug: CTX-4430
Drug: Mannitol
Drug: Placebo
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Celtaxsys, Inc.:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]
    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.


Estimated Enrollment: 36
Study Start Date: September 2013
Estimated Primary Completion Date: September 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: CTX-4430 Drug: CTX-4430
Ascending repeat doses of CTX-4430 will be administered orally, once-daily, to patients in 4 cohorts on days 1-15.
Drug: Mannitol
excipient in capsules
Other Name: osmitrol
Placebo Comparator: Placebo + Mannitol Drug: Mannitol
excipient in capsules
Other Name: osmitrol
Drug: Placebo
Repeat doses of placebo will be administered orally, once-daily, to patients in 4 cohorts on days 1-15

  Eligibility

Ages Eligible for Study:   18 Years to 55 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01944735

Contacts
Contact: Kirsten Gill +44 (0)28 9055 4049 kirsten.gill@celerion.com

Locations
United Kingdom
The Medicinces Evaluation Unit Recruiting
Wythenshawe, Manchester, United Kingdom, M23 9QZ
Contact: Alexander Horsley, MA MBChB MRCP PhD    44 (0) 161 2912046    alexander.horsley@manchester.ac.uk   
Celerion Recruiting
Belfast, Northern Ireland, United Kingdom, BT9 6AD
Contact: Johnston Stewart, MB, BSc, DRCOG, MFPM    +44 (0) 28 90 554040 ext 4014    johnston.stewart@celerion.com   
Royal Brompton Hospital Recruiting
London, United Kingdom, SW3 6NP
Contact: Diana Bilton, MD    44 (0)20 7351 8182    d.bilton@rbht.nhs.uk   
Sponsors and Collaborators
Celtaxsys, Inc.
Celerion
Thermo Fisher Scientific
Appledown Clinical Research Limited
Investigators
Principal Investigator: J Stuart Elborn, MD, FRCP Queen's University, Belfast
  More Information

No publications provided

Responsible Party: Celtaxsys, Inc.
ClinicalTrials.gov Identifier: NCT01944735     History of Changes
Other Study ID Numbers: CTX-4430-CF-001
Study First Received: September 6, 2013
Last Updated: March 26, 2014
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency
Ireland: Research Ethics Committee

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Mannitol
Diuretics, Osmotic
Diuretics
Natriuretic Agents
Physiological Effects of Drugs
Pharmacologic Actions
Cardiovascular Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on September 18, 2014