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

• Register: ClinicalTrials.gov
• Last refreshed on: 16 March 2015
• Main ID: NCT01944735
• Date of registration: 06/09/2013
• Prospective Registration: No
• Primary sponsor: Celtaxsys, Inc.
• Public title: Phase 1 Study Assessing the Safety and Tolerability of CTX-4430 in Cystic Fibrosis Patients
• Scientific 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
• Date of first enrolment: September 2013
• Target sample size: 17
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT01944735
• 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
• Phase: Phase 1

Countries of recruitment

United Kingdom

Contacts

• Name: J Stuart Elborn, MD, FRCP
• Affiliation: Queen's University, Belfast

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: 55 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Placebo

Drug: CTX-4430

Primary Outcome(s)

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]

Secondary ID(s)

CTX-4430-CF-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Celerion