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

• Sponsor: Celtaxsys, Inc. (Other)
• Overall Status: Completed
• CT.gov ID: NCT01944735
• Collaborator: Celerion (Industry)
• Enrollment: 17
• Locations: 4
• Arms: 2
• Duration (Months): 14
• Patients Per Site: 4.3
• Patients Per Site Per Month: 0.3

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis	Drug: CTX-4430; Drug: Placebo	Phase 1

Study Design

• Study Type: Interventional
• Actual Enrollment: 17 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, 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
• Study Start Date: Sep 1, 2013
• Actual Primary Completion Date: Oct 1, 2014
• Actual Study Completion Date: Nov 1, 2014

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Active Once daily oral capsule containing 50 or 100 mg of CTX-4430	Drug: CTX-4430; Ascending repeat doses of CTX-4430 will be administered orally, once-daily, to patients in 2 cohorts on days 1-15.
Placebo Comparator: Placebo Once daily oral capsule containing mannitol, visibly identical to CTX-4430 capsules	Drug: Placebo; Repeat doses of placebo will be administered orally, once-daily, to patients in 2 cohorts on days 1-15; Other Names: Mannitol

Outcome Measures

Primary Outcome Measures

1. Assessment of the safety and tolerability of multiple oral doses of CTX-4430 when administered to cystic fibrosis (CF) patients once-daily (QD) [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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 55 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Locations

	Site	City	State	Country	Postal Code
1	The Medicinces Evaluation Unit	Wythenshawe	Manchester	United Kingdom	M23 9QZ
2	Celerion	Belfast	Northern Ireland	United Kingdom	BT9 6AD
3	Glasgow Clinical Research Facility	Glasgow		United Kingdom	G31 2ER
4	Royal Brompton Hospital	London		United Kingdom	SW3 6NP

Sponsors and Collaborators

• Celtaxsys, Inc.
• Celerion

Investigators

• Principal Investigator: J Stuart Elborn, MD, FRCP, Queen's University, Belfast

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Celtaxsys, Inc.
• ClinicalTrials.gov Identifier: NCT01944735
• Other Study ID Numbers: CTX-4430-CF-001
• First Posted: Sep 18, 2013
• Last Update Posted: Mar 6, 2015
• Last Verified: Mar 1, 2015

• Additional relevant MeSH terms: Cystic Fibrosis; Fibrosis; Mannitol

Study Results

No Results Posted as of Mar 6, 2015