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

NCT ID: NCT01944735

Last Updated: 2015-03-06

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 17 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-09-30
• Study Completion Date: 2014-11-30

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.

Conditions

Cystic Fibrosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Active

Once daily oral capsule containing 50 or 100 mg of CTX-4430

Group Type: EXPERIMENTAL

CTX-4430

Intervention Type: DRUG

Ascending repeat doses of CTX-4430 will be administered orally, once-daily, to patients in 2 cohorts on days 1-15.

Placebo

Once daily oral capsule containing mannitol, visibly identical to CTX-4430 capsules

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Repeat doses of placebo will be administered orally, once-daily, to patients in 2 cohorts on days 1-15

Interventions

CTX-4430

Ascending repeat doses of CTX-4430 will be administered orally, once-daily, to patients in 2 cohorts on days 1-15.

Intervention Type: DRUG

Placebo

Repeat doses of placebo will be administered orally, once-daily, to patients in 2 cohorts on days 1-15

Intervention Type: DRUG

Other Intervention Names

Mannitol

Eligibility 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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 55 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Celerion

INDUSTRY

Sponsor Role: collaborator

Celtaxsys, Inc.

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

J Stuart Elborn, MD, FRCP

Role: PRINCIPAL_INVESTIGATOR

Queen's University, Belfast

Locations

The Medicinces Evaluation Unit

Wythenshawe, Manchester, United Kingdom

Celerion

Belfast, Northern Ireland, United Kingdom

Glasgow Clinical Research Facility

Glasgow, , United Kingdom

Royal Brompton Hospital

London, , United Kingdom

Countries

United Kingdom

Other Identifiers

CTX-4430-CF-001

Identifier Type: -

Identifier Source: org_study_id