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

Primary Purpose

Cystic Fibrosis

Status

Completed

Phase

Phase 1

Locations

United Kingdom

Study Type

Interventional

Intervention

CTX-4430

Placebo

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

18 Years - 55 Years (Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Adult male or female, 18 to 55 years of age (inclusive) at the time of screening.
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.
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.
Body mass index (BMI) ≥ 17.0 (kg/m2).
Non-tobacco/nicotine-containing product user for a minimum of 6 months prior to the first study drug administration.
Forced Expiratory Volume (FEV1) of >50% and <90% of predicted at screening.
Must agree to use adequate method of contraception.

Exclusion Criteria:

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.
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.
Has a history of lung transplantation.
History or presence of alcoholism or drug abuse within 2 years prior to the first study drug administration.
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.
Sitting blood pressure is less than 90/40 mmHg or greater than 140/90 mmHg, unless deemed clinically insignificant by the PI.
Pulse is higher than 100 b.p.m or lower than 50 b.p.m. unless deemed clinically insignificant by the PI.
Failure to satisfy the PI of fitness to participate for any other reason.
In the judgment of the PI, clinically significant hemoptysis (>30 cc per episode) within the last 180 days.

Sites / Locations

The Medicinces Evaluation Unit
Celerion
Glasgow Clinical Research Facility
Royal Brompton Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Active

Placebo

Arm Description

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

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

Outcomes

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)

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.

Secondary Outcome Measures

Full Information

NCT ID

NCT01944735

First Posted

September 6, 2013

Last Updated

March 4, 2015

Sponsor

Celtaxsys, Inc.

Collaborators

Celerion

1. Study Identification

Unique Protocol Identification Number

NCT01944735

Brief Title

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

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 Type

Interventional

2. Study Status

Record Verification Date

March 2015

Overall Recruitment Status

Completed

Study Start Date

September 2013 (undefined)

Primary Completion Date

October 2014 (Actual)

Study Completion Date

November 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Celtaxsys, Inc.

Collaborators

Celerion

4. Oversight

Data Monitoring Committee

Yes

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cystic Fibrosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

17 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Active

Arm Type

Experimental

Arm Description

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

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

CTX-4430

Intervention Description

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

Intervention Type

Drug

Intervention Name(s)

Placebo

Other Intervention Name(s)

Mannitol

Intervention Description

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

Primary Outcome Measure Information:

Title

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

Description

Time Frame

15 days

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

55 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Adult male or female, 18 to 55 years of age (inclusive) at the time of screening. 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. 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. Body mass index (BMI) ≥ 17.0 (kg/m2). Non-tobacco/nicotine-containing product user for a minimum of 6 months prior to the first study drug administration. Forced Expiratory Volume (FEV1) of >50% and <90% of predicted at screening. Must agree to use adequate method of contraception. Exclusion Criteria: 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. 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. Has a history of lung transplantation. History or presence of alcoholism or drug abuse within 2 years prior to the first study drug administration. 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. Sitting blood pressure is less than 90/40 mmHg or greater than 140/90 mmHg, unless deemed clinically insignificant by the PI. Pulse is higher than 100 b.p.m or lower than 50 b.p.m. unless deemed clinically insignificant by the PI. Failure to satisfy the PI of fitness to participate for any other reason. In the judgment of the PI, clinically significant hemoptysis (>30 cc per episode) within the last 180 days.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

J Stuart Elborn, MD, FRCP

Organizational Affiliation

Queen's University, Belfast

Official's Role

Principal Investigator

Facility Information:

Facility Name

The Medicinces Evaluation Unit

City

Wythenshawe

State/Province

Manchester

ZIP/Postal Code

M23 9QZ

Country

United Kingdom

Facility Name

Celerion

City

Belfast

State/Province

Northern Ireland

ZIP/Postal Code

BT9 6AD

Country

United Kingdom

Facility Name

Glasgow Clinical Research Facility

City

Glasgow

ZIP/Postal Code

G31 2ER

Country

United Kingdom

Facility Name

Royal Brompton Hospital

City

London

ZIP/Postal Code

SW3 6NP

Country

United Kingdom

12. IPD Sharing Statement

Learn more about this trial

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

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