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

NCT01944735 | Completed Phase 1 DMC

• 1 other identifier: CTX-4430-CF-001
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 4

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

Outcome Measures

Primary Outcomes (1)

• 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.

  15 days

Study Arms (2)

Active

EXPERIMENTAL

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

Drug: CTX-4430

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

CTX-4430 DRUG

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

Active

Placebo DRUG

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

Also known as: Mannitol

Placebo

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Celtaxsys, Inc.: lead
• Celerion: collaborator

Study Sites (4)

The Medicinces Evaluation Unit

Wythenshawe, Manchester, M23 9QZ, United Kingdom

Location

Celerion

Belfast, Northern Ireland, BT9 6AD, United Kingdom

Location

Glasgow Clinical Research Facility

Glasgow, G31 2ER, United Kingdom

Location

Royal Brompton Hospital

London, SW3 6NP, United Kingdom

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

acebilustat; Mannitol

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Sugar Alcohols; Alcohols; Organic Chemicals; Carbohydrates

Study Officials

• J Stuart Elborn, MD, FRCP

  Queen's University, Belfast

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 6, 2013
• First Posted: September 18, 2013
• Study Start: September 1, 2013
• Primary Completion: October 1, 2014
• Study Completion: November 1, 2014
• Last Updated: March 6, 2015

Record last verified: 2015-03

Locations

GB (4)