Healthy Volunteer Solid Oral Dose and Multiple Ascending Dose Evaluation of CTP-656

Study Overview
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Brief Summary
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Two-part study to assess CTP-656 dosed as a solid oral dosage form versus Kalydeco and multiple-ascending doses of CTP-656 dosed for 7 days.

Detailed Description
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This two-part study will assess in healthy male and female subjects a solid oral dose formulation of CTP-656 vs. Kalydeco® and the safety, tolerability and pharmacokinetic profiles of escalating CTP-656 solid oral doses following 7 days of dosing. In Part B, three doses of CTP-656 ranging from 75 mg up to 300 mg per day will be studied.

Official Title
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A Two-Part Phase 1 Healthy Volunteer Study: A Crossover Comparison of CTP-656 Solid Dose Formulation vs. Kalydeco® and a Double-Blind, Placebo-Controlled, Ascending Multiple Dose Evaluation of CTP-656

Conditions
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Healthy

Intervention / Treatment
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* Drug: CTP-656
* Drug: Placebo for CTP-656
* Drug: Kalydeco


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Healthy adults between 18 and 50 years of age, inclusive Body weight ≥ 50 kg and BMI within the range of 18 to 30 kg/m2, inclusive, at screening Exclusion Criteria: History of clinically significant central nervous system (eg, seizures), cardiac, pulmonary, metabolic, renal (including nephrolithiasis), hepatic, including history of Gilbert's syndrome or gastrointestinal (GI) conditions PR interval ≥ 220 msec or QRS duration ≥ 120 msec or QTcF interval > 450 msec obtained at screening visit or prior to the first dose of study drug Liver function tests greater than the upper limit of normal. Positive blood screen for human immunodeficiency virus (HIV antibody), hepatitis B virus surface antigen, or hepatitis C virus antibody at screening Urinalysis positive for greater than trace blood, protein or glucose A positive screen for alcohol, drugs of abuse, or tobacco use. Inability to comply with food and beverage restrictions during study participation. Donation or blood collection or acute loss of blood prior to screening.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 50 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Basic Science
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: CTP-656, 150 mg<br>Single Oral Dose | Drug: CTP-656<br> <br> |
| Active Comparator: Kalydeco, 150 mg<br>Single oral dose | Drug: Kalydeco<br> <br> |
| Experimental: CTP-656, 75 mg or matching placebo<br>Subjects will be administered 75 mg CTP-656 for 7 days. | Drug: CTP-656<br> <br> Drug: Placebo for CTP-656<br> <br> |
| Experimental: CTP-656, 150 mg or matching placebo<br>Subjects will be administered 150 mg CTP-656 for 7 days. | Drug: CTP-656<br> <br> Drug: Placebo for CTP-656<br> <br> |
| Experimental: CTP-656, high dose or matching placebo<br>Subjects will be administered up to 300 mg CTP-656 for 7 days. | Drug: CTP-656<br> <br> Drug: Placebo for CTP-656<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants with Adverse Events as a Measure of Safety and Tolerability | adverse events categorized by body system and MedDRA term | 7 days | 
| Measure exposure of test articles using area under the concentration time curve (AUC) |  | 96 hours | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Safety and Pharmacokinetics