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Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis on KALYDECO® as Background Therapy

Primary Purpose

Cystic Fibrosis

Status

Completed

Phase

Phase 1

Locations

United Kingdom

Study Type

Interventional

Intervention

PTI-428

Placebo

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Confirmed diagnosis of CF.
Stable on Kalydeco dosing for both label indication and per label dosing for a minimum of 3 months at the time of randomization
Forced expiratory volume in 1 second (FEV1) 40-90% predicted.
Non-smoker and non-tobacco user for a minimum of 30 days prior to screening and for the duration of the study.

Exclusion Criteria:

Participation in another clinical trial or treatment with an investigational agent within 30 days or 5 half-lives, whichever is longer, prior to Study Day 1.
History of cancer within the past five years (excluding cervical CIS with curative therapy for at least one year prior to screening and non-melanoma skin cancer).
History of organ transplantation.
Any sinopulmonary infection or CF exacerbation requiring a change or addition of medication (including antibiotics) within 1 month of Study Day 1 or any other clinically significant infection as determined by the investigator within 1 month of Day 1.
History of alcohol or drug abuse or dependence within 12 months of screening as determined by the Investigator.
Male and female of child-bearing potential, unless they are using highly effective methods of contraception during participation in the clinical study and for 4 weeks after termination from study.
Pregnant or nursing women.

Sites / Locations

Celerion
Medicines Evaluation Unit

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

PTI-428

Placebo

Arm Description

Subjects will receive once daily dosing of PTI-428 or placebo for 14 days.

Outcomes

Primary Outcome Measures

Safety and tolerability as assessed by adverse events, safety labs, electrocardiograms (ECGs), physical examinations, and vital signs

Secondary Outcome Measures

t1/2 of multiple oral doses

Tmax of multiple oral doses

Cmax of multiple oral doses

AUC0-t of multiple oral doses

Full Information

NCT ID

NCT03258424

First Posted

August 18, 2017

Last Updated

March 24, 2020

Sponsor

Proteostasis Therapeutics, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT03258424

Brief Title

Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis on KALYDECO® as Background Therapy

Official Title

A Phase I, Randomized, Placebo-Controlled, Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects With Cystic Fibrosis.

Study Type

Interventional

2. Study Status

Record Verification Date

March 2020

Overall Recruitment Status

Completed

Study Start Date

July 28, 2017 (Actual)

Primary Completion Date

October 3, 2018 (Actual)

Study Completion Date

October 3, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Proteostasis Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

The trial will consist of a single treatment group enrolling adult subjects with CF on background therapy with KALYDECO®. Approximately 16 subjects will be enrolled.

Detailed Description

The single treatment group will enroll adult subjects with CF currently on stable KALYDECO® background therapy for a minimum of 3 months at the time of randomization. Subjects will be randomized to either PTI-428 or placebo. Each dose will be administered once daily (QD) for a total of 14 Days. The subjects will continue treatment with KALYDECO® throughout the study.

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

15 (Actual)

8. Arms, Groups, and Interventions

Arm Title

PTI-428

Arm Type

Active Comparator

Arm Description

Subjects will receive once daily dosing of PTI-428 or placebo for 14 days.

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Subjects will receive once daily dosing of PTI-428 or placebo for 14 days.

Intervention Type

Drug

Intervention Name(s)

PTI-428

Intervention Description

PTI-428 or placebo will be given QD for 14 days.

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

PTI-428 or placebo will be given QD for 14 days.

Primary Outcome Measure Information:

Title

Safety and tolerability as assessed by adverse events, safety labs, electrocardiograms (ECGs), physical examinations, and vital signs

Time Frame

Baseline through Day 21

Secondary Outcome Measure Information:

Title

t1/2 of multiple oral doses

Time Frame

Baseline through Day 14

Title

Tmax of multiple oral doses

Time Frame

Baseline through Day 14

Title

Cmax of multiple oral doses

Time Frame

Baseline through Day 14

Title

AUC0-t of multiple oral doses

Time Frame

Baseline through Day 14

Other Pre-specified Outcome Measures:

Title

Change in nasal epithelial mRNA and protein expression over time

Time Frame

Baseline through Day 21

Title

Change in sweat chloride over time

Time Frame

Baseline through Day 21

Title

Change in FEV1 over time

Time Frame

Baseline through Day 21

Title

Change in weight over time

Time Frame

Baseline through Day 21

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Confirmed diagnosis of CF. Stable on Kalydeco dosing for both label indication and per label dosing for a minimum of 3 months at the time of randomization Forced expiratory volume in 1 second (FEV1) 40-90% predicted. Non-smoker and non-tobacco user for a minimum of 30 days prior to screening and for the duration of the study. Exclusion Criteria: Participation in another clinical trial or treatment with an investigational agent within 30 days or 5 half-lives, whichever is longer, prior to Study Day 1. History of cancer within the past five years (excluding cervical CIS with curative therapy for at least one year prior to screening and non-melanoma skin cancer). History of organ transplantation. Any sinopulmonary infection or CF exacerbation requiring a change or addition of medication (including antibiotics) within 1 month of Study Day 1 or any other clinically significant infection as determined by the investigator within 1 month of Day 1. History of alcohol or drug abuse or dependence within 12 months of screening as determined by the Investigator. Male and female of child-bearing potential, unless they are using highly effective methods of contraception during participation in the clinical study and for 4 weeks after termination from study. Pregnant or nursing women.

Facility Information:

Facility Name

Celerion

City

Belfast

ZIP/Postal Code

BT9 6AD

Country

United Kingdom

Facility Name

Medicines Evaluation Unit

City

Manchester

Country

United Kingdom

12. IPD Sharing Statement

Plan to Share IPD

Yes

Learn more about this trial

Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis on KALYDECO® as Background Therapy

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