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Phase I Study Evaluating Safety and Tolerability of Escalating Single and Multiple Doses of of PIPE-307 and Food Effect in Healthy Volunteers

Primary Purpose

Multiple Sclerosis

Status

Completed

Phase

Phase 1

Locations

Australia

Study Type

Interventional

Intervention

PIPE-307

Placebo oral tablet

About this trial

This is an interventional treatment trial for Multiple Sclerosis

Eligibility Criteria

18 Years - 55 Years (Adult)All SexesAccepts Healthy Volunteers

Inclusion Criteria:

Male or female between 18 and 55 years of age (inclusive) at time of signing informed consent.
BMI is between 18.0 and 32.0 kg/m2
Male or female subjects with reproductive potential agree to comply with protocol-approved double barrier contraceptive method 30 days prior to first dose and up to 90 days post last dose
Medically healthy with no clinically significant or relevant abnormalities in medical history physical exam, vital signs, electrocardiogram (ECG), or laboratory evaluations (hematology, chemistry, and urinalysis) as assessed by the Investigator.

Exclusion Criteria:

Has a current or recurrent diseases that could affect the investigational medicinal product or affect clinical or laboratory assessments
Experienced a significant systemic illness, as judged by the Investigator, within 30 days of the first dose
Has a history of a significant medical, including hepatic and/or renal disease as outlined in the protocol, or psychiatric disorder that may require treatment or make the participant unlikely to fully complete the study or increase risk to the participant.
History of alcohol or other substance abuse within the 12 months prior to dosing at the discretion of the Investigator
Routine alcohol consumption meeting or exceeding protocol limits
History of prior malignancy (except adequately treated non-melanoma skin cancer, carcinoma I-situ of the uterine cervix, ductal carcinoma in situ (DCIS), or localized prostate cancer
Donated or lost more than 400ml of blood within 56 days or plasma within 14 days prior to screening
Received an investigational agent with the last 30 days prior to dosing or within 5 half-lives of the investigational agent
Use of any prescription medication, over-the-counter medication, vitamin or supplement, herbal or homeopathic preparations with 7 days or 5 half-lives prior to study drug administration, as determined by the Investigator. Hormone replacement therapy and hormonal contraception is permissible throughout the study

Sites / Locations

Linear Clinical Research

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

PIPE-307

Placebo

Arm Description

Outcomes

Primary Outcome Measures

Safety: Treatment-Emergent Adverse Events (TEAE)

Number of participants with TEAEs

Secondary Outcome Measures

Safety: Cardiac repolarization using Fridericia-corrected QT interval (QTcF)

Change in mean QTcF

Pharmacokinetics (PK): Blood concentration levels of PIPE-307

PK: Urine concentration levels of PIPE-307

Exploratory: Impact of PIPE-307 on Cogstate

Cogstate tests have been designed, developed and validated to both identify and measure cognitive impairment, and to track or monitor cognitive changes. The tasks use novel visual and verbal stimuli to ensure assessment is cultural-neutral and not limited by a participant's level of education.

Full Information

NCT ID

NCT04725175

First Posted

January 18, 2021

Last Updated

November 2, 2021

Sponsor

Pipeline Therapeutics, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT04725175

Brief Title

Phase I Study Evaluating Safety and Tolerability of Escalating Single and Multiple Doses of of PIPE-307 and Food Effect in Healthy Volunteers

Official Title

A Phase I, Randomized, Double-Blind, Placebo-Controlled, Safety, Tolerability and Pharmacokinetic Study of Escalating Single and Multiple Doses of PIPE-307 and Food Effect in Normal Healthy Volunteers

Study Type

Interventional

2. Study Status

Record Verification Date

November 2020

Overall Recruitment Status

Completed

Study Start Date

February 26, 2021 (Actual)

Primary Completion Date

September 1, 2021 (Actual)

Study Completion Date

September 1, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Pipeline Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This is a randomized, double-blind study of PIPE-307 or placebo in normal healthy subjects. The study will be conducted in three parts: Part 1 will be a Single Ascending Dose (SAD) study enrolling approximately 48 subjects for a total duration of 6 weeks. Part 2 will be a Multiple Ascending Dose (MAD) study enrolling approximately 24 subjects for a total duration of 7 weeks, and part 3 will be a selected SAD cohort in a fed state to evaluate the effect of food on the bioavailability of PIPE-307, enrolling approximately 8 subjects from a selected SAD cohort for a duration of 6 weeks.

Detailed Description

This is a randomized, double-blind study of PIPE-307 or placebo given as single and multiple escalating doses in normal healthy subjects. The study will be conducted in three parts: Part 1 will be a Single Ascending Dose (SAD) study enrolling approximately 48 subjects for a total duration of 6 weeks. Part 2 will be a Multiple Ascending Dose (MAD) study enrolling approximately 24 subjects for a total duration of 7 weeks, and part 3 will be a selected SAD cohort in a fed state to evaluate the effect of food on the bioavailability of PIPE-307, enrolling approximately 8 subjects from a selected SAD cohort for a duration of 6 weeks. Safety will be assessed by periodic measurement of vital signs, physical examinations, electrocardiograms, blood laboratory analyses and occurrence of adverse events (AE).

6. Conditions and Keywords

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

Multiple Sclerosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Sequential Assignment

Masking

ParticipantInvestigator

Masking Description

All roles are masked with the exception of the pharmacist/dose preparer.

Allocation

Randomized

Enrollment

70 (Actual)

8. Arms, Groups, and Interventions

Arm Title

PIPE-307

Arm Type

Experimental

Arm Title

Placebo

Arm Type

Placebo Comparator

Intervention Type

Drug

Intervention Name(s)

PIPE-307

Intervention Description

Single and multiple ascending oral doses of PIPE-307 tablets

Intervention Type

Drug

Intervention Name(s)

Placebo oral tablet

Intervention Description

Single and multiple ascending oral doses of matching Placebo tablets

Primary Outcome Measure Information:

Title

Safety: Treatment-Emergent Adverse Events (TEAE)

Description

Number of participants with TEAEs

Time Frame

From baseline to 7 days post dosing for SAD cohorts and 21 days post dosing for MAD cohorts

Secondary Outcome Measure Information:

Title

Safety: Cardiac repolarization using Fridericia-corrected QT interval (QTcF)

Description

Change in mean QTcF

Time Frame

From baseline to 14 days post dose for SAD cohorts and 21 days post last dose for MAD cohorts

Title

Pharmacokinetics (PK): Blood concentration levels of PIPE-307

Time Frame

From baseline to 14 days post dose for SAD cohorts and 21 days post last dose for MAD cohorts

Title

PK: Urine concentration levels of PIPE-307

Time Frame

From baseline on day 1 through day 2 for SAD cohorts, and from baseline on day 1 though day 7 for the MAD cohorts

Title

Exploratory: Impact of PIPE-307 on Cogstate

Description

Time Frame

From baseline to day 2 for SAD cohorts and from baseline to day 7 for MAD cohorts

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: Male or female between 18 and 55 years of age (inclusive) at time of signing informed consent. BMI is between 18.0 and 32.0 kg/m2 Male or female subjects with reproductive potential agree to comply with protocol-approved double barrier contraceptive method 30 days prior to first dose and up to 90 days post last dose Medically healthy with no clinically significant or relevant abnormalities in medical history physical exam, vital signs, electrocardiogram (ECG), or laboratory evaluations (hematology, chemistry, and urinalysis) as assessed by the Investigator. Exclusion Criteria: Has a current or recurrent diseases that could affect the investigational medicinal product or affect clinical or laboratory assessments Experienced a significant systemic illness, as judged by the Investigator, within 30 days of the first dose Has a history of a significant medical, including hepatic and/or renal disease as outlined in the protocol, or psychiatric disorder that may require treatment or make the participant unlikely to fully complete the study or increase risk to the participant. History of alcohol or other substance abuse within the 12 months prior to dosing at the discretion of the Investigator Routine alcohol consumption meeting or exceeding protocol limits History of prior malignancy (except adequately treated non-melanoma skin cancer, carcinoma I-situ of the uterine cervix, ductal carcinoma in situ (DCIS), or localized prostate cancer Donated or lost more than 400ml of blood within 56 days or plasma within 14 days prior to screening Received an investigational agent with the last 30 days prior to dosing or within 5 half-lives of the investigational agent Use of any prescription medication, over-the-counter medication, vitamin or supplement, herbal or homeopathic preparations with 7 days or 5 half-lives prior to study drug administration, as determined by the Investigator. Hormone replacement therapy and hormonal contraception is permissible throughout the study

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Stephen Huhn, MD

Organizational Affiliation

Chief Medical Officer, Pipeline Therapeutics, Inc.

Official's Role

Study Director

Facility Information:

Facility Name

Linear Clinical Research

City

Nedlands

State/Province

Western Australia

ZIP/Postal Code

6009

Country

Australia

12. IPD Sharing Statement

Learn more about this trial

Phase I Study Evaluating Safety and Tolerability of Escalating Single and Multiple Doses of of PIPE-307 and Food Effect in Healthy Volunteers

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