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

Multiple Sclerosis Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04725175
• Other study ID #: PIPE-307-101
• Status: Completed
• Phase: Phase 1
• Start date: February 26, 2021
• Est. completion date: September 1, 2021

Study information

• Verified date: November 2020
• Source: Pipeline Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 70
• Est. completion date: September 1, 2021
• Est. primary completion date: September 1, 2021
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 55 Years

Eligibility

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

Related Conditions & MeSH terms

• Multiple Sclerosis

Intervention

Drug:
• PIPE-307
Single and multiple ascending oral doses of PIPE-307 tablets
• Placebo oral tablet
Single and multiple ascending oral doses of matching Placebo tablets

Locations

Country	Name	City	State
Australia	Linear Clinical Research	Nedlands	Western Australia

Sponsors (1)

Lead Sponsor	Collaborator
Pipeline Therapeutics, Inc.

Country where clinical trial is conducted

Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety: Treatment-Emergent Adverse Events (TEAE)	Number of participants with TEAEs	From baseline to 7 days post dosing for SAD cohorts and 21 days post dosing for MAD cohorts
Secondary	Safety: Cardiac repolarization using Fridericia-corrected QT interval (QTcF)	Change in mean QTcF	From baseline to 14 days post dose for SAD cohorts and 21 days post last dose for MAD cohorts
Secondary	Pharmacokinetics (PK): Blood concentration levels of PIPE-307		From baseline to 14 days post dose for SAD cohorts and 21 days post last dose for MAD cohorts
Secondary	PK: Urine concentration levels of PIPE-307		From baseline on day 1 through day 2 for SAD cohorts, and from baseline on day 1 though day 7 for the MAD cohorts
Secondary	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.	From baseline to day 2 for SAD cohorts and from baseline to day 7 for MAD cohorts