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

• ClinicalTrials.gov Identifier: NCT04725175
• Recruitment Status: Completed
• First Posted: January 26, 2021
• Last Update Posted: November 3, 2021
• Sponsor: Pipeline Therapeutics, Inc.
• Information provided by (Responsible Party): Pipeline Therapeutics, Inc.

Tracking Information

• First Submitted Date: January 18, 2021
• First Posted Date: January 26, 2021
• Last Update Posted Date: November 3, 2021
• Actual Study Start Date: February 26, 2021
• Actual Primary Completion Date: September 1, 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: January 22, 2021)

Safety: Treatment-Emergent Adverse Events (TEAE) [ Time Frame: From baseline to 7 days post dosing for SAD cohorts and 21 days post dosing for MAD cohorts ]

Number of participants with TEAEs

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: January 22, 2021)

• Safety: Cardiac repolarization using Fridericia-corrected QT interval (QTcF) [ Time Frame: From baseline to 14 days post dose for SAD cohorts and 21 days post last dose for MAD cohorts ]
  Change in mean QTcF
• 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 ]
• 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 ]
• Exploratory: Impact of PIPE-307 on Cogstate [ Time Frame: From baseline to day 2 for SAD cohorts and from baseline to day 7 for MAD cohorts ]
  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.

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• 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
• 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).
• Study Type: Interventional
• Study Phase: Phase 1

Study Design

Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Double (Participant, Investigator)
Masking Description:

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

Primary Purpose: Treatment

• Condition: Multiple Sclerosis

Intervention

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

Study Arms

• Experimental: PIPE-307
  Intervention: Drug: PIPE-307
• Placebo Comparator: Placebo
  Intervention: Drug: Placebo oral tablet

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: November 2, 2021): 70
• Original Estimated Enrollment (submitted: January 22, 2021): 72
• Actual Study Completion Date: September 1, 2021
• Actual Primary Completion Date: September 1, 2021 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 55 Years (Adult)
• Accepts Healthy Volunteers: Yes
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Australia

Administrative Information

• NCT Number: NCT04725175
• Other Study ID Numbers: PIPE-307-101
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: No

• IPD Sharing Statement: Not Provided
• Responsible Party: Pipeline Therapeutics, Inc.
• Study Sponsor: Pipeline Therapeutics, Inc.
• Collaborators: Not Provided

Investigators

• Study Director: Stephen Huhn, MD; Chief Medical Officer, Pipeline Therapeutics, Inc.

• PRS Account: Pipeline Therapeutics, Inc.
• Verification Date: November 2020