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.

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.

Condition or disease	Intervention/treatment	Phase
Multiple Sclerosis	Drug: PIPE-307; Drug: Placebo oral tablet	Phase 1

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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 70 participants
• 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
• 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
• Actual Study Start Date: February 26, 2021
• Actual Primary Completion Date: September 1, 2021
• Actual Study Completion Date: September 1, 2021

Arms and Interventions

Arm	Intervention/treatment
Experimental: PIPE-307	Drug: PIPE-307; Single and multiple ascending oral doses of PIPE-307 tablets
Placebo Comparator: Placebo	Drug: Placebo oral tablet; Single and multiple ascending oral doses of matching Placebo tablets

Outcome Measures

Primary Outcome Measures :

1. 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

Secondary Outcome Measures :

1. 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
2. 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 ]
3. 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 ]
4. 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.

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 55 Years (Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: Yes

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

Contacts and Locations

Locations

Australia, Western Australia

Linear Clinical Research

Nedlands, Western Australia, Australia, 6009

Sponsors and Collaborators

Pipeline Therapeutics, Inc.

Investigators

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

More Information

• Responsible Party: Pipeline Therapeutics, Inc.
• ClinicalTrials.gov Identifier: NCT04725175
• Other Study ID Numbers: PIPE-307-101
• First Posted: January 26, 2021
• Last Update Posted: November 3, 2021
• Last Verified: November 2020

• 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

Additional relevant MeSH terms:

Multiple Sclerosis; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases