Study Evaluating Safety and Tolerability of Escalating Single and Multiple Doses of PIPE-791 and Food Effect in Healthy Volunteers

NCT05983939 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: PTI-791-101
• Study Type: interventional
• Target: 56
• Locations: 1 country
• Sites: 1

Brief Summary

This study will be conducted in three parts: Part 1 will be a Single Ascending Dose (SAD), Part 2 will be a Multiple Ascending Dose (MAD), and Part 3 will be a selected SAD cohort in a fed state. Safety will be assessed by periodic measurement of vital signs, physical examinations, electrocardiograms, blood laboratory analyses and occurrence of adverse events (AE).

Conditions

Multiple Sclerosis

Keywords

Multiple Sclerosis; PIPE-791; Health volunteers

Outcome Measures

Primary Outcomes (1)

• Safety: Treatment-Emergent Adverse Events (TEAE)

  Number of participants with TEAEs

  Baseline to 14 days post dosing for SAD cohorts and 14 days post dosing for MAD cohorts

Secondary Outcomes (3)

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

  Baseline to 14 days post dosing for SAD cohorts and 14 days post dosing for MAD cohorts

• Pharmacokinetics (PK): Blood concentration levels of PIPE-791

  Baseline to 14 days post dosing for SAD cohorts and 14 days post dosing for MAD cohorts

• Pharmacokinetics: Urine concentration levels of PIPE-791

  Baseline on day 1 through day 2 for SAD cohorts and from baseline on day 1 through day 7 for MAD cohorts

Study Arms (2)

PIPE-791

EXPERIMENTAL

Drug: PIPE-791

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

PIPE-791 DRUG

Single and multiple ascending oral doses of PIPE-791 tablets.

PIPE-791

Placebo DRUG

Single and multiple ascending oral doses of matching placebo tablets.

Placebo

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Male or female between 18 and 55 years of age (inclusive) at the time of signing informed consent.
• Male or female subjects with reproductive potential agree to comply with protocol-approved double barrier contraceptive method 30 days prior to the 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 Investigatory.

You may not qualify if:

• Has a current or recurrent disease 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 the 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 in-situ of the uterine cervix, ductal carcinoma in situ (DCIS), or localized prostate cancer).
• Donated or lost more than 400 mL of blood within 56 days or plasma within 14 days prior to Screening.
• Received an investigational agent within the last 30 days, prior to screening, or five half-lives of the prior investigational agent.
• Use of any prescription medication, over-the-counter medication, vitamin or supplement, herbal or homeopathic preparation within 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.

Sponsors & Collaborators

• Contineum Therapeutics: lead

Study Sites (1)

Worldwide Clinical Trials

San Antonio, Texas, 78217, United States

Location

MeSH Terms

Conditions

Multiple Sclerosis

Condition Hierarchy (Ancestors)

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

Study Officials

• Stephen Huhn, MD

  Pipeline Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: All roles are masked with the exception of the pharmacist/dose preparer.
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 19, 2023
• First Posted: August 9, 2023
• Study Start: June 26, 2023
• Primary Completion: February 1, 2024
• Study Completion: February 1, 2024
• Last Updated: February 14, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)