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A First-in-Human Phase 1 Study of Plasmalogen Precursor PPI-1011 in Healthy Adult Volunteers to Assess Safety, Tolerability, and Pharmacokinetics

NCT05969977 · Status: UNKNOWN · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 56

Last updated 2023-08-01

No results posted yet for this study

Summary

PPI-1011 is being developed as a docosahexaenoic acid (DHA) containing plasmalogen precursor with good long-term stability, specifically for the treatment of rhizomelic chondrodysplasia punctata (RCDP), which is an ultra rare type of peroxisomal biogenesis disorder (PBD). The goal of treatment with PPI-1011 is to increase the levels of plasmalogens within circulation and tissues, with the hope that this will normalize plasmalogen levels in the body and result in clinical improvement to patients. The present study is a first-in-human (FIH), phase 1, randomized, double-blind, placebo-controlled, dose-escalation study to assess the safety, tolerability and pharmacokinetics (PK) of single and multiple ascending doses of PPI-1011 administered orally to healthy subjects. The study consists of 5 planned single-dose cohorts (n = 8 per cohort, total randomized 6 active: 2 placebo) with sentinel design. Following a review of the safety and PK data by the safety review committee and submission to Health Canada the study will be expanded to include 2 planned multiple-dose cohorts (n = 8 per cohort, total randomized 6 active: 2 placebo).

Conditions

  • Rhizomelic Chondrodysplasia Punctata

Interventions

DRUG

PPI-1011

PPI-1011 is a liquid oil with light yellow to amber color, clear to slightly opalescent and flows readily at room temperature. It is insoluble in water and miscible with dichloromethane and liquid coconut oil. It is a synthetic glycerolipid derivative, comprised of an assembly of naturally-occurring products including glycerol backbone, an ether-linked hexadecyl group at sn1 position, an all-cis-4, 7, 10, 13, 16, 19-docosahexaenoyl group at sn2 position, and a lipoyl group at sn3 position. It was designed as a plasmalogen precursor and is similar in structure to naturally occurring alkyl-diacyl lipids with the primary difference being the presence of a lipoic acid moiety at the sn3 position. PPI-1011 is formulated in liquid coconut oil containing 0.1% 1-thioglycerol as an antioxidant.

DRUG

Placebo

Placebo

Sponsors & Collaborators

  • BioPharma Services Inc.

    collaborator INDUSTRY

  • MED-LIFE DISCOVERIES LP

    lead INDUSTRY

Principal Investigators

  • Tara Smith, PhD · MED-LIFE DISCOVERIES LP

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2023-05-29
Primary Completion
2023-11-30
Completion
2023-11-30

Countries

  • Canada

Study Locations

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Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05969977 on ClinicalTrials.gov