A First-in-Human Phase 1 Study of Plasmalogen Precursor PPI-1011 in Healthy Adult Volunteers to A… (NCT05969977) | Clinical Trial Compass
UnknownPhase 1
A First-in-Human Phase 1 Study of Plasmalogen Precursor PPI-1011 in Healthy Adult Volunteers to Assess Safety, Tolerability, and Pharmacokinetics
Canada56 participantsStarted 2023-05-29
Plain-language 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).
Who can participate
Age range18 Years – 65 Years
SexALL
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Inclusion criteria
✓. Healthy, non-smoking (for at least 6 months prior to first study drug administration) male, and non-pregnant and non-lactating female (including post-menopausal and surgically sterile female) volunteers, 18-65 years of age, inclusive at the time of informed consent.
✓. Body mass index (BMI) that is within 18.5 - 30.0 kg/m2, inclusive, and subject weighs no more than 91.0 kg.
✓. Healthy, according to the medical history, ECG, vital signs, laboratory results and physical examination as determined by the PI/Sub-Investigator.
✓. Systolic blood pressure between 95-140 mmHg, inclusive, and diastolic blood pressure between 55-90 mmHg, inclusive, oxygen saturation level between 95% and 100%, inclusive, and heart rate between 55-100 bpm, inclusive, unless deemed otherwise by the PI/Sub-Investigator.
✓. Clinical laboratory values within BPSI's most recent acceptable laboratory test range, and/or values are deemed by the PI/Sub-Investigator as "Not Clinically Significant".
✓. Ability to comprehend and be informed of the nature of the study, as assessed by BPSI staff. Capable of giving written informed consent prior to any study related procedure. Must be able to communicate effectively with clinic staff.
✓. Ability to fast for at least 10 hours and consume standard meals.
What they're measuring
1
Treatment-emergent adverse events as assessed by the CTCAE scale
Timeframe: Up to 6 days post-dose in single ascending dose subjects and up to 7 days post the last dose in multiple ascending dose subjects.
. Availability to volunteer for the entire study duration and willing to adhere to all protocol requirements.
Exclusion criteria
✕. Known history or presence of any clinically significant hepatic, renal/genitourinary, gastrointestinal, cardiovascular, cerebrovascular, pulmonary, endocrine, immunological, musculoskeletal, neurological, psychiatric, dermatological or hematological disease or condition unless determined as not clinically significant by the PI/Sub-Investigator.
✕. Clinically significant history or presence of any clinically significant gastrointestinal pathology (e.g., chronic diarrhea, inflammatory bowel disease), unresolved gastrointestinal symptoms (e.g., diarrhea, vomiting), or other conditions known to interfere with the absorption, distribution, metabolism or excretion of the drug experienced within 7 days prior to first study drug administration, as determined by the PI/Sub-Investigator.
✕. Presence of any clinically significant illness within 30 days prior to first dosing, as determined by the PI/Sub-Investigator.
✕. Presence of any significant physical or organ abnormality as determined by the PI/Sub-Investigator.
✕. A known history or positive test result for human immunodeficiency virus (HIV), chronic Hepatitis B surface antigen, or Hepatitis C.
✕. A positive test result for drugs of abuse/recreational drugs (marijuana, amphetamines, barbiturates, cocaine, opiates, phencyclidine and benzodiazepines), alcohol test and cotinine. Positive pregnancy test for female subjects of childbearing potential.