Safety and Tolerability of PNT001 in Healthy Adults

NCT04096287 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: PNT001-001
• Study Type: interventional
• Target: 49
• Locations: 1 country
• Sites: 4

Brief Summary

This first in human study is a multi-center, randomized, double-blind, placebo-controlled single ascending dose study to evaluate the safety, tolerability, pharmacokinetics, and immunogenicity of intravenous PNT001 in healthy adult participants.

Conditions

Healthy

Outcome Measures

Primary Outcomes (7)

• Incidence of Treatment Emergent Adverse Events

  assess adverse events during 16 week duration of study

  16 weeks

• Incidence of Treatment Emergent Clinical Laboratory Test Abnormalities

  measure clinical laboratory values during 16 week duration of study

  16 weeks

• Incidence of Treatment Emergent Abnormalities in Physical Examination Findings

  observe skin, eyes, ears, nose, throat, cardiac and pulmonary status, abdomen, and extremities for any abnormalities

  16 weeks

• Incidence of Treatment Emergent Abnormalities in Neurological Examination Findings

  perform a neurological assessment of orientation, cranial nerve function, limb function for presence of involuntary movements, muscle mass, tone, and strength, coordination, reflexes, sensation, joint position, gait, Romberg test

  16 weeks

• Incidence of Treatment Emergent Abnormalities in Blood Pressure

  measure resting pulse rate as beats per minute

  16 weeks

• Incidence of Treatment Emergent Abnormalities in Pulse Rate

  measure systolic and diastolic blood pressure in mmHg

  16 weeks

• Incidence of Treatment Emergent Abnormalities in 12 lead Electrocardiogram Assessment

  measure QT and calculate QTcF value

  16 weeks

Secondary Outcomes (2)

• Pharmacokinetic properties of PNT001 in Serum

  16 weeks

• Pharmacokinetic properties of PNT001 in Cerebrospinal Fluid (CSF)

  28 days

Other Outcomes (6)

• Pharmacodynamic effects of PNT001 in Cerebrospinal Fluid - total tau

  28 days

• Pharmacodynamic effects of PNT001 in Cerebrospinal Fluid - cis-pT231 tau

  28 days

• Pharmacodynamic effects of PNT001 in Cerebrospinal Fluid - total pT231 tau

  28 days

Study Arms (2)

PNT001

EXPERIMENTAL

Single escalating doses of intravenous PNT001 administered as a 30 minute infusion at doses of 33mg, 100mg, 300mg, 900mg, 2700mg, and as a 60 minute infusion at 4000 mg

Biological: PNT001

Placebo

PLACEBO COMPARATOR

Single intravenous dose of vehicle administered as a 30 minute infusion up to 2700 mg and as a 60 minute infusion at 4000 mg

Drug: Placebo

Interventions

PNT001 BIOLOGICAL

PNT001 diluted in 5% dextrose

PNT001

Placebo DRUG

5% dextrose

Placebo

Eligibility Criteria

• Age: 21 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• The participant provides written informed consent.
• The participant is a male or female (not of childbearing potential), 21 to 65 years of age at time of screening.
• Female participants must have documented proof that they are not of childbearing potential and must not currently be breastfeeding.
• Male participants must agree to use barrier contraceptives plus spermicide and refrain from sperm donation during the entire study and for 200 days after dosing has been completed.
• The participant must not have participated in a clinical drug trial within 3 months of study start, or within 5 half-lives, unless the study blind has been broken and the participant was known to be on placebo
• The participant must have a body mass index of 18.5 to 30 kg/m\^2

You may not qualify if:

• Any contraindication or inability to undergo lumbar puncture due to anticoagulant use, platelet level, or coagulation study/INR result
• Any significant acute or chronic medical illness
• Any history of cancer within 5 years of enrollment, with the exception of resected skin basal cell carcinoma
• Any major surgery within 4 weeks of study drug administration
• Donation of blood or serum \> 500 mL to a blood bank or in a clinical study (except screening visit) within 3 months of study drug administration
• Inability to undergo venipuncture or tolerate venous access
• A history of smoking or using tobacco products within 3 months before study drug administration
• A history of drug or alcohol abuse as defined in DSM V, Diagnostic Criteria for Drug and Alcohol Abuse
• Evidence of any clinically significant neurological or psychiatric disorder that could interfere with study assessments as determined by investigator and sponsor
• A history or current status of schizophrenia, schizoaffective disorder or bipolar disorder, untreated major depression (DSM-V or ICD-10 criteria)
• Any significant illness or infection requiring intervention within the prior 30 days as determined by the investigator and sponsor
• An indication of potential suicidality risk based on the C-SSRS assessment
• Any of the following abnormalities:
• serum creatinine \> 1.5 mg/dL at screening
• AST or ALT \> 2x the upper limit of normal at screening

Sponsors & Collaborators

• Pinteon Therapeutics, Inc: lead

Study Sites (4)

Woodland Research Northwest

Rogers, Arkansas, 72758, United States

Location

Pacific Research Network, Inc.

San Diego, California, 92103, United States

Location

Hassman Research Institute

Marlton, New Jersey, 08053, United States

Location

Worldwide Clinical Trials

San Antonio, Texas, 78217, United States

Location

Study Officials

• Larry D. Altstiel, MD, PhD

  Pinteon Therapeutics, Inc

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 17, 2019
• First Posted: September 19, 2019
• Study Start: September 17, 2019
• Primary Completion: February 15, 2021
• Study Completion: February 15, 2021
• Last Updated: February 23, 2021

Record last verified: 2021-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)