A Single and Multiple Ascending Dose Study to Evaluate the Safety and Pharmacokinetics of PU-AD in Healthy Subjects

This is a first in human Phase 1 study in two parts with healthy volunteers receiving a single dose of PU AD in three small cohorts and a multiple ascending dose in two small cohorts.

Study Overview

• Status: Terminated
• Conditions: Alzheimer's Disease
• Intervention / Treatment: Drug: Placebo; Drug: Placebo; Drug: PU-AD; Drug: PU-AD

Detailed Description

This is a Phase 1, double-blind trial in two parts. A single ascending dose study in approximately 3 cohorts receiving a single oral dose of PU-AD or placebo and a multiple ascending dose study in 2 cohorts. Each subject in all cohorts will be administered an oral solution of PU AD or placebo under fasting conditions. Each cohort will contain subjects randomized to active treatment or placebo, evaluating safety and tolerance.

• Study Type: Interventional
• Enrollment (Actual): 40
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Texas
    • San Antonio, Texas, United States, 78209
      • ICON Early Phase Services

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

1. Male or female (Women of non-child bearing potential)
2. 18 to 60 years of age for part one, >/= 60 years of age for part two

Exclusion Criteria:

1. Women of child bearing potential or Female with positive pregnancy test or who is lactating.
2. History or presence of conditions, which in the judgment of the PI, are known to interfere with the absorption distribution, metabolism, or excretion of drugs.
3. History or presence of conditions that may place the subject at increased risk as determined by the PI.
4. Has taken other investigational drugs or participated in any clinical study within 30 days.
5. Any other condition or prior therapy that, in the PI's opinion, would make the subject unsuitable for the study, or unable or unwilling to comply with the study procedures

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Single Dose Placebo; Patients randomized to receive Placebo	Drug: Placebo; 3 cohorts receiving a single oral dose of Placebo at one time; Drug: Placebo; 2 cohorts receiving multiple oral dose of Placebo at one time
Experimental: Single Dose Active (PU-AD); Patients randomized to receive Active (PU-AD)	Drug: PU-AD; 3 cohorts receiving a single oral dose of PU-AD at one time.; Drug: PU-AD; 2 cohorts receiving multiple oral dose of PU-AD at one time
Experimental: Multiple Dose (Placebo); Patients randomized to receive Placebo	Drug: Placebo; 3 cohorts receiving a single oral dose of Placebo at one time; Drug: Placebo; 2 cohorts receiving multiple oral dose of Placebo at one time
Experimental: Multiple Dose Active (PU-AD); Patients randomized to receive Active (PU-AD)	Drug: PU-AD; 3 cohorts receiving a single oral dose of PU-AD at one time.; Drug: PU-AD; 2 cohorts receiving multiple oral dose of PU-AD at one time

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate the safety and tolerability of single and multiple doses of PU-AD in healthy subjects	Adverse Event (AE) incidence and changes from baseline in clinical laboratory test results. Number and percentage of subjects reporting any treatment emergent AE will be tabulated by system organ class and preferred term for each treatment (coded using Medical Dictionary for Regulatory Activities). Treatment-emergent AEs will be further classified by severity and relationship to treatment.	Day 1 to Day 3
To evaluate the safety and tolerability of single and multiple doses of PU-AD in healthy subjects	Adverse event incidence and changes from baseline in Electrocardiogram. Number and percentage of subjects reporting any treatment emergent AE will be tabulated by system organ class and preferred term for each treatment (coded using Medical Dictionary for Regulatory Activities). Treatment-emergent AEs will be further classified by severity and relationship to treatment.	Day 1 to Day 3
To evaluate the safety and tolerability of single and multiple doses of PU-AD in healthy subjects	Adverse event incidence and changes from baseline in vital signs . Number and percentage of subjects reporting any treatment emergent AE will be tabulated by system organ class and preferred term for each treatment (coded using Medical Dictionary for Regulatory Activities). Treatment-emergent AEs will be further classified by severity and relationship to treatment.	Day 1 to Day 3

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To determine the pharmacokinetics (PK) PU-AD in healthy subjects	Collect PK parameters to estimate human exposure,after dose administration for each cohort will be evaluated using a power model for dose proportionality. (Maximum observed concentration (Cmax).	Day 1 to Day 3
To determine the pharmacokinetics (PK) PU-AD in healthy subjects	Collect PK parameters to estimate human exposure,after dose administration for each cohort will be evaluated using a power model for dose proportionality. (Time to maximum observed concentration (tmax).	Day 1 to Day 3
To determine the pharmacokinetics (PK) PU-AD in healthy subjects	Collect PK parameters to estimate human exposure,after dose administration for each cohort will be evaluated using a power model for dose proportionality. (Area under the concentration-time curve (AUC).	Day 1 to Day 3

Collaborators and Investigators

• Sponsor: Samus Therapeutics, Inc.
• Investigators: Study Director: Michael H Silverman, M.D., Samus Therapeutics

Study record dates

Study Major Dates

• Study Start (Actual): June 24, 2019
• Primary Completion (Actual): December 23, 2019
• Study Completion (Actual): December 23, 2019

Study Registration Dates

• First Submitted: April 24, 2019
• First Submitted That Met QC Criteria: April 30, 2019
• First Posted (Actual): May 2, 2019

Study Record Updates

• Last Update Posted (Actual): April 18, 2023
• Last Update Submitted That Met QC Criteria: April 13, 2023
• Last Verified: April 1, 2023

More Information

Terms related to this study

• Keywords: PU-AD
• Additional Relevant MeSH Terms: Mental Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurocognitive Disorders; Neurodegenerative Diseases; Dementia; Tauopathies; Alzheimer Disease
• Other Study ID Numbers: PU-AD-01-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No