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

• ClinicalTrials.gov Identifier: NCT03935568
• Recruitment Status: Completed
• First Posted: May 2, 2019
• Last Update Posted: December 26, 2019
• Sponsor: Samus Therapeutics, Inc.
• Information provided by (Responsible Party): Samus Therapeutics, Inc.

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
Alzheimer's Disease	Drug: PU-AD; Drug: Placebo	Phase 1

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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 40 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Triple (Participant, Care Provider, Investigator)
• Primary Purpose: Treatment
• Official Title: A Single and Multiple Ascending Dose Study to Evaluate the Safety and Pharmacokinetics of PU-AD in Healthy Subjects
• Actual Study Start Date: June 24, 2019
• Actual Primary Completion Date: December 23, 2019
• Actual Study Completion Date: December 23, 2019

Arms and Interventions

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
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.
Experimental: Multiple Dose (Placebo); Patients randomized to receive Placebo	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; 2 cohorts receiving multiple oral dose of PU-AD at one time

Outcome Measures

Primary Outcome Measures :

1. To evaluate the safety and tolerability of single and multiple doses of PU-AD in healthy subjects [ Time Frame: Day 1 to Day 3 ]
   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.
2. To evaluate the safety and tolerability of single and multiple doses of PU-AD in healthy subjects [ Time Frame: Day 1 to Day 3 ]
   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.
3. To evaluate the safety and tolerability of single and multiple doses of PU-AD in healthy subjects [ Time Frame: Day 1 to Day 3 ]
   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.

Secondary Outcome Measures :

1. To determine the pharmacokinetics (PK) PU-AD in healthy subjects [ Time Frame: Day 1 to Day 3 ]
   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).
2. To determine the pharmacokinetics (PK) PU-AD in healthy subjects [ Time Frame: Day 1 to Day 3 ]
   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).
3. To determine the pharmacokinetics (PK) PU-AD in healthy subjects [ Time Frame: Day 1 to Day 3 ]
   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).

Eligibility Criteria

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

Criteria

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

Contacts and Locations

Locations

United States, Texas

ICON Early Phase Services

San Antonio, Texas, United States, 78209

Sponsors and Collaborators

Samus Therapeutics, Inc.

Investigators

• Study Director: Michael H Silverman, M.D.; Samus Therapeutics

More Information

• Responsible Party: Samus Therapeutics, Inc.
• ClinicalTrials.gov Identifier: NCT03935568
• Other Study ID Numbers: PU-AD-01-001
• First Posted: May 2, 2019
• Last Update Posted: December 26, 2019
• Last Verified: December 2019

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• 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

Keywords provided by Samus Therapeutics, Inc.:

PU-AD

Additional relevant MeSH terms:

Alzheimer Disease; Dementia; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tauopathies; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders