Alzheimer's DiseasePhase 1NCT03935568
A Single and Multiple Ascending Dose Study to Evaluate the Safety and Pharmacokinetics of PU-AD in Healthy Subjects
A Phase 1 study testing PU-AD in people with Alzheimer's Disease, run by Samus Therapeutics, Inc. at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- Alzheimer's Disease
- What is being tested?
- PU-AD
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- 24 Jun 2019to 23 Dec 2019
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Male or female (Women of non-child bearing potential)
- 18 to 60 years of age for part one, >/= 60 years of age for part two
Who cannot join
- Women of child bearing potential or Female with positive pregnancy test or who is lactating.
- 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.
- History or presence of conditions that may place the subject at increased risk as determined by the PI.
- Has taken other investigational drugs or participated in any clinical study within 30 days.
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the 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.
- Conditions
- Alzheimer's Disease
- Being tested
- PU-AD, Placebo
- Keywords
- PU-AD
- Medical subject headings
- Alzheimer Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PU-ADDrug
3 cohorts receiving a single oral dose of PU-AD at one time.
PlaceboDrug
3 cohorts receiving a single oral dose of Placebo at one time
PlaceboDrug
2 cohorts receiving multiple oral dose of Placebo at one time
PU-ADDrug
2 cohorts receiving multiple oral dose of PU-AD at one time
| Study group | Role | What they receive |
|---|---|---|
| Single Dose Placebo | Experimental | Patients randomized to receive Placebo Receives: Drug: Placebo |
| Single Dose Active (PU-AD) | Experimental | Patients randomized to receive Active (PU-AD) Receives: Drug: PU-AD |
| Multiple Dose (Placebo) | Experimental | Patients randomized to receive Placebo Receives: Drug: Placebo |
| Multiple Dose Active (PU-AD) | Experimental | Patients randomized to receive Active (PU-AD) Receives: Drug: PU-AD |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: Day 1 to Day 3
Secondary outcomes (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. (Maximum observed concentration (Cmax).
Measured: 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).
Measured: 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).
Measured: Day 1 to Day 3
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 May 2019
- Study start24 Jun 2019
- Primary completion23 Dec 2019
- Study completion23 Dec 2019
Why it stopped: Company ceased operations
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Alzheimer's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Samus Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Michael H Silverman, M.D., Study director, Samus Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03935568
- Sponsor's protocol ID
- PU-AD-01-001
- Status last verified
- Apr 2023
- First submitted
- 24 Apr 2019
- Last updated
- 18 Apr 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Silverman MH, Duggan S, Bardelli G, Sadler B, Key C, Medlock M, Reynolds L, Wallner B. Safety, Tolerability and Pharmacokinetics of Icapamespib, a Selective Epichaperome Inhibitor, in Healthy Adults. J Prev Alzheimers Dis. 2022;9(4):635-645. doi: 10.14283/jpad.2022.71.
PubMed 36281667
Study at a glance
From the registry record, last updated by the sponsor on 18 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single and Multiple Ascending Dose Study to Evaluate the Safety and Pharmacokinetics of PU-AD in Healthy Subjects
- ClinicalTrials.gov ID
- NCT03935568
- Acronym / sponsor ID
- PU-AD-01-001
- Conditions
- Alzheimer's Disease
- Keywords
- PU-AD
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Company ceased operations
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Samus Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 24 Jun 2019 (actual)
- Primary completion
- 23 Dec 2019 (actual)
- Study completion
- 23 Dec 2019 (actual)
- First posted
- 2 May 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Single Dose Placebo | Experimental | Drug: PlaceboPatients randomized to receive Placebo |
| Single Dose Active (PU-AD) | Experimental | Drug: PU-ADPatients randomized to receive Active (PU-AD) |
| Multiple Dose (Placebo) | Experimental | Drug: PlaceboPatients randomized to receive Placebo |
| Multiple Dose Active (PU-AD) | Experimental | Drug: PU-ADPatients randomized to receive Active (PU-AD) |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Male or female (Women of non-child bearing potential)
- 18 to 60 years of age for part one, >/= 60 years of age for part two
Key exclusion criteria (5 listed)
- Women of child bearing potential or Female with positive pregnancy test or who is lactating.
- 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.
- History or presence of conditions that may place the subject at increased risk as determined by the PI.
- Has taken other investigational drugs or participated in any clinical study within 30 days.
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PU-AD3 cohorts receiving a single oral dose of PU-AD at one time. | Drug | Single Dose Active (PU-AD) |
| Placebo3 cohorts receiving a single oral dose of Placebo at one time | Drug | Single Dose Placebo |
| Placebo2 cohorts receiving multiple oral dose of Placebo at one time | Drug | Multiple Dose (Placebo) |
| PU-AD2 cohorts receiving multiple oral dose of PU-AD at one time | Drug | Multiple Dose Active (PU-AD) |
Primary outcome
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.
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.
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.
3 secondary outcomes
To determine the pharmacokinetics (PK) PU-AD in healthy subjects
Time frame: Day 1 to Day 3
To determine the pharmacokinetics (PK) PU-AD in healthy subjects
Time frame: Day 1 to Day 3
To determine the pharmacokinetics (PK) PU-AD in healthy subjects
Time frame: Day 1 to Day 3
Results and safety
The study was stopped early (reason given: Company ceased operations). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Apr 2023.
- Derived from this study: PubMed 36281667Silverman MH, Duggan S, Bardelli G, Sadler B, Key C, Medlock M, Reynolds L, Wallner B. Safety, Tolerability and Pharmacokinetics of Icapamespib, a Selective Epichaperome Inhibitor, in Healthy Adults. J Prev Alzheimers Dis. 2022;9(4):635-645. doi: 10.14283/jpad.2022.71.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michael H Silverman, M.D. study director
Samus Therapeutics
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PU-AD in people with Alzheimer's Disease, run by Samus Therapeutics, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 18 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Company ceased operations). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the safety and tolerability of single and multiple doses of PU-AD in healthy subjects (time frame: Day 1 to Day 3). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03935568, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Apr 2023.