NCT03784300 is a Phase 1 clinical trial of 50 mg PTI-125 in Alzheimer Disease, Early Onset, sponsored by Pain Therapeutics.

Who is sponsoring NCT03784300?

NCT03784300 is sponsored by Pain Therapeutics.

What drugs are being tested in NCT03784300?

Interventions in NCT03784300: 50 mg PTI-125, 100 mg PTI-125, 200 mg PTI-125.

What condition does NCT03784300 study?

NCT03784300 studies Alzheimer Disease, Early Onset, Alzheimer Disease.

Is NCT03784300 still recruiting?

NCT03784300 is currently completed. Last updated 10 May 2021.

Are results published for NCT03784300?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2021-05-10 · How we verify

NCT03784300

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

A Safety Study of PTI-125 in Healthy Volunteers

Completed Phase 1 Results posted Last updated 10 May 2021

What this trial tests

Phase 1 trial testing 50 mg PTI-125 in Alzheimer Disease, Early Onset in 24 participants. Completed in 27 March 2018.

Timeline

18 August 2017

Primary endpoint
9 October 2017

27 March 2018

Quick facts

Lead sponsor	Pain Therapeutics
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	triple
Primary purpose	prevention
Enrollment	24
Start date	18 August 2017
Primary completion	9 October 2017
Estimated completion	27 March 2018
Sites	1 location across United States

Drugs / interventions tested

50 mg PTI-125 — full drug profile →
100 mg PTI-125 — full drug profile →
200 mg PTI-125 — full drug profile →

Conditions studied

Alzheimer Disease, Early Onset — all drugs for Alzheimer Disease, Early Onset →
Alzheimer Disease — all drugs for Alzheimer Disease →

Sponsor

Pain Therapeutics — full company profile →

Who can join

Adults 18 to 45, any sex, with Alzheimer Disease, Early Onset or Alzheimer Disease. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Maximum Plasma Concentration (Cmax) Primary · Blood samples will be drawn on Day 1 after dosing at 20, 40, and 60 minutes and at 2, 3, 4, 6, 8, 12, 24, 36, 48 and 72 hours.

The peak drug concentration will be obtained directly from the data without interpolation.

Group	Value	95% CI
50 mg PTI-125	315	± 96.6
50 mg PTI-125 Placebo	NA	± NA
100 mg PTI-125	550	± 146
100 mg PTI-125 Placebo	NA	± NA
200 mg PTI-125	1240	± 276
200 mg PTI-125 Placebo	NA	± NA

Time to Maximum Plasma Concentration (Tmax) (Tmax) Primary · Blood samples will be drawn on Day 1 after dosing at 20, 40, and 60 minutes and at 2, 3, 4, 6, 8, 12, 24, 36, 48 and 72 hours.

The time to peak drug concentration will be obtained directly from the data without interpolation

Group	Value	95% CI
50 mg PTI-125	1.56	± 0.69
50 mg PTI-125 Placebo	NA	± NA
100 mg PTI-125	1.08	± 0.48
100 mg PTI-125 Placebo	NA	± NA
200 mg PTI-125	1.28	± 0.57
200 mg PTI-125 Placebo	NA	± NA

Time to Last Quantifiable Plasma Concentration (Tlast) Primary · Blood samples will be drawn on Day 1 after dosing at 20, 40, and 60 minutes and at 2, 3, 4, 6, 8, 12, 24, 36, 48 and 72 hours.

The time to the last quantifiable drug concentration will be obtained directly from the data without interpolation.

Group	Value	95% CI
50 mg PTI-125	44.0	± 16.4
50 mg PTI-125 Placebo	NA	± NA
100 mg PTI-125	46.0	± 4.6
100 mg PTI-125 Placebo	NA	± NA
200 mg PTI-125	50.00	± 11.8
200 mg PTI-125 Placebo	NA	± NA

Last Quantifiable Plasma Concentration (Clast) Primary · Blood samples will be drawn on Day 1 after dosing at 20, 40, and 60 minutes and at 2, 3, 4, 6, 8, 12, 24, 36, 48 and 72 hours.

The concentration of the last quantifiable drug will be obtained directly from the data without interpolation concentration

Group	Value	95% CI
50 mg PTI-125	1.04	± 0.495
50 mg PTI-125 Placebo	NA	± NA
100 mg PTI-125	0.795	± 0.294
100 mg PTI-125 Placebo	NA	± NA
200 mg PTI-125	0.806	± 0.292
200 mg PTI-125 Placebo	NA	± NA

Elimination Rate Constant (λz) Primary · Blood samples will be drawn on Day 1 after dosing at 20, 40, and 60 minutes and at 2, 3, 4, 6, 8, 12, 24, 36, 48 and 72 hours.

The elimination rate constant (λz) will be calculated.

Group	Value	95% CI
50 mg PTI-125	0.141	± 0.0539
50 mg PTI-125 Placebo	NA	± NA
100 mg PTI-125	0.157	± 0.0155
100 mg PTI-125 Placebo	NA	± NA
200 mg PTI-125	0.144	± 0.0516
200 mg PTI-125 Placebo	NA	± NA

Termination Elimination Half-Life (T1/2) Primary · Blood samples will be drawn on Day 1 after dosing at 20, 40, and 60 minutes and at 2, 3, 4, 6, 8, 12, 24, 36, 48 and 72 hours.

The terminal elimination half-life (T1/2) will be calculated.

Group	Value	95% CI
50 mg PTI-125	6.05	± 3.87
50 mg PTI-125 Placebo	NA	± NA
100 mg PTI-125	4.45	± 0.39
100 mg PTI-125 Placebo	NA	± NA
200 mg PTI-125	5.93	± 3.87
200 mg PTI-125 Placebo	NA	± NA

Area Under the Curve (AUC) Primary · Blood samples will be drawn on Day 1 after dosing at 20, 40, and 60 minutes and at 2, 3, 4, 6, 8, 12, 24, 36, 48 and 72 hours.

The AUC from time zero to the time of the last quantifiable concentration (AUClast) will be calculated.

Group	Value	95% CI
50 mg PTI-125	2040	± 893
50 mg PTI-125 Placebo	NA	± NA
100 mg PTI-125	3130	± 1150
100 mg PTI-125 Placebo	NA	± NA
200 mg PTI-125	8130	± 1530
200 mg PTI-125 Placebo	NA	± NA

Area Under the Curve to Infinity (AUCinf) Primary · Blood samples will be drawn on Day 1 after dosing at 20, 40, and 60 minutes and at 2, 3, 4, 6, 8, 12, 24, 36, 48 and 72 hours.

The AUC from time zero extrapolated to infinity (AUCinf) will be calculate.

Group	Value	95% CI
50 mg PTI-125	2180	± 897
50 mg PTI-125 Placebo	NA	± NA
100 mg PTI-125	3260	± 1150
100 mg PTI-125 Placebo	NA	± NA
200 mg PTI-125	8250	± 1530
200 mg PTI-125 Placebo	NA	± NA

Percent Extrapolated of Area Under the Curve to Infinity (AUCextrap[%]). Primary · Blood samples will be drawn on Day 1 after dosing at 20, 40, and 60 minutes and at 2, 3, 4, 6, 8, 12, 24, 36, 48 and 72 hours.

The percentage of AUCinf based on extrapolation (AUCextrap\[%\]).

Group	Value	95% CI
50 mg PTI-125	0.410	± 0.187
50 mg PTI-125 Placebo	NA	± NA
100 mg PTI-125	0.151	± 0.0366
100 mg PTI-125 Placebo	NA	± NA
200 mg PTI-125	0.144	± 0.0156
200 mg PTI-125 Placebo	NA	± NA

Oral Clearance (Cl/F) Primary · Blood samples will be drawn on Day 1 after dosing at 20, 40, and 60 minutes and at 2, 3, 4, 6, 8, 12, 24, 36, 48 and 72 hours.

The apparent oral clearance will be calculated.

Group	Value	95% CI
50 mg PTI-125	25.7	± 8.42
50 mg PTI-125 Placebo	NA	± NA
100 mg PTI-125	33.0	± 8.16
100 mg PTI-125 Placebo	NA	± NA
200 mg PTI-125	24.9	± 4.53
200 mg PTI-125 Placebo	NA	± NA

Volume of Distribution (Vz/F) Primary · Blood samples will be drawn on Day 1 after dosing at 20, 40, and 60 minutes and at 2, 3, 4, 6, 8, 12, 24, 36, 48 and 72 hours.

Vz/F, apparent volume of distribution will be calculated.

Group	Value	95% CI
50 mg PTI-125	199	± 64.9
50 mg PTI-125 Placebo	NA	± NA
100 mg PTI-125	215	± 64.8
100 mg PTI-125 Placebo	NA	± NA
200 mg PTI-125	214	± 154
200 mg PTI-125 Placebo	NA	± NA

Adverse events — posted to ClinicalTrials.gov

Time frame: 7 days. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

50 mg PTI-125

Serious: 0/6 (0%)

Deaths: 0/6

50 mg PTI-125 Placebo

Serious: 0/2 (0%)

Deaths: 0/2

100 mg PTI-125

Serious: 0/6 (0%)

Deaths: 0/6

100 mg PTI-125 Placebo

Serious: 0/2 (0%)

Deaths: 0/2

200 mg PTI-125

Serious: 0/6 (0%)

Deaths: 0/6

200 mg PTI-125 Placebo

Serious: 0/2 (0%)

Deaths: 0/2

Other adverse events (3 terms — click to expand)

Reaction	System	50 mg PTI-125	50 mg PTI-125 Placebo	100 mg PTI-125	100 mg PTI-125 Placebo	200 mg PTI-125	200 mg PTI-125 Placebo
lightheadedness	Nervous system disorders	—	—	—	—	—	—
transient musculoskeletal wall pain	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—
Acne	Skin and subcutaneous tissue disorders	—	—	—	—	—	—

Data from ClinicalTrials.gov NCT03784300 adverse events section.

Sponsor's own description

A Phase I, Single Center, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose, Pharmacokinetic and Safety Study of PTl-125 in Healthy Volunteers

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

Role of actin cytoskeleton in podocytes.
Sever S. · · 2021 · cited 18× · PMID 33188449 · DOI 10.1007/s00467-020-04812-z
Promising candidates from drug clinical trials: Implications for clinical treatment of Alzheimer's disease in China.
Cao Y, Yu F, Lyu Y, Lu X. · · 2022 · cited 16× · PMID 36457865 · DOI 10.3389/fneur.2022.1034243
The potential of ARL4C and its-mediated genes in atherosclerosis and agent development.
Liu D, Wang J, Zhang S, Jiang H, et al · · 2025 · PMID 40176913 · DOI 10.3389/fphar.2025.1513340

Verify or expand the search:

Other recruiting trials for Alzheimer Disease, Early Onset

Currently open trials in the same condition.

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NCT06688968 — Neuropsychological Profiles and Musical Engagement in Parkinson's and Alzheimer's Disease · NA · recruiting
NCT06668610 — A Multifocal TDCS-EEG Protocol for Improving Symptoms of Mild Cognitive Impairment and Early Dementia · NA · recruiting
NCT06268886 — Study to Evaluate the Efficacy, Safety, and Tolerability of an Anti-MTBR Tau Monoclonal Antibody (BMS-986446) in Partici · Phase 2 · active not recruiting
NCT05983575 — A Pivotal Study of LIPUS-Brain in Patients With Early Alzheimer's Disease · Phase 3 · active not recruiting

Other Pain Therapeutics trials

Trials by the same sponsor.

NCT03748706 — PTI-125 for Mild-to-moderate Alzheimer's Disease Patients · Phase 2 · completed
NCT03475862 — Nasal Human Abuse Potential of PTI-821 · Phase 1 · completed

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT03784300 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Pain Therapeutics
Last refreshed: 10 May 2021

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03784300.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing

NCT03784300

Quick facts

Drugs / interventions tested

Conditions studied

Sponsor

Who can join

Results — posted to ClinicalTrials.gov

Adverse events — posted to ClinicalTrials.gov

Sponsor's own description

Publications & conference data

Related trials

Other recruiting trials for Alzheimer Disease, Early Onset

Other Pain Therapeutics trials

Verify against primary sources