Safety, Tolerability, and Pharmacokinetics of ASCT-83 in Healthy Adults

NCT07363395 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 3 other identifiers: ASCT-83-1011R44NS145934-01CDMRP-HT94252510912
• Study Type: interventional
• Target: 72
• Locations: 0 countries
• Sites: N/A

Brief Summary

The goal of this clinical trial is to learn if ASCT-83 is safe and well-tolerated and measure how ASCT-83 is absorbed, distributed, and eliminated from the body over time. The study will be conducted in healthy adults. The main questions this study will answer are:

• Is ASCT-83 safe at clinical doses?
• Does ASCT-83 have side effects at clinical doses?
• How is ASCT-83 absorbed, distributed, and eliminated from the body? Researchers will compare ASCT-83 to a placebo (a look-alike substance that contains no drug). The study has two parts: participants in Part 1 will receive only one dose of ASCT-83 or placebo participants in Part 2 will receive one dose of ASCT-83 or placebo a day for 7 days. Participants will visit the clinic to take ASCT-83 or placebo, to receive health checkups and undergo health tests. Participants in Part 1 will spend 5 days/4 nights in the clinic, participants in Part 2 will spend 11 days/10 nights in the clinic. In addition, there will be up to 3 outpatient visits. The results of this study will help determine safe dose levels and support the design of future clinical trials.

Conditions

Neuropathic Pain

Keywords

Neuropathic Pain; Nerve Regeneration; Analgesic; CTDSP1 Inhibitor; REST (RE1-silencing transcription factor); Macrocyclic Peptide; Target Engagement; First-in-Human (FIH); SAD/MAD; Healthy Volunteers; Pharmacokinetics (PK); Diabetic Peripheral Neuropathy (DPN); Peripheral Nerve Injury (PNI)

Outcome Measures

Primary Outcomes (4)

• Incidence, severity and dose relationships of Treatment-Emergent Adverse Events (TEAEs) and non-TEAEs, Adverse Events of Special Interest (AESIs), Serious Adverse Events (SAEs), premature treatment and study discontinuation due to Adverse Events (AEs)

  The investigator will assess and record information pertaining to the AE, which includes but is not limited to the following: date of onset, event diagnosis (when known) and/or signs and symptoms, duration, severity, seriousness (i.e. serious adverse event or not serious), expected/unexpected, and relationship to the study therapy or procedure, action(s) taken, and outcome. Severity grading: 1 Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. 2 Moderate; minimal, local, or noninvasive intervention indicated; limiting age-appropriate instrumental ADL. 3 Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL. 4 Life-threatening consequences; urgent intervention indicated. 5 Death related to AE.

  Single dose part of the study: from enrollment to 30-32 days after the end of treatment. Multiple dose part of the study: from enrollment to 36-38 days after the end of treatment.

• Incidence and severity of changes in clinical safety parameters, including vital signs (including body weight), laboratory (including markers of inflammation), ECG results, and skin assessments (including injection site reactions).

  Single dose part of the study: from enrollment to 30-32 days after the end of treatment. Multiple dose part of the study: from enrollment to 36-38 days after the end of treatment.

• Incidence of suicidality

  Suicide risk will be assessed through a questionnaire (Columbia-Suicide Severity Rating Scale (C-SSRS)). A score ≥4 is considered suicidal ideation.

  Single dose part of the study: from enrollment to 30-32 days after the end of treatment. Multiple dose part of the study: from enrollment to 36-38 days after the end of treatment.

• Incidence and type of abnormal skin assessments, including injection-site reactions.

  The FDA Toxicity Grading Scale (TGS) for injection/vaccine sites will be used: Mild/Grade 1, Moderate/Grade 2, Severe/Grade 3, Life-threatening/Grade 4.

  Single dose part of the study: from enrollment to 30-32 days after the end of treatment. Multiple dose part of the study: from enrollment to 36-38 days after the end of treatment.

Secondary Outcomes (11)

• Area under the concentration-time curve from time zero to infinity (AUC0-inf) of ASCT-83

  SAD: pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24, 36, 48, 72 hours post-dose. MAD: pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24 hours post-dose on Day 1; pre-dose on Days 3 to 7; 24, 36, 48, and 72 hours after the final dose on Day 7

• Area under the concentration-time curve from time zero to 24 h (AUC0-24h) of ASCT-83

  SAD: pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24, 36, 48, 72 hours post-dose. MAD: pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24 hours post-dose on Day 1; pre-dose on Days 3 to 7; 24, 36, 48, and 72 hours after the final dose on Day 7

• Peak plasma concentration (Cmax) of ASCT-83

  SAD: pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24, 36, 48, 72 hours post-dose. MAD: pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24 hours post-dose on Day 1; pre-dose on Days 3 to 7; 24, 36, 48, and 72 hours after the final dose on Day 7

• Time to reach maximum plasma concentration (Tmax) of ASCT-83

  SAD: pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24, 36, 48, 72 hours post-dose. MAD: pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24 hours post-dose on Day 1; pre-dose on Days 3 to 7; 24, 36, 48, and 72 hours after the final dose on Day 7

• Terminal elimination half-life (t½) of ASCT-83

  SAD: pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24, 36, 48, 72 hours post-dose. MAD: pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 24 hours post-dose on Day 1; pre-dose on Days 3 to 7; 24, 36, 48, and 72 hours after the final dose on Day 7

Other Outcomes (2)

• Quantification of Pharmacodynamic (PD) Biomarkers of Target Engagement: SMAD1/5/8

  SAD: pre-dose on Day 1 and 24 hours after dosing (Day 2). MAD: pre-dose on Day 1 and 4 hours after the third and last dosing.

• Quantification of Pharmacodynamic (PD) Biomarkers of Target Engagement: BDNF

  SAD: pre-dose on Day 1 and 24 hours after dosing (Day 2). MAD: pre-dose on Day 1 and 4 hours after the third and last dosing.

Study Arms (11)

SAD-Placebo

PLACEBO COMPARATOR

One dose of placebo solution administered as two subcutaneous injections of 1.5 ml (second injection within 10 minutes of the first)

Drug: Placebo

SAD, ASCT-83 dose 1

EXPERIMENTAL

Total dose: 0.5 mg ASCT-83. Administration: two 1.5 mL subcutaneous injections of 0.17 mg/mL (second injection within 10 minutes of the first)

Drug: ASCT-83

SAD, ASCT-83 dose 2

EXPERIMENTAL

Total dose: 1 mg ASCT-83. Administration: two 1.5 mL subcutaneous injections of 0.33 mg/mL (second injection within 10 minutes of the first)

Drug: ASCT-83

SAD-ASCT dose 3

EXPERIMENTAL

Total dose: 2 mg ASCT-83. Administration: two 1.5 mL subcutaneous injections of 0.67 mg/mL (second injection within 10 minutes of the first)

Drug: ASCT-83

SAD-ASCT dose 4

EXPERIMENTAL

Total dose: 4 mg ASCT-83. Administration: two 1.5 mL subcutaneous injections of 1.33 mg/mL (second injection within 10 minutes of the first)

Drug: ASCT-83

SAD, ASCT-83 dose 5

EXPERIMENTAL

Total dose: 7.5 mg ASCT-83. Administration: two 1.5 mL subcutaneous injections of 2.5 mg/mL (second injection within 10 minutes of the first)

Drug: ASCT-83

SAD, ASCT-83 dose 6

EXPERIMENTAL

Total dose: 10 mg ASCT-83. Administration: two 1.5 mL subcutaneous injections of 3.33 mg/mL (second injection within 10 minutes of the first)

Drug: ASCT-83

MAD-Placebo

PLACEBO COMPARATOR

Two daily subcutaneous injections of 1.5 ml placebo for 7 days, with the two daily injections given max 10 minutes apart.

Drug: Placebo

MAD, ASCT-83 dose 1

EXPERIMENTAL

Two daily subcutaneous injections of 1.5 ml of ASCT-83 for 7 days, with the two daily injections given max 10 minutes apart. Dose to be determined.

Drug: ASCT-83

MAD, ASCT-83 dose 2

EXPERIMENTAL

Two daily subcutaneous injections of 1.5 ml of ASCT-83 for 7 days, with the two daily injections given max 10 minutes apart. Dose to be determined.

Drug: ASCT-83

MAD, ASCT-83 dose 3

EXPERIMENTAL

Two daily subcutaneous injections of 1.5 ml of ASCT-83 for 7 days, with the two daily injections given max 10 minutes apart. Dose to be determined.

Drug: ASCT-83

Interventions

ASCT-83 DRUG

ASCT-83 is a 23-amino acid, macrocyclic peptide with a molecular weight of approximately 3 kilodaltons under development for the treatment of neuropathic pain (NeP). ASCT-83 25 mg/mL sterile solution for injection consists of 25 mg of ASCT-83 drug substance dissolved in 1 mL of histidine buffer. The product also contains mannitol to adjust the osmolarity of the final product. The product is stored at -20°C prior to dilution to achieve the target dose.

MAD, ASCT-83 dose 1; MAD, ASCT-83 dose 2; MAD, ASCT-83 dose 3; SAD, ASCT-83 dose 1; SAD, ASCT-83 dose 2; SAD, ASCT-83 dose 5; SAD, ASCT-83 dose 6; SAD-ASCT dose 3; SAD-ASCT dose 4

Placebo DRUG

Placebo solution contaninig the same histidine buffer and mannitol concentrations as ASCT-83, with polyoxyl 35 castor oil (0.1% w/v) added to match the appearance of the active solution.

MAD-Placebo; SAD-Placebo

Eligibility Criteria

• Age: 18 Years - 64 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• To participate in the study, you must:
• be medically healthy based on medical history, physical exam, labs, and ECG
• be 18-64 years (inclusive)
• at screening, have a body mass index (BMI) \> 19 to \< 35 kg/m²
• be willing and able to understand and voluntarily sign an informed consent
• Female participants of childbearing potential (women who can become pregnant) must:
• agree to follow the study contraceptive requirements from screening through 28 days after the final dose of study drug.
• agree to undergo pregnancy testing, including a negative pregnancy test at screening and at admission.
• agree not to donate ova (eggs) from screening through 28 days after the final dose of study drug.
• Male participants must:
• have a vasectomy greater than 6 months prior to screening or be willing to follow the contraceptive requirements from screening through 90 days after final study drug administration
• agree to be willing to abstain from sperm donation from screening through 90 days after final study drug administration

You may not qualify if:

• You cannot participate in the study if you:
• are a study site staff, Alcamena employee or immediate family member, or have participated in another clinical trial within 30 days or 5 half-lives of a prior investigational product.
• have evidence of liver disease or abnormal liver tests (ALT/AST, Alk Phos, GGT, or bilirubin \> ULN); positive hepatitis B or C serology (people with Gilbert syndrome may be included).
• have a history of kidney disease, protein in the urine or reduced kidney function based on screening blood tests.
• have a history of cancer with active disease, suspected relapse, treatment within 6 months, or ongoing therapy affecting immune, liver, or kidney function.
• have a history of heart disease, including abnormal heart rhythm, heart attack, long QT syndrome, or abnormal heart rhythm findings on screening ECG, or a family history of sudden unexplained death.
• are currently receiving medications that affect or suppress the immune system, including steroids given by any route.
• have a known autoimmune condition (such as lupus, rheumatoid arthritis, sarcoidosis, or vitiligo), even if it is not being treated.
• have a positive HIV test at screening or a history of immune deficiency.
• have abnormal blood tests suggesting ongoing inflammation
• have an active infection or is currently being treated for an infection.
• have a skin condition that could interfere with study skin assessments (such as psoriasis).
• are allergic or sensitive to the study drug or its ingredients.
• have a history of alcohol or drug abuse or addiction within the past five years, have a positive drug or alcohol test at screening, or have smoked within one month before screening.
• have a serious mental health condition, such as major depression, schizophrenia, severe anxiety, eating disorders, severe attention deficit disorder, personality disorders, or suicidal thoughts or behavior within the past five years that could affect safety or study participation.

Sponsors & Collaborators

• Alcamena Stem Cell Therapeutics: lead
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator
• Congressionally Directed Medical Research Programs: collaborator

MeSH Terms

Conditions

Neuralgia; Peripheral Nerve Injuries

Condition Hierarchy (Ancestors)

Peripheral Nervous System Diseases; Neuromuscular Diseases; Nervous System Diseases; Pain; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Trauma, Nervous System; Wounds and Injuries

Study Officials

• Edmund Nesti, PhD

  Alcamena Stem Cell Therapeutics

  STUDY DIRECTOR

• Trisha Shamp, PhD, PA-C, ECG-BA, CVPA-BC

  Nucleus Network

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Alissa Anderson

CONTACT

651-583-8518; a.anderson@nucleusnetwork.com

Kate Jankins

CONTACT

+442089614130; kjenkins@hmrlondon.com

Study Design

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

Study Record Dates

• First Submitted: January 9, 2026
• First Posted: January 23, 2026
• Study Start: January 1, 2026
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: January 23, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share