Phase 1 Single-blind Placebo-controlled Dose Escalation Safety Study of AD-NP1

NCT07762001 | Active Not Recruiting Phase 1 FDA Drug

• 2 other identifiers: 25-1584PR250528
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 1

Brief Summary

This is a Phase 1 single-blind, placebo-controlled, dose-escalation study designed to evaluate the safety of AD-NP1, a humanized monoclonal antibody targeting human ectonucleotide pyrophosphatase/phosphodiesterase-1 (ENPP1). The study aims to assess the maximum tolerated dose (MTD), pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of AD-NP1 in healthy volunteers. Participants will be randomized to receive either AD-NP1 or placebo, with safety and efficacy monitored in accordance with Good Clinical Practice (GCP) guidelines. The study also seeks to explore changes in health-related quality of life (HRQOL) during the trial.

Conditions

Maximum Tolerated Dose; Dose Escalation

Keywords

Healthy volunteers; Dose toxicity; Monoclonal antibody

Outcome Measures

Primary Outcomes (1)

• Safety and tolerability of escalating doses of a single IV dose of AD-NP1

  Assessed by the incidence of treatment-related Adverse Events (AEs) using the Common Terminology Criteria for Adverse Events (CTCAE) v5.0.

  Day of infusion (Day 0) to 28 days post-dose

Secondary Outcomes (13)

• Maximum Tolerated Dose of AD-NP1

  Day of infusion (Day 0) to 28 days post-dose

• Maximum Plasma Concentration (Cmax) as a pharmacokinetics (PK) parameter

  Baseline (pre-dose) up to Day 60 post-dose

• Time to Maximum Plasma Concentration (Tmax) as a pharmacokinetics (PK) parameter

  Baseline (pre-dose) up to Day 60 post-dose

• Apparent Volume of Distribution (Vd) as a pharmacokinetics (PK) parameter

  Baseline (pre-dose) up to Day 60 post-dose

• Area Under the Plasma Concentration-Time Curve (AUC) as a pharmacokinetics (PK) parameter

  Baseline (Pre-dose), 30 minutes, 1, 2, 4, 6 hours, and Days 1, 3, 7, 14, 28, 60 post-dose.

Study Arms (8)

Cohort 1 - AD-NP1 (30mg/kg)

EXPERIMENTAL

Subjects receive a single intravenous dose of AD-NP1 at 30 mg/kg

Drug: AD-NP1

Cohort 1 - Placebo

PLACEBO COMPARATOR

Subjects receive a single intravenous dose of placebo matched in volume to the 30 mg/kg dose of AD-NP1

Drug: Placebo

Cohort 2 - AD-NP1 (40 mg/kg)

EXPERIMENTAL

Subjects receive a single intravenous dose of AD-NP1 at 40 mg/kg

Drug: AD-NP1

Cohort 2 - Placebo

PLACEBO COMPARATOR

Subjects receive a single intravenous dose of placebo matched in volume to the 40 mg/kg dose of AD-NP1

Drug: Placebo

Cohort 3 - AD-NP1 (60 mg/kg)

EXPERIMENTAL

Subjects receive a single intravenous dose of AD-NP1 at 60 mg/kg

Drug: AD-NP1

Cohort 3 - Placebo

PLACEBO COMPARATOR

Subjects receive a single intravenous dose of placebo matched in volume to the 60 mg/kg dose of AD-NP1

Drug: Placebo

Cohort 4 - AD-NP1 (100 mg/kg)

EXPERIMENTAL

Subjects receive a single intravenous dose of AD-NP1 at 100 mg/kg

Drug: AD-NP1

Cohort 4 - Placebo

PLACEBO COMPARATOR

Subjects receive a single intravenous dose of placebo matched in volume to the 100 mg/kg dose of AD-NP1

Drug: Placebo

Interventions

AD-NP1 DRUG

AD-NP1 is a humanized monoclonal antibody targeting the catalytic domain of human ENPP1. It is administered intravenously at escalating doses (30 mg/kg, 40 mg/kg, 60 mg/kg, and 100 mg/kg).

Cohort 1 - AD-NP1 (30mg/kg); Cohort 2 - AD-NP1 (40 mg/kg); Cohort 3 - AD-NP1 (60 mg/kg); Cohort 4 - AD-NP1 (100 mg/kg)

Placebo DRUG

Placebo is a normal saline solution administered intravenously in volumes matched to the corresponding AD-NP1 doses (30 mg/kg, 40 mg/kg, 60 mg/kg, and 100 mg/kg)

Cohort 1 - Placebo; Cohort 2 - Placebo; Cohort 3 - Placebo; Cohort 4 - Placebo

Eligibility Criteria

• Age: 21 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Body weight ≤ 90kg
• Ability to understand and the willingness to sign a written informed consent document
• Study subject must have read, understood, and provided written informed consent and HIPAA authorization after the nature of the study has been fully explained

You may not qualify if:

• No use of any tobacco products for at least 6 months
• Woman/women of childbearing potential (WOCBP) must agree not to become pregnant from the time of study enrollment until at least 6 months after the completion of the monoclonal antibody infusion. If a WOCBP is sexually active and has no history of hysterectomy or tubal ligation, she must agree to use hormonal or barrier birth control with spermicidal gel
• Sexually active male subjects must use a barrier method of contraception during the study
• Screening laboratory values must meet the following criteria:
• WBC (\>3,000 - \<11,000/mm\^3)
• Platelets (\>100,000/mm\^3)
• Hemoglobin (\>10.5 gm/dl)
• Creatinine (\<1.1 x upper limit of normal \[ULN\])
• BUN (\<1.25 x ULN)
• AST (\<1.1 x ULN)
• ALT (\<1.1 x ULN)
• Alkaline Phosphatase (\<1.1 x ULN)
• Bilirubin (\<1.1 x ULN)
• Glucose-non-fasting (\> 60 mg/dl and \< 115 mg/dl)
• Human immunodeficiency virus (HIV)-infected individuals on effective antiretroviral therapy with undetectable viral load within 6 months are eligible for this trial

Sponsors & Collaborators

• Arjun Deb, MD: lead
• United States Department of Defense: collaborator

Study Sites (1)

UCLA Clinical and Translational Research Center

Los Angeles, California, 90095, United States

Location

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: BASIC SCIENCE
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: IND Holder and Sponsor

Study Record Dates

• First Submitted: August 6, 2026
• First Posted: August 13, 2026
• Study Start: June 2, 2026
• Primary Completion (Estimated): January 1, 2028
• Study Completion (Estimated): May 1, 2028
• Last Updated: August 13, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)