A Study to Evaluate the Efficacy and Safey of PTP-001 (MOTYS™) in Knee Osteoarthritis Patients

NCT07219771 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: KOA-25-03
• Study Type: interventional
• Target: 260
• Locations: 2 countries
• Sites: 17

Brief Summary

This is a multicenter, prospective, randomized, double-blind, placebo-controlled, parallel-arm, Phase 3 study of intra-articular administration of PTP-001 (MOTYS) for the treatment of knee osteoarthritis. The purpose of the trial is to evaluate the efficacy, safety, and tolerability of a single intra-articular injection of PTP-001 compared to placebo over a 52-week period in participants with radiographic and symptomatic knee OA.

Conditions

Knee Osteoarthritis

Keywords

Osteoarthritis; Knee Osteoarthritis

Outcome Measures

Primary Outcomes (2)

• Proportion of participants meeting strict responder criteria for improvement in knee function.

  To qualify as a "strict responder for improvement in knee function", an improvement of ≥ 50% in WOMAC Function subscale score is required, with an absolute change ≥ 20 points in the score.

  6 months

• Proportion of participants meeting strict responder criteria for improvement in knee pain.

  To qualify as a "strict responder for improvement in knee pain", an improvement of ≥ 50% in WOMAC Pain subscale score is required, with an absolute change ≥ 20 points in the score.

  6 months

Secondary Outcomes (5)

• Proportion of participants meeting strict responder criteria for improvement in knee function.

  9 months and 12 months

• Proportion of participants meeting strict responder criteria for improvement in knee pain.

  9 months and 12 months

• Change from pretreatment Baseline in participant self-reported assessment of OA by PGA-OA score.

  6 months, 9 months, and 12 months

• Change from pretreatment Baseline in participant self-reported function of the treated knee as assessed by WOMAC Function subscale score.

  6 months, 9 months, and 12 months

• Change from pretreatment Baseline in participant self-reported pain at the treated knee as assessed by WOMAC Pain subscale score.

  6 months, 9 months, and 12 months

Study Arms (2)

PTP-001 200 mg

EXPERIMENTAL

A single intra-articular injection in the target knee of PTP-001 200 mg

Biological: PTP-01

Placebo / saline vehicle

PLACEBO COMPARATOR

A single intra-articular injection in the target knee of 4 mL of placebo control (0.9% sodium chloride injection, USP)

Other: Placebo control / saline vehicle

Interventions

PTP-01 BIOLOGICAL

Allogeneic human placental tissue particulate (PTP-001) is administered as a single intra-articular injection to the target knee after resuspension with saline.

Also known as: MOTYS™, allogeneic human placental tissue particulate

PTP-001 200 mg

Placebo control / saline vehicle OTHER

The placebo control, physiological saline (0.9% sodium chloride injection, USP), is administered as an intra-articular injection to the target knee.

Also known as: Normal saline, Physiological saline

Placebo / saline vehicle

Eligibility Criteria

• Age: 40 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males and females aged 40 to 80 years.
• Presenting with symptomatic knee OA with Kellgren-Lawrence (KL) radiographic classification of 2 or 3 (mild or moderate), as assessed by the central reading facility.
• Primary source of pain throughout the body is due to OA in the target knee.
• Target knee pain ≥ 20 and ≤ 40 out of 50 on the WOMAC® numerical rating scale (NRS) 3.1 pain questionnaire (sum of 5 questions) at Screening and Baseline.
• Onset of symptomatic OA of the target knee was at least 6 months prior to Screening.
• Insufficient, failed response, or intolerance to analgesics and / or non-steroidal anti-inflammatory drugs (NSAIDs), as reported by the participant.
• If female, must meet all of the following:
• Not breast feeding,
• Not planning to become pregnant during the study,
• Must abstain from ova / egg donation during the study,
• If of childbearing potential, must have a negative pregnancy test result within 72 hours prior to receiving the intra-articular injection, and must commit to the use of a highly effective form of birth control for at least 90 days after the injection.
• Willing to use acetaminophen as the only oral rescue (as needed) analgesic medication for target knee pain during the study.
• Willing to abstain from taking any illicit or unauthorized medications for treatment of OA or any other concurrent condition during the study.
• Willing to comply with study visit schedule and post-injection restrictions.
• Written informed consent is obtained from the participant.

You may not qualify if:

• Participant is non-ambulatory (unable to walk \> 50 feet / 15 meters without assistance).
• Clinically severe obesity as defined by the National Institutes of Health (body mass index ≥ 40 kg/m2) at Screening and / or Baseline.
• Contralateral (non-target) knee pain is ≥ 10 out of 50 on the WOMAC® NRS 3.1 pain questionnaire (sum of 5 questions) at Screening or at Baseline.
• At Baseline, difference between the first Baseline and second Baseline WOMAC pain scores is ≥ 3 for the target knee.
• Contralateral (non-target) knee pain is experienced for ≥ 14 days in the month.
• Use of any analgesia during the washout period (5 half-lives) at Screening or Baseline.
• Participation in any investigational study within 30 days (or 5 half-lives, whichever is longer) prior to Screening or planning to participate in any other investigational drug or device clinical trials within 30 days of study completion.
• Currently requires use of a lower extremity prosthesis and / or a structural knee brace (ie, a knee brace that contains hardware).
• Clinically significant effusion of the target knee at either the Screening or Baseline visits as determined by physical examination (eg, ballotable patella or positive bulge sign).
• Note: Participants presenting with effusion may be enrolled in the study after undergoing knee aspiration to remove excess fluid in the target joint.
• Severe (excessive) malalignment of the tibial-femoral axis assessed radiographically (by previous X-ray, rather than that performed for Kellgren-Lawrence assessment).
• Presence of active infection in the target knee or systemic infection requiring treatment within the 3 months prior to Screening.
• Clinical diagnosis of inflammatory arthritis (eg, rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, systemic lupus erythematosus, etc.) established by clinical history, examination, or serology.
• Participant is receiving, has received, or plans to receive any of the following therapies:
• Prior administration of hyaluronic acid, extended-release corticosteroid (eg, Zilretta®), platelet-rich plasma (PRP), or stem cell therapies by intra-articular injection(s) of the target knee within 6 months prior to Screening.

Sponsors & Collaborators

• Doron Therapeutics Inc.: lead
• Novotech (Australia) Pty Limited: collaborator

Study Sites (17)

Central Research Associates, LLC dba Flourish Research

Birmingham, Alabama, 35205, United States

RECRUITING

Horizon Clinical Research

La Mesa, California, 91942, United States

RECRUITING

Focus Clinical Research

West Hills, California, 91307, United States

RECRUITING

Conquest Research, LLC

Winter Park, Florida, 32789, United States

RECRUITING

Chicago Clinical Research Institute, Inc.

Chicago, Illinois, 60607, United States

RECRUITING

Lehigh Center for Clinical Research

Allentown, Pennsylvania, 18104, United States

RECRUITING

Coastal Carolina Research Center, LLD

North Charleston, South Carolina, 29405, United States

RECRUITING

JBR Clinical Research, LLC

Salt Lake City, Utah, 84107, United States

RECRUITING

Emeritus Research Sydney

Botany, New South Wales, 2019, Australia

RECRUITING

Genesis Research Services

Broadmeadow, New South Wales, 2292, Australia

RECRUITING

Royal North Shore Hospital

St Leonards, New South Wales, 2065, Australia

RECRUITING

University of the Sunshine Coast Clinical Trials, Birtinya

Birtinya, Queensland, 4575, Australia

RECRUITING

University of the Sunshine Coast Clinical Trials, Morayfield

Morayfield, Queensland, 4506, Australia

RECRUITING

University of the Sunshine Coast Clinical Trials, Noosa

Noosaville, Queensland, 4566, Australia

RECRUITING

Momentum Clinical Research Taringa

Taringa, Queensland, 4068, Australia

RECRUITING

Momentum Clinical Research Wellers Hill

Tarragindi, Queensland, 4121, Australia

TERMINATED

Momentum Clinical Research Sunshine

St Albans, Victoria, 3021, Australia

RECRUITING

Related Publications (2)

• Flannery CR, Buddin KE, Begum L, Nasert MA, Catalfamo B, Semler EJ, Fortier LA. Composition and Bioactivity of a Placental Tissue Particulate (PTP-001) Indicate Greater Potential than Platelet-Rich Plasma for the Treatment of Osteoarthritis. Cartilage. 2023 Dec;14(4):467-472. doi: 10.1177/19476035231159748. Epub 2023 Mar 13.

  PMID: 36912174 RESULT

• Flannery CR, Seaman SA, Buddin KE, Nasert MA, Semler EJ, Kelley KL, Long M, Favret J, Pavesio A, Loeser RF. A novel placental tissue biologic, PTP-001, inhibits inflammatory and catabolic responses in vitro and prevents pain and cartilage degeneration in a rat model of osteoarthritis. Osteoarthritis Cartilage. 2021 Aug;29(8):1203-1212. doi: 10.1016/j.joca.2021.03.022. Epub 2021 May 20.

  PMID: 34023528 RESULT

Related Links

• Related Info
• Related Info

MeSH Terms

Conditions

Osteoarthritis, Knee; Osteoarthritis

Interventions

Sodium Chloride; Saline Solution

Condition Hierarchy (Ancestors)

Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases

Intervention Hierarchy (Ancestors)

Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds; Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Study Officials

• Professor David Hunter

  Chair of Rheumatology at University of Sydney and Royal North Shore Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Mary Kathryn Kottke

CONTACT

919-355-4630; mk.kottke@dorontherapeutics.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: This is a double-blinded trial, in which neither participant nor investigator will know the content of the investigational product. Additionally, unblinded personnel that prepare the syringe and administer the injection must not be involved in any other operational aspects of the trial.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 21, 2025
• First Posted: October 22, 2025
• Study Start: October 14, 2025
• Primary Completion (Estimated): April 1, 2027
• Study Completion (Estimated): October 1, 2027
• Last Updated: April 29, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (8); AU (9)