Phase 1b Clinical Trial to Evaluate PEP and EUFLEXXA for Knee Osteoarthritis (KOA)

NCT06463132 | Not Yet Recruiting Phase 1 DMC FDA Drug FDA Device

• 1 other identifier: PRO-00126
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

Evaluate the safety and exploratory efficacy of single intra-articular injections of PEP reconstituted with 0.9% Normal Saline at a low dose (one vial PEP) and high dose (two vials PEP), with and without EUFLEXXA, for the treatment of Knee Osteoarthritis

Conditions

Knee Osteoarthritis

Keywords

Knee Osteoarthritis; Exosome; Osteoarthritis; Joint Disease; Hyaluronic Acid; Extracellular Vesicle; Musculoskeletal Diseases; Arthritis; Randomized Clinical Trial; Rheumatic Diseases; Osteoarthritis, Knee; Biologic

Outcome Measures

Primary Outcomes (2)

• Primary Safety Follow up 90 Days post injection occurrence of DLTs

  The safety of a single intra-articular injection of PEP, with or without EUFLEXXA, through the initial 90-day treatment period as assessed by the occurrence of DLTs with severity grading based on the National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI CTCAE).

  First 90 days

• Long Term Safety Follow-Up Occurrence of DLTs From Day 91 to Day 365

  The long-term safety after treatment with a single intra-articular injection of PEP, with or without EUFLEXXA as assessed from the incidence of DLTs during the Day 91 through Day 365 follow up period.

  Day 365

Secondary Outcomes (2)

• Exploratory Endpoints of Efficacy-Radiographic Response

  Day 365

• Exploratory Endpoints of Efficacy-Participant-Reported responses:

  Day 365

Study Arms (4)

1A PEP Low Dose

EXPERIMENTAL

1A PEP Low Dose in Saline

Drug: PEP

1B PEP High Dose

EXPERIMENTAL

1B PEP High Dose in Saline

Drug: PEP

2a PEP-EUFLEXXA Low Dose

EXPERIMENTAL

2A PEP-EUFLEXXA Low Dose

Combination Product: PEP/Euflexxa

2b PEP-EUFLEXXA High Dose

EXPERIMENTAL

2B PEP-EUFLEXXA High Dose

Combination Product: PEP/Euflexxa

Interventions

PEP/Euflexxa COMBINATION_PRODUCT

Euflexxa (PMA: P010029)

2a PEP-EUFLEXXA Low Dose; 2b PEP-EUFLEXXA High Dose

PEP DRUG

PEP (Purified Exosome Product)

1A PEP Low Dose; 1B PEP High Dose

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age: Adults aged 18-90 years of age
• Type of Participant and Disease Characteristics:
• Diagnosis of unilateral or bilateral symptomatic KOA of at least 6 months duration (meets American College of Rheumatology/Arthritis \[ACR\] criteria)
• Daily knee pain in the more symptomatic knee of ≥ 30 and ≤ 80 on a 100-point VAS for greater than 6 months
• Kellgren Lawrence Grade 2 to 3 osteoarthritis in the more symptomatic knee based on standard knee radiographs
• Failed conservative management including at least 2 of the following:
• Lack of improvement with attempted weight loss in the past year if body mass index (BMI) \> 30 kg/m2;
• Lack of response to a 4-week trial in the past year of oral acetaminophen or NSAIDs taken as needed;
• Lack of response to a 4-week trial in the past year of topical NSAIDs or capsaicin applied as needed;
• Lack of improvement after injection therapy with cortisone, PRP or hyaluronic acid;
• Lack of improvement after a 4-week course of physical therapy in the past year; or
• Lack of improvement after a 4-week trial of quadriceps strengthening home exercise program in the past year.
• Requesting injection therapy for pain management
• Contraceptive use by participants and their partners should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

You may not qualify if:

• Signed informed consent as described in Appendix 1 (Section 10.1) which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
• Ability to comply with protocol.
• Steroid or viscosupplement injection in the planned treatment knee(s) within the past 3 months.
• PRP injection in the planned treatment knee(s) within the past 6 months.
• History of documented allergy to intra-articular EUFLEXXA.
• History of cancer including melanoma (with the exception of localized skin cancer) in the past 2 years.
• HIV positive participants.
• BMI \> 40 kg/m2.
• Arthroscopic debridement in the planned treatment knee(s) in the last 6 months.
• Cartilage restoration procedure in the planned treatment knee(s) in the last 5 years.
• History of gout or pseudogout.
• History of or evidence of active rheumatologic disease, severe peripheral neuropathy, clinically evident cardiac or respiratory disease that interferes with functional status.
• Poorly controlled diabetes defined as a glycated hemoglobin (HbA1c) concentration of ≥ 8.0%.
• Currently taking any cancer treatment regimen (including aromatase inhibitors).
• Calcium pyrophosphate deposition disease (CPPD) evident on X-ray.

Sponsors & Collaborators

• Rion Inc.: lead
• Caidya Clinical Research Organization: collaborator

Study Sites (1)

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

MeSH Terms

Conditions

Osteoarthritis, Knee; Osteoarthritis; Joint Diseases; Musculoskeletal Diseases; Arthritis; Rheumatic Diseases

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases

Central Study Contacts

Shariq Khan, M.S.

CONTACT

847-702-4063; khan@riontx.com

Maureen Merrifield, Ph.D.

CONTACT

2819146228; merrifield@riontx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Arm 1A, PEP Low Dose Arm 1B, PEP High Dose Arm 2A, PEP-EUFLEXXA Low Dose Arm 2B, PEP-EUFLEXXA High Dose

Study Record Dates

• First Submitted: June 7, 2024
• First Posted: June 17, 2024
• Study Start: November 1, 2024
• Primary Completion: November 1, 2025
• Study Completion: November 1, 2025
• Last Updated: June 17, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)