A First-in-Human Study of XSTEM-OA in Patients With Knee Osteoarthritis

A First-in-Human Open-Label Phase 1/2a Study to Evaluate Safety, Tolerability and Efficacy of a Single Intra-Articular Injection of Allogeneic Mesenchymal Stem Cells Selected for Integrin α10β1 (XSTEM-OA) in Adult Patients With Symptomatic Knee Osteoarthritis

The aim of the study is to assess safety, tolerability and preliminary efficacy of XSTEM-OA when administered as a single intra-articular injection to patients with symptomatic knee osteoarthritis. The study is divided into two parts; a dose escalation (Part A) and an optional dose expansion part (Part: B). Three dose levels are planned to be evaluated in Part A and selected dose level(s) may be further expanded in Part B.

Study Overview

• Status: Completed
• Conditions: Osteoarthritis, Knee
• Intervention / Treatment: Biological: XSTEM-OA

• Study Type: Interventional
• Enrollment (Actual): 24
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Broadmeadow, New South Wales, Australia, 2292
      • Genesis Research Services
  • Victoria
    • Camberwell, Victoria, Australia, 3124
      • Emeritus Research

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 40 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Major inclusion criteria:

• Aged ≥40 and ≤75 years at Screening
• Radiological evidence of predominantly medial osteoarthritis in the knee, uni- or bilateral, of KL grade II to III
• Moderate to severe pain associated with osteoarthritis in the knee as measured by a VAS pain score of ≥35 and ≤90 mm

Major exclusion criteria:

• Body mass index (BMI) of ≥35 at Screening
• Ongoing signs or symptoms of systemic or local infection
• Known knee infection in the study knee within 6 months of Screening
• History of clinically relevant concomitant joint disease, clinically-relevant knee deformities, or any clinically significant medical history of the ligament, or realignment surgery or joint replacement surgery
• Medical history of any autoimmune disease
• History of surgery in the study knee that occured within 6 months of Screening
• Treatment with immunosuppressive therapy (systemic or local) or any medication affecting the bone or cartilage metabolism within 6 months of Screening
• Patients who are immunocompromised as caused by a disease

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: XSTEM-OA; Single intra-articular injection of XSTEM-OA	Biological: XSTEM-OA; XSTEM-OA is an allogeneic, adipose tissue-derived, integrin alpha10beta1-selected and expanded mesenchymal stem cell (MSC) product for the treatment of osteoarthritis.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety and tolerability: 12-lead electrocardiogram (ECG)	Change in 12-lead electrocardiograms (ECGs) compared to baseline.	From start of study until 18 months after injection
Safety and tolerability: Laboratory examinations	Changes in laboratory tests compared to baseline. The clinical laboratory tests include haematology, chemistry, coagulation and urinalysis.	From start of study until 18 months after injection
Safety and tolerability: Physical examinations	Changes in physical examination compared to baseline.	From start of study until 18 months after injection
Safety and tolerability: Vital signs	Changes in vital signs compared to baseline. Vital signs include blood pressure, pulse rate, respiratory rate and oral body temperature.	From start of study until 18 months after injection
Safety and tolerability: Adverse events (AEs)	Incidence, nature and severity of treatment-related AEs assessed by the Common Terminology Criteria for Adverse Events (CTCAE)	From start of study until 24 months after injection

Secondary Outcome Measures

Outcome Measure	Time Frame
Preliminary efficacy: Changes in Visual Analogue Scale (VAS) for pain compared to baseline	From start of study until 18 months after injection
Preliminary efficacy: Changes in Knee injury and Osteoarthritis Outcome Score (KOOS) compared to baseline	From start of study until 18 months after injection
Preliminary efficacy: Change in EuroQoL 5 Dimension 5 Level test (EQ-5D-5L) compared to baseline	From start of study until 18 months after injection
Preliminary efficacy: Change in 40 m fast paced walk test compared to baseline	From start of study until 18 months after injection
Preliminary efficacy: Change in whole knee health compared to baseline, based on MRI assessment by the MRI Osteoarthritis Knee Score (MOAKS)	From start of study until 18 months after injection
Preliminary efficacy: Changes in joint space narrowing based on x-ray compared to baseline	From start of study until 18 months after injection
Preliminary efficacy: Change in cartilage structure and morphology compared to baseline, based on MRI assessment of cartilage volume and composition	From start of study until 18 months after injection
Preliminary efficacy: Change in inflammatory activity compared to baseline, based on MRI assessment of synovitis	From start of study until 18 months after injection

Other Outcome Measures

Outcome Measure	Time Frame
Exploratory outcome: Change in Visual Analogue Scale (VAS) for pain compared to baseline	24 months after injection
Exploratory outcome: Change in KOOS compared to baseline	24 months after injection
Exploratory outcome: Change in EQ-5D-5L compared to baseline	24 months after injection
Exploratory outcome: Change in 40 m fast paced walk test compared to baseline	24 months after injection
Exploratory outcome: Change in cartilage structure and morphology compared to baseline, based on MRI assessment of cartilage volume and composition	24 months after injection
Exploratory outcome: Change in whole knee health compared to baseline, based on MRI	24 months after injection
Exploratory outcome: Change in inflammatory activity compared to baseline, based on MRI assessment of synovitis	24 months after injection
Exploratory outcome: Change in joint space narrowing based on x-ray compared to baseline	24 months after injection

Collaborators and Investigators

• Sponsor: Xindu Pty Ltd
• Collaborators: Xintela AB
• Investigators: Study Director: Per Norlén, MD, PhD, Xintela AB; Principal Investigator: Stephen Hall, Dr, Emeritus Research

Study record dates

Study Major Dates

• Study Start (Actual): June 22, 2022
• Primary Completion (Actual): June 18, 2025
• Study Completion (Actual): June 18, 2025

Study Registration Dates

• First Submitted: April 4, 2022
• First Submitted That Met QC Criteria: April 19, 2022
• First Posted (Actual): April 25, 2022

Study Record Updates

• Last Update Posted (Estimated): August 26, 2025
• Last Update Submitted That Met QC Criteria: August 25, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Arthritis; Joint Diseases; Rheumatic Diseases; Osteoarthritis; Osteoarthritis, Knee
• Other Study ID Numbers: XIN-XSTEM-101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No