This is a first-in-human, single-arm, open-label, dose-escalation, exploratory Phase I clinical trial designed to evaluate the safety, tolerability, and preliminary efficacy of local injection of mesenchymal stem cell-derived exosomes for non-traumatic osteonecrosis of the femoral head (ONFH).
A total of 15 treatment-naïve patients with non-traumatic, non-septic ONFH at ARCO stages I-IIIA, treated at the outpatient clinic of our hospital between June 2026 and June 2027, will be enrolled. Under ultrasound guidance, exosomes will be administered via an anterior approach through a single intra-articular injection into the hip joint. Three dose cohorts will be evaluated: \\(4 \\times 10\^{10}\\), \\(1.2 \\times 10\^{11}\\), and \\(3.6 \\times 10\^{11}\\) particles per administration per hip. The low-dose cohort will include 3 patients, while the medium- and high-dose cohorts will each include 6 patients. The dose-limiting toxicity observation period will extend from the first administration to 14 days after the final administration.
Exosomes will be administered once every 2 weeks at a volume of 3 mL per injection. Three administrations will constitute one treatment course, with a total treatment duration of 4 weeks. The primary endpoints are safety outcomes, including the incidence of adverse events, serious adverse events, and dose-limiting toxicities after administration. Secondary exploratory efficacy endpoints include changes in the Visual Analog Scale (VAS) pain score, Harris Hip Score (HHS), and radiological findings of the femoral head. The follow-up period will be 12 months.
Age 18-70 years, inclusive, with no restriction on sex.
A diagnosis of non-traumatic osteonecrosis of the femoral head (ONFH) confirmed by clinical manifestations and imaging findings, with ARCO stage II or IIIA disease. Patients with advanced femoral head collapse (ARCO stage IIIB or IV) will be excluded.
The target hip must be treatment-naïve, defined as no prior hip-specific interventional or injection-based treatment for the affected side.
Ability to understand and voluntarily provide written informed consent, with an expectation of completing long-term follow-up.
Presence of an active infection in the target joint (e.g., septic arthritis or infectious osteomyelitis), or skin disruption, infection, or other abnormalities at the intended injection site.
Receipt of intra-articular injection therapy in the affected hip within 3 months before screening, including corticosteroids or sodium hyaluronate.
Presence of any active systemic autoimmune disease (e.g., rheumatoid arthritis or systemic lupus erythematosus) or a primary or secondary immunodeficiency disorder.
History of malignancy.
Sequential
Exosome Intervention Group
Biological/VaccineParticipants will undergo dose escalation starting from the lowest dose, with sequential dose levels of 4×10\*10、1.2×10\*11、3.6×10\*11particles per administration per hip.
Number of participants with dose-limiting toxicities (DLTs)
Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs), graded by CTCAE v6.0
Number of participants with suspected unexpected serious adverse reactions (SUSARs)
Change from baseline in Visual Analogue Scale (VAS) pain score
The Visual Analogue Scale (VAS) is a 0-to-10-point scale for pain assessment. A score of 0 represents no pain, 1-3 indicates mild pain, 4-6 indicates moderate pain, and 7-10 represents severe pain, with 10 defined as the worst imaginable pain.
Change from baseline in Harris Hip Score
The Harris Hip Score (HHS) is a clinician-rated 0-to-100-point scale for hip-related function and disability, covering four domains: pain, function, range of motion, and deformity. Higher scores represent better hip performance. Scores of 90-100 are defined as excellent, 80-89 as good, 70-79 as fair, and scores below 70 as poor.
Change from baseline in WOMAC score
The WOMAC score is a patient-reported outcome assessing pain, joint stiffness and physical function for hip or knee disorders. It contains 24 items with individual item scores ranging from 0 (none) to 4 (extreme). The total score ranges from 0 to 96, where higher scores indicate more severe symptoms. Total scores 0-24 represent minimal symptoms, 25-41 mild, 42-69 moderate, 70-86 severe, and ≥87 extreme symptoms.
Change from baseline in necrotic lesion volume of the femoral head on MRI
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Beijing Jishuitan Hospital
Lead sponsor
Carrier Biomed (Suzhou) Co., Ltd
Collaborator
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