Infusion of stem cells for osteoporosis in postmenopausal women

A Single-center, Open-label, Single-arm, 3+3 Dose-escalation Phase I Clinical Study to Evaluate Safety and Tolerability of hMSC100 (Human UC-MSC Injection) in Postmenopausal Women With Osteoporosis and at High Risk of Fracture

This study is testing if giving stem cells from umbilical cords to postmenopausal women with osteoporosis can be safe and help improve their bone health.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 18 (estimated)
• Ages: 45 Years to 85 Years
• Sex: Female
• Sponsor: Help Therapeutics (Industry-sponsored)
• Drugs / interventions: denosumab, radiation
• Locations: 1 site (Nanjing, Jiangsu)
• Trial ID: NCT06949137 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety and tolerability of human umbilical cord-derived mesenchymal stem cells (hMSC100) administered intravenously to postmenopausal women with osteoporosis who are at high risk of fractures. Participants will be women aged 45 to 85 who have been diagnosed with osteoporosis and meet specific criteria related to fracture history and bone mineral density. The study aims to determine how well these stem cells can be tolerated and their potential effects on bone health.

Who should consider this trial

Good fit: Ideal candidates are postmenopausal women aged 45 to 85 with osteoporosis and a history of fractures or low bone mineral density.

Not a fit: Patients who are not postmenopausal or do not meet the criteria for osteoporosis may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve bone health and reduce fracture risk in postmenopausal women with osteoporosis.

How similar studies have performed: While the use of mesenchymal stem cells in treating osteoporosis is a novel approach, similar studies have shown promise in other conditions, indicating potential for success.

Eligibility criteria

Inclusion Criteria:

All of the following criteria must be met for inclusion:

1. Willingness to participate in the clinical trial and signing of informed consent;
2. Female, age between 45 (inclusive) and 85 (inclusive) years, with primary menopause for ≥2 years;
3. Body weight ≥40 kg, and body mass index (BMI) between 18 kg/m² (inclusive) and 30 kg/m² (inclusive);
4. Meets the diagnostic criteria for osteoporosis, exhibiting at least one of the following:

   * Sustained a fragility fracture within the past 2 years;

     * Suffered a fracture while receiving anti-osteoporosis medication;

       * History of multiple-site fractures (including vertebral, hip, proximal humerus, or distal radius, etc.);

         * Bone mineral density (BMD) T-score \< -3.0 at the lumbar spine (L1-L4) or hip (total hip or femoral neck) as measured by DXA;

           * High risk of falling;

             * Calculated by FRAX (Fracture Risk Assessment Tool), a 10-year risk of major osteoporotic fracture \>30% or hip fracture risk \>4.5%;

               * Currently using medications known to cause skeletal harm \[such as high-dose glucocorticoids (prednisolone ≥7.5 mg/day for over 3 months), etc.\].

Exclusion Criteria:

1. Diseases affecting bone metabolism: various metabolic bone diseases such as osteogenesis imperfecta and osteomalacia; Paget's disease of bone, hypercalcemia, hypocalcemia (participants must not have used calcium supplements within 24 hours before blood sampling for serum calcium screening); Cushing's syndrome, hyperprolactinemia; hypopituitarism; acromegaly, etc.; hyperparathyroidism or hypoparathyroidism; secondary menopause, etc.
2. Secondary osteoporosis and other severe conditions, such as primary bone tumors (e.g., multiple myeloma, osteosarcoma, chondrosarcoma), secondary bone tumors, hematologic malignancies, or drug-induced osteoporosis.
3. Current malignancy, history of malignancy not cured for at least 5 years, or disability due to severe or long-term diseases (such as stroke, Parkinson's disease, multiple sclerosis) resulting in inability to ambulate.
4. Severe infectious diseases, autoimmune diseases (e.g., systemic lupus erythematosus), uncontrolled severe hypertension, or diabetes mellitus with severe complications or unstable blood glucose; severe cardiovascular, cerebrovascular, or other significant diseases.
5. Allergic constitution: known allergy to products derived from mammalian cells or to the investigational product of clinical significance.
6. Patients who have undergone major organ or bone marrow transplantation; patients who have received external radiation or skeletal implantation of radioactive materials.
7. Patients who have previously received any form of cell therapy.
8. Previous treatment with anti-osteoporosis drugs or medications affecting bone metabolism:

   * Treatment with any PTH analog within the past 6 months (including participation in clinical trials of similar products);

     * Treatment with any RANKL inhibitor (such as denosumab) within the past year;

       * Cumulative use of oral bisphosphonates for ≥3 years; or cumulative use \>3 months but \<3 years with the last dose administered within 6 months before screening, or intravenous bisphosphonate use within 24 months before screening.
9. DXA measurement: ① Fewer than two lumbar vertebrae were measurable by DXA; ② Height, weight, or body size may impede accurate DXA measurement.
10. HBV-DNA ≥1,000 copies (cps)/mL or above the upper limit of normal; hepatitis C virus (HCV) antibody positive and HCV RNA copies above the upper limit of normal; positive syphilis antibody, or HIV antibody.
11. Malabsorption syndromes, such as Crohn's disease and chronic pancreatitis. Known impaired absorption of calcium or vitamin D.
12. History of neurological or psychiatric disorders.
13. Current uncontrolled thyroid disease, hyperthyroidism, or hypothyroidism; thyroid stimulating hormone (TSH) level below normal range; TSH elevated (\>5.5 μIU/mL but ≤10.0 μIU/mL) and serum T4 outside the normal range; TSH \>10.0 μIU/mL.
14. Known severe hepatic insufficiency (AST or ALT ≥2×ULN, ALP or total bilirubin ≥1.5×ULN), liver cirrhosis, unstable liver disease, or known clinically significant biliary anomalies as judged by the investigator (excluding Gilbert's syndrome or asymptomatic gallstones); known moderate to severe chronic kidney disease (eGFR \<60 mL/min/1.73 m²).
15. Evidence of alcohol abuse or drug abuse within 12 months before screening, as determined by the investigator, may interfere with the understanding or completion of the study by the participant.
16. Participation in another clinical trial within 6 months before screening.
17. Other conditions deemed by the investigator as unsuitable for participation.

Where this trial is running

Nanjing, Jiangsu

• HelpThera — Nanjing, Jiangsu, China (Recruiting)

Study contacts

• Principal investigator: Juan Li, MD, PhD — The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
• Study coordinator: Jiaxian Wang, MD, PhD

Conditions Osteoporosis in Post-menopausal Women; Osteoporosis; mesenchymal stem cell; osteooprpsis; post menopausal; intravenous; hMSC100; fracture