Recruiting
Phase 1/2
Phase Ⅰ/Ⅱa Trial Evaluating Intravenous hUC-MSCs Injection for Safety, Tolerability and Efficacy in Patients With POI
Condition(s) studied
Premature Ovarian Insufficiency
Investigational drug(s) / intervention(s)
hUC-MSCs Injection (Phase I)hUC-MSCs Injection (Phase IIa)Placebo
hUC-MSCs Injection (Phase I): hUC-MSCs Injection, administered via intravenous infusion as a single dose on D0.
hUC-MSCs Injection (Phase IIa): hUC-MSCs Injection, 3 infusions in total, dosing interval ≥7 days.
Placebo: 3 infusions in total, dosing interval ≥7 days
Study summary
A Phase I/IIa clinical study evaluating the safety, tolerability and efficacy of intravenously administered human umbilical cord mesenchymal stem cell (hUC-MSCs) injection in patients with premature ovarian insufficiency (POI).
The primary objective of the Phase I dose-escalation stage is to assess the safety and tolerability of intravenous infusion of hUC-MSCs injection for the treatment of POI, and to determine the recommended phase II dose (RP2D) for the Phase IIa clinical trial.
The primary objective of the Phase IIa dose-expansion stage is to evaluate the efficacy of intravenous infusion of hUC-MSCs injection in POI treatment, and to generate data to support pivotal/confirmatory clinical trials.
Eligibility
Inclusion Criteria:
1. Aged 18 to 40 years at the time of signing the informed consent form (exclusive of the boundary values);
2. Meet the diagnostic criteria specified in the Expert Consensus on Clinical Diagnosis and Treatment of Premature Ovarian Insufficiency (2023 Edition): oligomenorrhea (menstrual cycle longer than 35 days) or amenorrhea for more than 4 months, with basal serum follicle-stimulating hormone (FSH) \> 25 U/L (tested on Days 2-4 of the menstrual cycle or during amenorrhea, at least twice with an interval of ≥4 weeks);
3. Satisfy one of the following two criteria:
1. Total number of antral follicles (AFC) with a diameter of 2-10 mm in bilateral ovaries \< 5;
2. Serum anti-Müllerian hormone (AMH) ≤ 7.85 pmol/L (equivalent to 1.1 ng/mL);
4. Have received standardized hormone replacement therapy (HRT) at a stable dose for ≥3 months prior to drug administration with stable hormone levels, and maintain stable HRT per medical advice throughout the trial (within 24 weeks after enrollment);
5. Have no fertility requirements;
6. Agree to use effective contraceptive measures throughout the trial period (such as complete abstinence, condoms, cervical caps plus spermicides, intrauterine devices, etc.);
7. Negative serum β-hCG test result during the screening period (to rule out pregnancy);
8. Fully understand the trial information and sign the informed consent form.
Exclusion Criteria:
1. Subjects with primary amenorrhea;
2. Subjects with thyroid disorders (hypothyroidism/hyperthyroidism or thyroid malignant tumors) or resistant ovary syndrome (ROS);
3. Subjects with abnormal female karyotypes (e.g., Turner syndrome, Fragile X syndrome);
4. Positive serology tests (HBV antibody, HCV antibody, HIV antibody, syphilis). Exceptions: HBV carriers, patients with stable HBV after drug treatment (HBV DNA titer ≤500 IU/mL or copies \<1000 copies/mL), and patients with cured HCV (negative HCV RNA test) may be enrolled if deemed eligible by the investigator;
5. Subjects receiving or planning to use the following medications during the trial: oral or systemic corticosteroids, danazol, anticoagulants, Chinese herbal medicines or botanical supplements that may affect hormone levels or ovarian function;
6. History of hypersensitivity to human serum albumin;
7. Pregnant or breastfeeding subjects;
8. Participation in another clinical trial within the past 3 months, or receipt of other cell therapies (excluding blood transfusion);
9. History of malignant tumors;
10. Subjects with contraindications or cautions for estrogen use, including known or suspected breast cancer, endometrial cancer, other known or suspected sex hormone-dependent malignancies, active venous or arterial thromboembolic diseases within the last 6 months, undiagnosed abnormal genital bleeding, severe hepatic or renal insufficiency \[serum aspartate aminotransferase (AST) \>3×ULN, serum alanine aminotransferase (ALT) \>3×ULN, estimated glomerular filtration rate (eGFR) \<60 mL/min\], etc.;
11. Uncontrolled diabetes, hypertension (\>150/100 mmHg), heart disease, etc.;
12. Any other conditions judged by the investigator to render the subject unsuitable for participation in this study.
Primary outcome measure(s)
- Phase I : Incidence and number of subjects with treatment-emergent adverse events and serious adverse events. — 48 weeks
In this Phase I stage, safety is the primary endpoint. Types, frequencies, severities and causal relationships of all adverse events (AEs) and serious adverse events (SAEs) occurring from the first administration of study drug to the end of safety follow-up (48 weeks), assessed in accordance with the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 6.0; together with the proportion of adverse events judged by the investigator to be related to the study drug.
- Phase II ：FSH — at Week 24 post-treatment
To evaluate the changes from baseline in serum follicle-stimulating hormone (FSH) levels at Week 24 post-treatment.
- Phase II ：E2 — at Week 24 post-treatment
To evaluate the changes from baseline in serum festradiol (E2) levels at Week 24 post-treatment.
- Phase II ：AMH — at Week 24 post-treatment
To evaluate the changes from baseline in serum anti-Müllerian hormone (AMH) levels at Week 24 post-treatment.
Trial sites (1)
| Facility | City | Region | Status |
|---|
| Peking Union Medical College Hospital |
Beijing |
Beijing Municipality |
Recruiting |
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