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.
Aged 18 to 40 years at the time of signing the informed consent form (exclusive of the boundary values);
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);
Total number of antral follicles (AFC) with a diameter of 2-10 mm in bilateral ovaries < 5;
Serum anti-Müllerian hormone (AMH) ≤ 7.85 pmol/L (equivalent to 1.1 ng/mL);
Subjects with primary amenorrhea;
Subjects with thyroid disorders (hypothyroidism/hyperthyroidism or thyroid malignant tumors) or resistant ovary syndrome (ROS);
Subjects with abnormal female karyotypes (e.g., Turner syndrome, Fragile X syndrome);
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;
Parallel
hUC-MSCs Injection (Phase I)
DrughUC-MSCs Injection, administered via intravenous infusion as a single dose on D0.
hUC-MSCs Injection (Phase IIa)
DrughUC-MSCs Injection, 3 infusions in total, dosing interval ≥7 days.
Placebo
Drug3 infusions in total, dosing interval ≥7 days
6
Treatment groupsSee each treatment group below.
Phase I : Incidence and number of subjects with treatment-emergent adverse events and serious adverse events.
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
To evaluate the changes from baseline in serum follicle-stimulating hormone (FSH) levels at Week 24 post-treatment.
Phase II ：E2
To evaluate the changes from baseline in serum festradiol (E2) levels at Week 24 post-treatment.
Phase II ：AMH
To evaluate the changes from baseline in serum anti-Müllerian hormone (AMH) levels at Week 24 post-treatment.
Phase I: FSH
To evaluate the changes from baseline in serum follicle-stimulating hormone (FSH) levels.
Phase I ：E2
To evaluate the changes from baseline in serum estradiol (E2) levels
Phase I : AMH
To evaluate the changes from baseline in serum anti-Müllerian hormone (AMH) levels.
Phase II: Incidence and number of subjects with treatment-emergent adverse events and serious adverse events.
In this Phase II stage, safety is the secondary 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.
Click on the lead sponsor to view all of their trials.
