Phase Ⅰ/Ⅱa Trial Evaluating Intravenous hUC-MSCs Injection for Safety, Tolerability and Efficacy in Patients With POI
Phase Ⅰ/Ⅱa Clinical Trial to Evaluate the Safety, Tolerability and Efficacy of Intravenous Injection of Human Umbilical Cord Mesenchymal Stem Cells (hUC-MSCs) in Patients With Premature Ovarian Insufficiency (POI)
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.
Studienübersicht
Status
Status
Bedingungen
Bedingungen
Intervention / Behandlung
Intervention / Behandlung
Detaillierte Beschreibung
Premature ovarian insufficiency (POI) is defined as ovarian dysfunction occurring in women before the age of 40, which is mainly characterized by menstrual abnormalities (amenorrhea, oligomenorrhea or polymenorrhea), elevated gonadotropin levels (FSH > 25 U/L), and fluctuating decline in estrogen levels. The incidence of POI is age-specific: 1 in 250 women develops POI before 35 years of age, and 1 in 100 women develops POI before 40 years of age. The incidence rate of POI in China is 2.8%, showing an upward trend year by year with a younger onset age.
hormone replacement therapy (HRT) relieves symptoms caused by low estrogen through estrogen supplementation. Sequential estrogen-progestogen therapy is recommended for POI patients with an intact uterus. Since patients with POI require long-term HRT, natural or nearly natural estrogens and progestogens can be selected to minimize adverse effects on the mammary gland, metabolism, cardiovascular system and other systems. Nevertheless, HRT still increases the risks of breast cancer, heart disease and stroke.
Studies have demonstrated that mesenchymal stem cells can home to ovarian stroma, regulate the balance of Th1/Th2 cytokines and the expression of endometrial natural killer cells, alleviate inflammatory responses, inhibit apoptosis of ovarian granulosa cells and reduce ovarian interstitial fibrosis, thereby improving the ovarian microenvironment. Meanwhile, these cells can secrete growth factors including VEGF, IGF-1 and HGF, promote proliferation of various ovarian cells and angiogenesis, repair ovarian structure and restore ovarian ovarian function. This study aims to evaluate the safety, tolerability and efficacy of intravenously administered hUC-MSCs in the treatment of patients with POI.
Studientyp
Studientyp
Einschreibung (Geschätzt)
Einschreibung
Phase
Phase
- Phase 2
- Phase 1
Kontakte und Standorte
Studienkontakt
Studienkontakt
- Name: Xiaohong Wang,MD
- Telefonnummer: +86 755 6683 5786
- E-Mail: xiaohong@wingor.net
Studienorte
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Beijing Municipality
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Beijing, Beijing Municipality, China, 100730
- Rekrutierung
- Peking Union Medical College Hospital
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Kontakt:
- Qi Yu, Prof.
- Telefonnummer: +86 10 6915 8621
- E-Mail: yuqimd@163.com
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Teilnahmekriterien
Zulassungskriterien
Zulassungskriterien
Studienberechtigtes Alter
- Erwachsene
Akzeptiert gesunde Freiwillige
Beschreibung
Inclusion Criteria:
- 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);
Satisfy one of the following two criteria:
- 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);
- 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);
- Have no fertility requirements;
- Agree to use effective contraceptive measures throughout the trial period (such as complete abstinence, condoms, cervical caps plus spermicides, intrauterine devices, etc.);
- Negative serum β-hCG test result during the screening period (to rule out pregnancy);
- Fully understand the trial information and sign the informed consent form.
Exclusion Criteria:
- 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;
- 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;
- History of hypersensitivity to human serum albumin;
- Pregnant or breastfeeding subjects;
- Participation in another clinical trial within the past 3 months, or receipt of other cell therapies (excluding blood transfusion);
- History of malignant tumors;
- 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.;
- Uncontrolled diabetes, hypertension (>150/100 mmHg), heart disease, etc.;
- Any other conditions judged by the investigator to render the subject unsuitable for participation in this study.
Studienplan
Wie ist die Studie aufgebaut?
Designdetails
- Hauptzweck: Behandlung
- Zuteilung: Zufällig
- Interventionsmodell: Parallele Zuordnung
- Maskierung: Vervierfachen
Anzahl der Arme
Waffen und Interventionen
Teilnehmergruppe / ArmTeilnehmergruppe / Arm |
Intervention / BehandlungIntervention / Behandlung |
|---|---|
|
Experimental: Phase I, Dose Cohort 1
Human Umbilical Cord Mesenchymal Stem Cells Injection
|
hUC-MSCs Injection, administered via intravenous infusion as a single dose on D0.
Andere Namen:
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Experimental: Phase I, Dose Cohort 2
Human Umbilical Cord Mesenchymal Stem Cells Injection
|
hUC-MSCs Injection, administered via intravenous infusion as a single dose on D0.
Andere Namen:
|
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Experimental: Phase I, Dose Cohort 3
Human Umbilical Cord Mesenchymal Stem Cells Injection
|
hUC-MSCs Injection, administered via intravenous infusion as a single dose on D0.
Andere Namen:
|
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Experimental: Phase IIa, Medium-Dose Cohort
Human Umbilical Cord Mesenchymal Stem Cells Injection
|
hUC-MSCs Injection, 3 infusions in total, dosing interval ≥7 days.
Andere Namen:
|
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Experimental: Phase IIa, High-Dose Cohort
Human Umbilical Cord Mesenchymal Stem Cells Injection
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hUC-MSCs Injection, 3 infusions in total, dosing interval ≥7 days.
Andere Namen:
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Placebo-Komparator: Phase IIa, Placebo Cohort
Multiple Electrolytes Injection
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3 infusions in total, dosing interval ≥7 days
|
Was misst die Studie?
Primäre Ergebnismessungen
Primäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
|---|---|---|
|
Phase I : Incidence and number of subjects with treatment-emergent adverse events and serious adverse events.
Zeitfenster: 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.
|
48 weeks
|
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Phase II :FSH
Zeitfenster: at Week 24 post-treatment
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To evaluate the changes from baseline in serum follicle-stimulating hormone (FSH) levels at Week 24 post-treatment.
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at Week 24 post-treatment
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Phase II :E2
Zeitfenster: at Week 24 post-treatment
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To evaluate the changes from baseline in serum festradiol (E2) levels at Week 24 post-treatment.
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at Week 24 post-treatment
|
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Phase II :AMH
Zeitfenster: at Week 24 post-treatment
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To evaluate the changes from baseline in serum anti-Müllerian hormone (AMH) levels at Week 24 post-treatment.
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at Week 24 post-treatment
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Sekundäre Ergebnismessungen
Sekundäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
|---|---|---|
|
Phase I: FSH
Zeitfenster: 48 weeks
|
To evaluate the changes from baseline in serum follicle-stimulating hormone (FSH) levels.
|
48 weeks
|
|
Phase I :E2
Zeitfenster: 48 weeks
|
To evaluate the changes from baseline in serum estradiol (E2) levels
|
48 weeks
|
|
Phase I : AMH
Zeitfenster: 48 weeks
|
To evaluate the changes from baseline in serum anti-Müllerian hormone (AMH) levels.
|
48 weeks
|
|
Phase II: Incidence and number of subjects with treatment-emergent adverse events and serious adverse events.
Zeitfenster: 48 weeks
|
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.
|
48 weeks
|
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Phase I/II: AFC
Zeitfenster: 48 weeks
|
Changes from baseline in antral follicle count (AFC) by transvaginal ultrasound.
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48 weeks
|
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Phase I/II: endometrial thickness
Zeitfenster: 48 weeks
|
Changes from baseline in endometrial thickness measured by transvaginal ultrasound.
|
48 weeks
|
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Phase I/II: menstrual recovery status
Zeitfenster: 48 weeks
|
Menstrual recovery status will be assessed via patient inquiry at each visit.
|
48 weeks
|
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Phase I/II:Modified Kupperman Index
Zeitfenster: 48 weeks
|
To evaluate the changes in the Modified Kupperman Index from baseline across all post-baseline follow-up visits. Modified Kupperman Index will be assessed through physician interview.The total score ranges from 0 to 63 points. Total score stratification: >30 points indicates severe symptoms, 16-30 points moderate symptoms, 6-15 points mild symptoms, and <6 points normal status. |
48 weeks
|
Mitarbeiter und Ermittler
Sponsor
Sponsor
Studienaufzeichnungsdaten
Haupttermine studieren
Studienbeginn (Tatsächlich)
Studienbeginn
Primärer Abschluss (Geschätzt)
Primärer Abschluss
Studienabschluss (Geschätzt)
Studienabschluss
Studienanmeldedaten
Zuerst eingereicht
Zuerst eingereicht
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
Zuerst gepostet (Tatsächlich)
Zuerst gepostet
Studienaufzeichnungsaktualisierungen
Letztes Update gepostet (Tatsächlich)
Letztes Update gepostet
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
Zuletzt verifiziert
Zuletzt verifiziert
Mehr Informationen
Begriffe im Zusammenhang mit dieser Studie
Schlüsselwörter
Zusätzliche relevante MeSH-Bedingungen
- Urogenitale Erkrankungen
- Genitalerkrankungen
- Erkrankungen des endokrinen Systems
- Weibliche Urogenitalerkrankungen
- Weibliche Urogenitalerkrankungen und Schwangerschaftskomplikationen
- Genitalerkrankungen, weiblich
- Eierstockerkrankungen
- Adnexerkrankungen
- Gonadenstörungen
- Primäre Ovarialinsuffizienz
- Qualität, Zugang und Bewertung im Gesundheitswesen
- Untersuchungstechniken
- Epidemiologische Methoden
- Gesundheitsbewertungsmechanismen
- Qualität der Gesundheitsversorgung
- Öffentliche Gesundheit
- Umwelt und öffentliche Gesundheit
- Epidemiologische Studienmerkmale
- Klinische Studien als Thema
- Klinische Studien als Thema
- Klinische Studien, Phase I als Thema
Andere Studien-ID-Nummern
Andere Studien-ID-Nummern
- WG108-POI-I/IIa
Plan für individuelle Teilnehmerdaten (IPD)
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