Phase 1 Clinical Trial of CordSTEM-ST (CBT210-POI_P1)

Single-dose, Phase 1 Clinical Trial to Evaluate the Safety, and Tolerability of Allogeneic Umbilical Cord-derived Mesenchymal Stem Cell Therapy in Patients With Premature Ovarian Insufficiency

This clinical study will evaluate the safety and tolerability of CordSTEM-ST after administering a single dose in patients with premature ovarian insufficiency (POI), as well as identify the maximum tolerated dose (MTD) and evaluate the potential therapeutic effects.

Study Overview

• Status: Completed
• Conditions: Premature Ovarian Insufficiency
• Intervention / Treatment: Biological: umbilical cord-derived mesenchymal stem cell

Detailed Description

3 or 6 subjects will be enrolled sequentially by applying the "traditional 3+3 design" to evaluate the dose limiting toxicities (DLTs) for 28 days from the first administration date of the investigational product according to the protocol. A DLT is defined as the occurrence of a grade 3 or higher adverse drug reaction (ADR) according to the National Cancer Institute (NCI)-Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.

First three subjects will be enrolled in Study Group 1 (low dose group) to be evaluated for DLTs up to 28 days after IP administration. If none of three subjects develope DLT, three subjects for Study Group 2 (high dose group) will be recruited. If one out of three subjects in Study Group 1 develop DLT, additional three subjects in Study Group 1 will be enrolled to be evaluated for DLT. If two or more subjects develop DLT in initial three subjects, the study will be terminated.

This study will be followed up by a long-term follow-up study under the separate protocol.

• Study Type: Interventional
• Enrollment (Actual): 6
• Phase: Phase 1

Contacts and Locations

Study Locations

• South Korea
  • Gyeonggi-do
    • Seongnam-si, Gyeonggi-do, South Korea, 463-712
      • CHA Bundang Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Females aged ≥ 25 years and < 40 years
• Individuals diagnosed with premature ovarian insufficiency: Individuals who have a follicle stimulating hormone (FSH) level of 40 IU/L or higher in the results of two tests conducted at least 4 weeks apart and are amenorrheic for at least 4 months
• Individuals who voluntarily decide to participate and provide written consent

Exclusion Criteria:

• Individuals diagnosed with primary amenorrhea*

  * No secondary sex characteristics by age 13, Absence of menarche for 5 years after initial breast development, or Absence of menstruation by age 15

• Individuals diagnosed with polycystic ovary syndrome
• Individuals with any other conditions that may affect the result of this study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Study Group 1 (Low dose CordSTEM-ST); Low dose CordSTEM-ST, 1 (single) administration	Biological: umbilical cord-derived mesenchymal stem cell; allogeneic umbilical cord-derived mesenchymal stem cell; Other Names: CordSTEM-ST
Experimental: Study Group 2 (High dose CordSTEM-ST); High dose CordSTM-ST, 1 (single) administration	Biological: umbilical cord-derived mesenchymal stem cell; allogeneic umbilical cord-derived mesenchymal stem cell; Other Names: CordSTEM-ST

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of adverse events	Number of adverse event cases reported	up to 6 months after the IP administration
Abnormality cases in lab test results	Number of clinically significant abnormalities found in lab test results.	up to 6 months after the IP administration
Abnormality cases physical exam results	Number of clinically significant abnormalities found in physical exam results	up to 6 months after the IP administration
Abnormality cases in vital signs	Number of clinically significant abnormalities found in vital signs	up to 6 months after the IP administration

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percent change from baseline in blood Follicle Stimulating Hormone (FSH) level	blood Follicle Stimulating Hormone (FSH) level measured in mIU/ml	up to 6 months after the IP administration
Percent change from baseline in blood Anti-Mullerian Hormone (AMH) level	blood Anti-Mullerian Hormone (AMH) level measured in ng/ml	up to 6 months after the IP administration
Percent change from baseline blood Estradiaol (E2) level	blood Estradiaol (E2) level measured in pg/ml	up to 6 months after the IP administration
Percent change from baseline in number of mature follicles	number of mature follicles measured in number of follicles	up to 6 months after the IP administration
Percent change from baseline in number of antral follicles	number of antral follicles measured in number of follicles	up to 6 months after the IP administration
Percent change from baseline in endometrial thickness	endometrial thickness measured in cm	up to 6 months after the IP administration
Status of menstruation resumption	Collected by patient interview	up to 6 months after the IP administration

Collaborators and Investigators

• Sponsor: CHABiotech CO., Ltd

Study record dates

Study Major Dates

• Study Start (Actual): August 30, 2024
• Primary Completion (Actual): April 28, 2025
• Study Completion (Actual): April 28, 2025

Study Registration Dates

• First Submitted: August 26, 2024
• First Submitted That Met QC Criteria: August 27, 2024
• First Posted (Actual): August 29, 2024

Study Record Updates

• Last Update Posted (Actual): November 19, 2025
• Last Update Submitted That Met QC Criteria: November 17, 2025
• Last Verified: August 1, 2024

More Information

Terms related to this study

• Keywords: Stem cell therapy; allogeneic stem cell; umbilical cord derived mesenchymal stem cell
• Additional Relevant MeSH Terms: Urogenital Diseases; Genital Diseases; Endocrine System Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Genital Diseases, Female; Ovarian Diseases; Adnexal Diseases; Gonadal Disorders; Primary Ovarian Insufficiency
• Other Study ID Numbers: CBT210-POI_P1

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No