NCT05794425

Brief Summary

The multicenter collaborative clinical study conducted a systematic clinical observation in the treatment of bone marrow failure diseases via UCB\&UC-MSCs , in order to observe its clinical efficacy and safety.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
100

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Aug 2023

Typical duration for phase_1

Geographic Reach
1 country

8 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 20, 2023

Completed
14 days until next milestone

First Posted

Study publicly available on registry

April 3, 2023

Completed
4 months until next milestone

Study Start

First participant enrolled

August 1, 2023

Completed
1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2025

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2026

Completed
Last Updated

November 29, 2023

Status Verified

November 1, 2023

Enrollment Period

1.7 years

First QC Date

March 20, 2023

Last Update Submit

November 26, 2023

Conditions

Bone Marrow Failure Disorders

Outcome Measures

Primary Outcomes (1)

  • Objective Response Rate (ORR)

    To evaluate efficacy of UCB in combination with UC-MSCs in the treatment of bone marrow failure disorders

    Up to two years

Secondary Outcomes (2)

  • Progression-free Survival (PFS)

    Up to two years

  • Overall Survival (OS)

    Up to two years

Study Arms (2)

Cyclosporine A

EXPERIMENTAL
Drug: Cyclosporine A

UCB+UC-MSCs

EXPERIMENTAL
Biological: Umbilical cord blood & Umbilical cord derived mesenchymal stem cells

Interventions

Cyclosporine ADRUG

3-5 mg per kilogram per day.

Cyclosporine A
Umbilical cord blood & Umbilical cord derived mesenchymal stem cellsBIOLOGICAL

Intravenous infusion of umbilical cord blood and umbilical cord derived mesenchymal stem cells (5-8×10\^7) at 1 week interval.

UCB+UC-MSCs

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Patients with bone marrow failure disorders, including aplastic anemia, pure red cell aplastic anemia, immune-related pancytopenia, paroxysmal nocturnal hemoglobinuria, myelodysplastic syndrome, acute arrest of hemopoiesis, and cytopenia of unknown significance;
  • Patients with no severe impairment of liver and kidney function (total bilirubin (TBIL): ≤ 1.5×ULN; ALT or AST: ≤ 2.5×ULN; Alkaline phosphatase: ≤ 3×ULN; Serum creatinine: ≤ 1.5×ULN);
  • The prothrombin time (PT) or activated partial thrombin time (APTT) or international normalized ratio (INR): ≤ 1.5×ULN in the absence of anticoagulant therapy;
  • The left ventricular ejection fraction (LVEF): ≥50% by cardiac echocardiography
  • Hepatitis B surface antigen (HBsAg) and Hepatitis B core antibody (HBcAb) were negative. If any of the above items is positive, the HBV DNA titer in peripheral blood must be lower than the detection limit or 1×10\^3 copys/ml;
  • Patients with no plans for stem cell transplantation;
  • Patients with ECOG 0-2;
  • Those who voluntarily participate in this clinical study and have signed an informed consent .

You may not qualify if:

  • Patients who have suffered malignant tumors other than squamous cell carcinoma of the skin, basal cell carcinoma of the skin, malignant melanoma cured by surgery, and carcinoma in situ of the cervix in the past 5 years;
  • Patient with severe cardiac insufficiency (e.g. grade Ⅲ、Ⅳ by NYHA classification NYHA), and medically uncontrolled hypertension (systolic blood pressure ≥ 160 mmHg or diastolic blood pressure ≥110 mmHg);
  • Patients with severe mental illness;
  • Patients with clinically significant infection should be recruited with delay;
  • AST or ALT: above 3 times the upper limit of normal, creatinine, total bilirubin, or alkaline: phosphatase (ALP): above 1.5 times the upper limit of normal;
  • Patients with test positive for HIV, HCV or syphilis;
  • Patients with severe allergies or allergic to the active ingredients, excipients of the drug, or blood products in this clinical trials;
  • Patients who are pregnant or breastfeeding, or have a childbirth plan in the near future;
  • Patients receive allogeneic/autologous hematopoietic stem cell transplantation by the assessment of investigators;

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (8)

Shandong Provincial Third Hospital

Jinan, Shandong, 250031, China

RECRUITING

Affiliated Hospital of Shandong University of Traditional Chinese Medicine

Jinan, Shandong, China

RECRUITING

Jining first people's Hospital

Jining, Shandong, China

RECRUITING

Linyi People's Hospital

Linyi, Shandong, China

RECRUITING

Rizhao People's Hospital

Rizhao, Shandong, China

RECRUITING

Tai'an Central Hospital

Tai’an, Shandong, China

RECRUITING

Weihai Municipal Hospital

Weihai, Shandong, China

RECRUITING

Yantai Ludong Hospital (Shandong Provincial Hospital Group)

Yantai, Shandong, China

RECRUITING

MeSH Terms

Conditions

Bone Marrow Failure Disorders

Interventions

Cyclosporine

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

CyclosporinsPeptides, CyclicMacrocyclic CompoundsPolycyclic CompoundsPeptidesAmino Acids, Peptides, and Proteins

Study Officials

  • zhe Yu, MD

    Shandong Province Third hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

zhe Yu, MD

CONTACT

86-18753103739doctoryu1120@163.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 20, 2023

First Posted

April 3, 2023

Study Start

August 1, 2023

Primary Completion

April 1, 2025

Study Completion

April 1, 2026

Last Updated

November 29, 2023

Record last verified: 2023-11

Data Sharing

IPD Sharing
Will not share

Locations

CN(8)