MedDataX Logo

Phase I Clinical Study of Haploid Hematopoietie Stem CellTransplantation Combined With Hypoxic 3D-Cultured Umbilical Cord Mesenchymal Stem Cells for the Treatment of Severe Aplastic Anemia

NCT ID: NCT07299123

Last Updated: 2025-12-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

ENROLLING_BY_INVITATION

Clinical Phase

PHASE1/PHASE2

Total Enrollment

40 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-08-01

Study Completion Date

2026-12-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

Efficacy and Safety of Sequential Infusion of Hypoxic 3D-Cultured Umbilical Cord Mesenchymal Stem Cells in Haploidentical Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia: A Multicenter, Randomized, Phase 1 Trial

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

The enrolled patients with an HLA-haploidentical relative for HSCT received the fludarabine (Flu) + cyclophosphamide (Cy) + antithymocyte globulin (ATG) conditioning regimen. For the patients with acute SAA (SAA-I), intravenous administration of 30 mg/(m2 day) of Flu and 500-800 mg/(m2 day) of Cy was performed from days -5 to -2, and 5μg/(kg day) of ATG was administered from days -4 to -1. For the patients with chronic SAA (SAA-II), the same treatment of ATG and Cy was applied as above with the supplement of 0.6 mg/(kg 6 h) of busulfan (BU) from days -8 to -5 prior to transplantation. Donor selection and hematopoietic stem cell mobilization and collection were conducted based on the consensus of The Chinese Society of Hematology regarding indications, conditioning regimens, and donor selection for allogeneic hematopoietic stem cell transplantation. On day 0, HSCs were infused intravenously. Both groups received 5 × 10⁵/kg UC-MSCs at 4 h before HSC infusion. The control group was transfused with conventional 2D-cultured UC-MSCs, whereas the experimental group received 3D hypoxia-preconditioned UC-MSCs. Standard GVHD prophylaxis consisted of mycophenolate mofetil, cyclosporine A, and methotrexate (MTX).

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Severe Aplastic Anemia (SAA) Severe Aplastic Anemia, Refractory Severe Aplastic Anemia

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

Severe Aplastic Anemia Hematopoietic Stem Cell Transplantation Mesenchymal Stem Cells

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

3D hypoxia-preconditioned UC-MSC group

Group Type EXPERIMENTAL

3D hypoxia-preconditioned UC-MSC group

Intervention Type DRUG

Received 5 × 10⁵/kg 3D hypoxia-preconditioned UC-MSCs at 4 h before HSC infusion

2D UC-MSC group

Group Type ACTIVE_COMPARATOR

2D UC-MSC group

Intervention Type DRUG

Received 5 × 10⁵/kg 2D UC-MSCs at 4 h before HSC infusion

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

3D hypoxia-preconditioned UC-MSC group

Received 5 × 10⁵/kg 3D hypoxia-preconditioned UC-MSCs at 4 h before HSC infusion

Intervention Type DRUG

2D UC-MSC group

Received 5 × 10⁵/kg 2D UC-MSCs at 4 h before HSC infusion

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

The inclusion criteria were (1) age 6 to 60 years; (2) diagnosis of SAA or very SAA according to the International Aplastic Anemia Study Group\[21\]; (3) without any severe pulmonary, cardiac, liver, or renal diseases or any active infection; and (4) adequate performance status (Eastern Cooperative Oncology Group score 0-2).

Exclusion Criteria

Fanconi anemia was excluded after evaluation of diepoxybutane-stimulated peripheral blood (PB) chromosome breakages. Patients with congenital aplastic anemia like dyskeratosis or Diamond-Blackfan anemia were also excluded.
Minimum Eligible Age

6 Years

Maximum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

The Fourth Medical Center of Chinese PLA General Hospital

UNKNOWN

Sponsor Role collaborator

Beijing 302 Hospital

OTHER

Sponsor Role collaborator

The First Medical Center of Chinese PLA General Hospital

OTHER

Sponsor Role collaborator

Beijing Dajikanghua Biotechnology Co., Ltd.

UNKNOWN

Sponsor Role collaborator

Pollon Life Co., Ltd.

UNKNOWN

Sponsor Role collaborator

Yan'an University Affiliated Hospital

OTHER

Sponsor Role collaborator

General Hospital of Shenzhen University

UNKNOWN

Sponsor Role collaborator

The University of Hong Kong-Shenzhen Hospital

OTHER

Sponsor Role collaborator

Beijing Friendship Hospital

OTHER

Sponsor Role collaborator

Chinese PLA General Hospital

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Jian Bo

Chief Physician / Associate Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

28 Fuxing Road, Beijing, China.

Beijing, , China

Site Status

Countries

Review the countries where the study has at least one active or historical site.

China

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

S2021-384-01

Identifier Type: -

Identifier Source: org_study_id