Phase I Clinical Study of Haplo-HSCT Combined With Hypoxic 3D-Cultured Umbilical Cord MSC for the Treatment of SAA

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

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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

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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

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Detailed Description

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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

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

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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3D hypoxia-preconditioned UC-MSC group

Group Type EXPERIMENTAL

3D hypoxia-preconditioned UC-MSC group

Intervention Type DRUG

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

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

Interventions

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3D hypoxia-preconditioned UC-MSC group

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

Intervention Type DRUG

2D UC-MSC group

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

Intervention Type DRUG

Eligibility Criteria

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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

Exclusion Criteria：(1)Age 6 years or \> 60 years; (2)Failure to meet the IAASG-defined diagnosis of SAA/vSAA, or confirmed (3)Fanconi anemia, congenital aplastic anemia (e.g., dyskeratosis congenita, Diamond-Blackfan anemia); (4)Presence of severe pulmonary, cardiac, hepatic, or renal insufficiency; Uncontrolled active infection; (5)Eastern Cooperative Oncology Group (ECOG) performance status score ≥ 3.

Minimum Eligible Age

6 Years

Maximum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No