Conditioning Regimen Containing Low Dose ATG for The Treatment of Acquired SAA Receiving sUCBT

NCT06039436 | Unknown

• 1 other identifier: CBSAA001
• Study Type: observational
• Target: 72
• Locations: 1 country
• Sites: 1

Brief Summary

To evaluate the safety and efficacy of Single Umbilical Cord blood transplantation (sUCBT) containing low dose ATG based conditioning regimen in the treatment of acquired Severe Aplastic Anemia (SAA).

Conditions

Severe Aplastic Anemia

Keywords

Anti-Thymocyte Globulin (Rabbit); Umbilical Cord Blood Transplantation; Severe Aplastic Anemia

Outcome Measures

Primary Outcomes (1)

• The one-year probability of GRFS

  GRFS is defined as survival without grade III-IV acute GVHD immunosuppression requiring chronic GVHD or relapse.

  one year

Secondary Outcomes (11)

• The cumulative incidence of platelet engraftment at 28 days after transplantation

  28 days

• The cumulative incidence of neutrophil engraftment at 28 days after transplantation

  28 days

• Time of platelet engraftment

  28 days

• Time of neutrophil engraftment

  28 days

• The cumulative incidence of acute GVHD at 100 days after transplantation

  100 days

Study Arms (1)

ATG group

Patients in the ATG group will be treated with conditioning regimen containing ATG 2mg/kg on day -7 prior to transplant.

Drug: Anti-Thymocyte Globulin (Rabbit)

Interventions

Anti-Thymocyte Globulin (Rabbit) DRUG

Indications for trial stopping: subjects occur pregnancy events, severe organ function impairments, or emergency events related or unrelated to treatment; or subjects are unwilling to continue participating in the clinical trial.

ATG group

Eligibility Criteria

• Age: 1 Year - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Probability Sample

Study Population

patients with indications for hematopoietic stem cell transplantation and initially diagnosed with severe aplastic anemia or have not responded to immunosuppressive therapy

You may qualify if:

• Patients diagnosed with severe aplastic anemia who do not respond to initial diagnosis or immunosuppressive therapy;
• Patients meet diagnostic criteria for severe aplastic anemia (Camitta criteria);
• Age from 1 to 50 years old, male or female;
• Patients without HLA-matched sibling or unrelated donors avaliable;
• Patients who have undergone unrelated cord blood transplantation need to have HLA high-resolution matching、TNC (before cord blood freezing)≥3.0×107/kg、CD34+(before cord blood freezing)≥1.5×107/kg、tubule recovery rate≥80%;
• patients and their family members or guardians agreed to the study and signed informed consent;
• No severe organ failure;
• ECOG score≤2 and HCT-CI≤2;
• serum ferritin (SF)\<1000ug/L;
• women of reproductive agemust have a negative pregnancy test and agree to use effective contraception during treatment and for one year thereafter.

You may not qualify if:

• patients with inherited bone marrow failure syndromes;
• Positive for HLA-A, B, Cw, DRB1, DQB1, DPB1 site-specific HLA antibodies for donor cord blood with solid-phase fluorescence assay;
• patients who have previously received allogeneic hematopoietic stem cell transplantation or solid organ transplantation;
• Uncontrolled bacterial, viral, or fungal infections;
• HIV infection or active viral hepatitis B or C infection;
• Pregnant or breastfeeding women;
• patients with a history of primary malignancy within 3 years prior to transplant treatment;
• patients receiving ATG treatment within 2 weeks before transplantation;
• patients with drug dependent or uncontrolled mental illness or cognitive impairment;
• Participants in other similar clinical studies within 3 months;
• Patients allergic to ATG;

Sponsors & Collaborators

• Anhui Provincial Hospital: lead

Study Sites (1)

The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)

Hefei, Anhui, 230036, China

RECRUITING

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

Antilymphocyte Serum

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures

Study Officials

• Xiaoyu Zhu, ph.D

  The First Affiliated Hospital of University of Science and Technology of China (Anhui Provicial Hospital)

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiaoyu Zhu, ph.D

CONTACT

15255456091; xiaoyuz@ustc.edu.cn

Baolin Tang, M.D

CONTACT

13856901277; zwcrystalbl@163.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 12 Months
• Sponsor Type: OTHER GOV
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief physician

Study Record Dates

• First Submitted: September 9, 2023
• First Posted: September 15, 2023
• Study Start: October 1, 2023
• Primary Completion: April 30, 2025
• Study Completion: May 31, 2025
• Last Updated: October 24, 2023

Record last verified: 2023-10

Locations

CN (1)