Cord Blood Transplantation in Severe Aplastic Anemia (APCORD)

Assistance Publique - Hôpitaux de Paris

Status and phase: Completed; Phase 2

Conditions: Hereditary Diseases; Acquired Aplastic Anemia; Relapse; Absence of an HLA Identical Donor

Treatments: Procedure: Cord Blood Transplantation

Study type: Interventional

Funder types: Other

Identifiers

NCT01343953

P090201

Details and patient eligibility

About

This is a Prospective Phase II Study to evaluate Cord Blood Transplantation in Inherited or Acquired Severe Aplastic Anemia Refractory or in Relapse after Immunosuppressive Therapy in the absence of an HLA identical donor;

Full description

This is a Prospective Phase II Study to evaluate Cord Blood Transplantation in Inherited or Acquired Severe Aplastic Anemia Refractory or in Relapse after Immunosuppressive Therapy in the absence of an Human Leucocyte Antigen (HLA) identical donor; 26 patients will be recruited, according to a one step Fleming design

Enrollment: 26 estimated patients

Sex: All

Ages: 3 to 55 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Age: 3-55 years old
• inherited aplastic anemia (with severe aplastic anemia criteria) or acquired aplastic anemia in relapse or treatment failure after immunosuppressive therapy without clonal evolution
• Karnovsky Index >= 60%
• Informed consent

Exclusion criteria

• HLA identical donor

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

26 participants in 1 patient group

Cord Blood Transplantation

Experimental group

Treatment:

Procedure: Cord Blood Transplantation