Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia

NCT00578266 | Completed Phase 1 DMC

• 1 other identifier: 06-006216
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief Summary

For patients with severe aplastic anemia (SAA) who have failed to respond to immunosuppressive therapy and lack an HLA identical family member, our objectives are to make an initial assessment of the safety and efficacy of allogenic stem cell transplantation from either a matched unrelated donor or a mismatched reacted donor using the conditioning regimen of Cytoxan, reduced total body irradiation (TBI) and Campath IH. The principle measures of safety and efficacy will be :

1. 1.Patient survival probability at 100 days, 1 year and 2 years.
2. 2.Incidence of graft versus host disease (GVHD), as well as incidence of acute GVHD and chronic GVHD within 6 months and 2 years.
3. 3.Engraftment at 6 months, 1 year and 2 years

Conditions

Anemia, Aplastic

Keywords

Severe Aplastic Anemia (SAA); Transplant for Severe Aplastic Anemia (SAA)

Outcome Measures

Primary Outcomes (1)

• 1. Engraftment at 6 months, 1 year and 2 years 2.Incidence of graft versus host disease (GVHD), as well as incidence of acute GVHD and chronic GVHD within 6 months and 2 years

  2 years

Secondary Outcomes (1)

• Patient survival probability at 100 days, 1 year and 2 years.

  2 years

Study Arms (1)

No Arms

OTHER

Drug: Cyclophosphamide,Campath IH and TBI

Interventions

Cyclophosphamide,Campath IH and TBI DRUG

DAY 5 TREATMENT * 6 5 CYTOXAN 50 mg/kg WITH MESNA * 5 CYTOXAN 50 mg/kg WITH MESNA; * 4 CYTOXAN 50 mg/kgWITH MESNA; CAMPATH 3-10 mg * 3 CYTOXAN 50 mg/kg WITH MESNA; CAMPATH; * 2 TBI; CAMPATH; TACROLIMUS * 1 TBI (second fraction); CAMPATH (am) 0 STEM CELL INFUSION (pm)

No Arms

Eligibility Criteria

• Age: Up to 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosis of SAA based on bone marrow aspirate and biopsy results. Failure to respond to immunosuppressive therapy and/or lack of an HLA identical family member.
• A 10/10 or 9/10 HLA matched unrelated donor or a 9/10 matched related donor available after high resolution typing.

You may not qualify if:

• Patients with Aplastic anemia and active infection must be treated to maximally resolve this problem before beginning the conditioning regimen.
• HIV seropositive patients
• Patients who have clonal cytogenetic abnormalities or a myelodysplastic syndrome.
• Patient greater than 60 years of age.
• Women who are pregnant or nursing.
• Patients with active hepatitis
• Patients with severe cardiac dysfunction defined as shortening fraction \<25%.
• Patients with severe renal dysfunction defined as creatinine clearance \<40ml/mim/1.73m2.
• Patient with severe pulmonary dysfunction with FEV1, FVC and DLCO 40% of predicted or 3 SD below normal.

Sponsors & Collaborators

• Mayo Clinic: lead

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

Cyclophosphamide; Whole-Body Irradiation

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Radiotherapy; Therapeutics; Investigative Techniques

Study Officials

• Shakila P. Khan, M.D.

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: December 17, 2007
• First Posted: December 21, 2007
• Study Start: February 1, 2007
• Primary Completion: August 16, 2016
• Study Completion: August 16, 2016
• Last Updated: March 24, 2017

Record last verified: 2017-03

Locations

US (1)