MedTrace Logo

Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation

NCT01319851 · Status: TERMINATED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2017-07-27

Study results available
· View outcomes & findings →

Summary

Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications. Much of the risk stems from the use of high dose radiation and chemotherapy for conditioning, the treatment administered just prior to transplant that eliminates the patients' marrow and immune system, effectively preventing rejection of the donors' cells. Attempts to make blood and marrow transplantation safer for children with non-malignant diseases by using lower doses of radiation and chemotherapy have largely failed because of a high rate of graft rejection.

In many such cases, it is likely that the graft is rejected because the recipient is sensitized to proteins on donor cells, including bone marrow cells, by blood transfusions. The formation of memory immune cells is a hallmark of sensitization, and these memory cells are relatively insensitive to chemotherapy and radiation. Alefacept, a drug used to treat psoriasis, on the other hand, selectively depletes these cells. The investigators are conducting a pilot study to begin to determine whether incorporating alefacept into a low dose conditioning regimen can effectively mitigate sensitization and, thereby, prevent rejection of allogeneic blood and marrow transplants for multiply transfused children with non-malignant hematological diseases.

Conditions

  • Thalassemia
  • Sickle Cell Disease
  • Glanzmann Thrombasthenia
  • Wiskott-Aldrich Syndrome
  • Chronic-granulomatous Disease
  • Severe Congenital Neutropenia
  • Leukocyte Adhesion Deficiency
  • Schwachman-Diamond Syndrome
  • Diamond-Blackfan Anemia
  • Fanconi Anemia
  • Dyskeratosis-congenita
  • Chediak-Higashi Syndrome
  • Severe Aplastic Anemia

Interventions

DRUG

Alefacept

0.25 mg/kg IV on day -40 and day -39 0.5 mg/kg IV on days -33, -26, -19 and -12 Alefacept was diluted in sterile water (2 ml total volume) and administered via i.v. push followed by a normal saline flush per package insert.

Sponsors & Collaborators

  • Children's Healthcare of Atlanta

    collaborator OTHER

  • Emory University

    lead OTHER

Principal Investigators

  • John Horan, MD · Emory University/Children's Healthcare of Atlanta

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-09-30
Primary Completion
2013-09-30
Completion
2013-09-30

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01319851 on ClinicalTrials.gov