CompletedPhase 1

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

United States10 participantsStarted 2014-01

Plain-language summary

This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem cell transplant for serious non-malignant diseases as well as to assess the immunological effects of abatacept. Participants will be followed for 2 years.

Who can participate

Age range21 Years

SexALL

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Inclusion criteria

✓. Glanzmann thrombasthenia
✓. Wiskott-Aldrich syndrome or other combined immune deficiency
✓. Chronic-granulomatous disease
✓. Severe congenital neutropenia (with resistance to granulocyte-colony stimulating factor (GCSF) or chronic requirement of GCSF doses ≥10 mcg/kg)
✓. Leukocyte adhesion deficiency
✓. Shwachman-Diamond syndrome
✓. Diamond-Blackfan anemia ((transfusion dependent, including steroid failure or inability to wean steroids)
✓. Thalassemia major

What they're measuring

Tolerability of Abatacept

Timeframe: 1 year post-transplant

Trial details

NCT IDNCT01917708

SponsorEmory University

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2019-09-19

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