Immune Disorder HSCT Protocol

The Washington University

Status and phase

Enrolling

Phase 2

Conditions

Autoimmune Lymphoproliferative Syndrome

Chediak-Higashi Syndrome

X-linked Agammaglobulinemia

Common Variable Immune Deficiency

Severe Combined Immunodeficiency

Hyper-IgM

X-linked Lymphoproliferative Syndrome

IPEX

DiGeorge Syndrome

Wiskott-Aldrich Syndrome

Immune Dysregulatory Disorders

Immune Deficiency Disorders

Hemophagocytic Lymphohistiocytosis

Chronic Granulomatous Disease

Treatments

Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan

Study type

Interventional

Funder types

Other

Identifiers

NCT01821781

201301135

Details and patient eligibility

About

This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders. A regimen that maximizes host immune suppression is expected to reduce graft rejection and optimize donor cell engraftment.

Enrollment

20 estimated patients

Sex

All

Ages

Under 21 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

</= 28 years of age
Performance status >/= 40
DLCO >/= 40%
LVEF >/=40% or LVSF >/=26%
Serum creatinine < 2x ULN
Liver enzymes </= 5x ULN
Negative pregnancy test
Suitably matched donor (6/6 matched sib UCB, 8/8 matched sib BM or PBSC, 5-6/6 matched unrelated UCB, 7-8/8 matched unrelated BM, double cord)

Exclusion criteria

Known diagnosis of HIV I/II
Pregnant or breastfeeding
Uncontrolled invasive fungal or bacterial infections within 1 month prior to starting alemtuzumab
Uncontrolled viral infection within 1 week prior to starting alemtuzumab