Immune Disorder HSCT Protocol

Phase 2RecruitingNCT01821781

Washington University School of Medicine20 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Immune Deficiency Disorders Severe Combined Immunodeficiency Chronic Granulomatous Disease X-linked Agammaglobulinemia Wiskott-Aldrich Syndrome

Interventions

Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalanDRUG

Between days -23 and -15: alemtuzumab test dose, 3mg IV or SQ Day -14: alemtuzumab, 10mg IV or SQ Day -13: alemtuzumab, 15mg IV or SQ Day -12: alemtuzumab, 20mg IV or SQ Days -8 to -4: fludarabine, 30mg/m2 IV Day -4: thiotepa 4mg/kg IV q 12 hours Day -3: melphalan, 140mg/m2 IV Day 0: stem cell infusion Day +7: G-CSF

Eligibility

Sex: ALLMax age: 21 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

Number of participants with donor engraftment

Time frame: 1 year post transplant

Secondary Outcomes

Major Transplant Related Toxicities

Time frame: 1 years post transplant

Time to neutrophil recovery

Time frame: within 100 days post transplant

Number of patient with acute GVHD

Time frame: 180 days post transplant

Number of participants with infectious complications