Immune Disorder HSCT Protocol

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-03-31

Study Completion Date

2027-03-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Immune Deficiency Disorders Severe Combined Immunodeficiency Chronic Granulomatous Disease X-linked Agammaglobulinemia Wiskott-Aldrich Syndrome Hyper-IgM DiGeorge Syndrome Chediak-Higashi Syndrome Common Variable Immune Deficiency Immune Dysregulatory Disorders Hemophagocytic Lymphohistiocytosis IPEX Autoimmune Lymphoproliferative Syndrome X-linked Lymphoproliferative Syndrome

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Preparative

Group Type EXPERIMENTAL

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

Intervention Type DRUG

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

Interventions

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

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

Intervention Type DRUG

Eligibility Criteria

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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

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No