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Register:	ClinicalTrials.gov
Last refreshed on:	19 February 2015
Main ID:	NCT00006056
Date of registration:	05/07/2000
Prospective Registration:	No
Primary sponsor:	Fairview University Medical Center
Public title:	Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders
Scientific title:
Date of first enrolment:	March 2000
Target sample size:	40
Recruitment status:	Active, not recruiting
URL:	http://clinicaltrials.gov/show/NCT00006056
Study type:	Interventional
Study design:	Primary Purpose: Treatment
Phase:	N/A

Countries of recruitment
United States

Contacts

Name:	K. Scott Baker
Address:
Telephone:
Email:
Affiliation:	Fairview University Medical Center

Key inclusion & exclusion criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

Patients diagnosed with any of the following active but stable, or nonactive/quiescent,
hemophagocytic disorders:

- Hemophagocytic lymphohistiocytosis (HLH)

- Fever greater than 38.5 degrees Celsius

- Splenomegaly (greater than 3 cm below costal margin)

- Hemophagocytosis in bone marrow or spleen or lymph nodes

- Disease may be confirmed by positive family history

- No evidence of malignancy

- Hypertriglyceridemia and/or hypofibrinogenemia

- Fasting triglycerides at least 2.0 mmol/L or at least 3 standard deviations above
normal for age

- Fibrinogen no greater than 1.5 g/L or no greater than 3 standard deviations above
normal

- Cytopenia (affecting at least 2 of 3 lineages in the peripheral blood)

- Hemoglobin less than 9.0 g/L

- Platelet count less than 100,000/mm3

X-linked lymphoproliferative disorder (XLP)

Two or more maternally related males manifesting at least one of the following XLP
phenotypes:

- Fulminant infectious mononucleosis

- Dysgammaglobulinemia

- Malignant lymphoma/lymphoproliferative disorder

- Aplastic anemia

- Lymphoid granulomatosis/vasculitis OR

- A maternally related male in an established XLP kindred who has strong genetic (RFLP)
linkage to the XLP locus

Chediak-Higashi syndrome

Partial oculocutaneous albinism (hair, skin, eyes)

Frequent bacterial infections

Large peroxidase positive granules in leukocytes of peripheral blood or bone marrow

Positive family history or parental consanguinity is supportive of the diagnosis

May not have entered accelerated phase as defined by any of the following:

- Lymphadenopathy

- Pancytopenia

- Histiocytes with hemophagocytosis in bone marrow, lymph nodes, liver, or spleen

Viral associated hemophagocytic syndrome (VAHS)

Relapsed after prior therapy or supportive care

Diagnostic criteria as for HLH

No hemophagocytic disorders secondary to underlying malignancy

Patients 35 years of age and under must have a hematopoietic stem cell donor that is one
of the following:

- HLA A and B identical OR

- Single HLA A or B serologic mismatch with DRB1 identity OR

- HLA A or B serologic identity with a single DRB1 mismatch

Patients 36 to 55 years of age must have a hematopoietic stem cell donor that is one of
the following:

- HLA A and B and HLA DRB1 identical OR

- Single HLA A or B serologic mismatch with DRB1 identity

Patients receiving umbilical cord blood must have an unrelated donor with no more than two
antigen HLA A, B, or DRB1 mismatches

--Patient Characteristics--

Performance status: Karnofsky 70-100% OR Age less than 16 years: Lansky 50-100%

Life expectancy: Not severly limited by another disease

Hepatic: SGOT less than 3 times normal Bilirubin less than 2.5 mg/dL

Renal: Creatinine normal OR Creatinine clearance or glomerular filtration rate greater
than 50% normal

Cardiovascular: If symptomatic, ventricular ejection fraction must be greater than 40% and
must improve with exercise OR Shortening fraction normal on echocardiogram

Pulmonary:

- If symptomatic, DLCO greater than 45% predicted (corrected for hemoglobin)

- In children unable to perform pulmonary function testing, oxygen saturation must be
greater than 95%

Other: HIV negative No significant active infections

Age minimum: N/A
Age maximum: 55 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Graft Versus Host Disease

Familial Erythrophagocytic Lymphohistiocytosis

Hemophagocytic Lymphohistiocytosis

Virus-Associated Hemophagocytic Syndrome

X-Linked Lymphoproliferative Syndrome

Chediak-Higashi Syndrome

Intervention(s)

Drug: anti-thymocyte globulin

Drug: busulfan

Drug: cyclosporine

Drug: cyclophosphamide

Drug: filgrastim

Procedure: allogeneic hematopoietic stem cell transplantation

Drug: etoposide

Drug: methotrexate

Primary Outcome(s)

Secondary Outcome(s)

Secondary ID(s)

199/15106

UMN-MT-1997-08

UMN-MT-9708

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Ethics review

Results

Results available:
Date Posted:
Date Completed:
URL:

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