World Health Organization site
Skip Navigation Links

Main
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 19 February 2015
Main ID:  NCT00006056
Date of registration: 05/07/2000
Prospective Registration: Yes
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
Chediak-Higashi Syndrome
Familial Erythrophagocytic Lymphohistiocytosis
Graft Versus Host Disease
Hemophagocytic Lymphohistiocytosis
Virus-Associated Hemophagocytic Syndrome
X-Linked Lymphoproliferative Syndrome
Intervention(s)
Drug: anti-thymocyte globulin
Drug: busulfan
Drug: cyclophosphamide
Drug: cyclosporine
Drug: etoposide
Drug: filgrastim
Drug: methotrexate
Procedure: allogeneic hematopoietic stem cell transplantation
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:
Disclaimer: Trials posted on this search portal are not endorsed by WHO, but are provided as a service to our users. In no event shall the World Health Organization be liable for any damages arising from the use of the information linked to in this section. None of the information obtained through use of the search portal should in any way be used in clinical care without consulting a physician or licensed health professional. WHO is not responsible for the accuracy, completeness and/or use made of the content displayed for any trial record.
Copyright - World Health Organization - Version 3.6 - Version history