Clinical Trials Logo

Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT00006056
Other study ID # 199/15106
Secondary ID UMN-MT-1997-08UM
Status Active, not recruiting
Phase N/A
First received July 5, 2000
Last updated June 23, 2005
Start date March 2000

Study information

Verified date October 2003
Source Office of Rare Diseases (ORD)
Contact n/a
Is FDA regulated No
Health authority Unspecified
Study type Interventional

Clinical Trial Summary

OBJECTIVES: I. Determine the efficacy of unrelated donor hematopoietic stem cell transplantation in the treatment of patients with life threatening hemophagocytic disorders.

II. Determine the rate of disease free survival, incidence of graft failure, and incidence of graft versus host disease in these patients after undergoing this treatment regimen.


Description:

PROTOCOL OUTLINE: Patients receive oral busulfan twice a day on days -9 to -6; cyclophosphamide IV over 1 hour on days -5 to -2; etoposide IV over 4 hours on days -5 to -3; and anti-thymocyte globulin IV twice a day on days -2 and -1 and days 1 and 2. Patients undergo allogeneic hematopoietic stem cell transplantation on day 0. Filgrastim (G-CSF) is administered subcutaneously beginning on day 1 and continuing until blood counts recover. Patients receive graft versus host disease prophylaxis with methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1-4 hours (orally once the patients resumes eating) every 12 hours (every 8 hours for pediatric patients) starting on or prior to day -3 and continuing up to 1 year.

Patients are followed at days 28 and 100, at 6 months and 1 year, and then annually for 5 years.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 40
Est. completion date
Est. primary completion date
Accepts healthy volunteers No
Gender Both
Age group N/A to 55 Years
Eligibility 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

Study Design

Primary Purpose: Treatment


Related Conditions & MeSH terms


Intervention

Drug:
anti-thymocyte globulin

busulfan

cyclophosphamide

cyclosporine

etoposide

filgrastim

methotrexate

Procedure:
allogeneic hematopoietic stem cell transplantation


Locations

Country Name City State
United States Fairview University Medical Center Minneapolis Minnesota

Sponsors (1)

Lead Sponsor Collaborator
Fairview University Medical Center

Country where clinical trial is conducted

United States, 

See also
  Status Clinical Trial Phase
Recruiting NCT03357159 - Anti T-lymphocyte Immunoglobulin With Post Transplant Cyclophosphamide to Prevent GVHD Post Allogeneic Transplantation Phase 2
Completed NCT00003270 - Chemotherapy, Radiation Therapy, and Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer Phase 2
Terminated NCT02877082 - Tacrolimus, Bortezomib, & Thymoglobulin in Preventing Low Toxicity GVHD in Donor Blood Stem Cell Transplant Patients Phase 2
Recruiting NCT01385124 - Cannabidiol for Graft Versus Host Disease (GVHD) Prophylaxis in Allogeneic Stem Cell Transplantation Phase 1/Phase 2
Withdrawn NCT01616680 - Brentuximab Vedotin in Treating Patients With Steroid-Resistant Acute Graft-Versus-Host Disease Phase 2
Recruiting NCT01810926 - T&B Depletion Non Malignant Phase 2
Completed NCT01379209 - Intravenous Administration of RGI-2001 in Patient Undergoing Allogenic Hematopoietic Stem Cell Transplantation (AHSCT) Phase 1/Phase 2
Completed NCT01233921 - Palifermin in Preventing Chronic Graft-Versus-Host Disease in Patients Who Have Undergone Donor Stem Cell Transplant for Hematologic Cancer N/A
Recruiting NCT00986557 - T-Lymphocyte Infusion or Standard Therapy in Treating Patients at Risk of Cytomegalovirus Infection After a Donor Stem Cell Transplant Phase 2
Enrolling by invitation NCT00972660 - Safety and Efficacy Study of Allogenic Mesenchymal Stem Cells to Treat Extensive Chronic Graft Versus Host Disease Phase 2
Terminated NCT00555048 - Alemtuzumab, Busulfan, and Cyclophosphamide Followed By a Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer Phase 1/Phase 2
Terminated NCT00373815 - Everolimus in Combination With Cyclosporine A and Prednisolone for the Treatment of Graft Versus Host Disease Phase 1
Terminated NCT00608517 - Treatment of Single or Double Umbilical Cord Trans + Graft-versus-host Disease (GVHD) Prophylaxis w/ Tacrolimus & Mycophenolate Mofetil N/A
Completed NCT00056875 - Recombinant Human Keratinocyte Growth Factor in Unrelated and Related Transplants Phase 1/Phase 2
Recruiting NCT05808985 - Intestinal Microbiome-based Research for the Prevention of Acute GVHD Phase 2
Completed NCT00813618 - Study of MEDI 507 in the Treatment of Pediatric Patients Phase 1
Completed NCT00003398 - Bone Marrow Transplantation in Treating Patients With Hematologic Cancer Phase 4
Terminated NCT00005641 - Removal of T Cells to Prevent Graft-Versus-Host Disease in Patients Undergoing Bone Marrow Transplantation Phase 2
Completed NCT02663622 - Phase II Trial of Efprezimod Alfa (CD24Fc, MK-7110) for the Prevention of Acute Graft-Versus-Host Disease (GVHD) Following Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) (MK-7110-002) Phase 2
Completed NCT00577278 - A Phase II Study of Allo-HCT for B-Cell NHL Using Zevalin, Fludarabine and Melphalan Phase 2