Chediak-Higashi SyndromeNCT00006056
Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders
A study testing anti-thymocyte globulin, busulfan and cyclophosphamide in people with Chediak-Higashi Syndrome and Graft Versus Host Disease, run by Fairview University Medical Center at 1 site in United States.
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… Read more about this study
- Who is it for?
- Ages 0 to 55 years
- Conditions the study is for
- Chediak-Higashi Syndrome, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome
- What is being tested?
- anti-thymocyte globulin, busulfan and cyclophosphamide
- Type of treatment
- Surgery or procedure
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2000to not given
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
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
Trial locations and contacts
1 site in 1 country.
United States1 site
Fairview University Medical Center
Minneapolis, Minnesota, 55455
About this study
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.
Read the detailed 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.
- Conditions
- Chediak-Higashi Syndrome, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, Virus-Associated Hemophagocytic Syndrome
- Being tested
- anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, etoposide
- Keywords
- Chediak-Higashi syndrome, X-linked lymphoproliferative syndrome, disease-related problem/condition, familial erythrophagocytic lymphohistiocytosis, genetic diseases and dysmorphic syndromes, graft versus host disease, hematologic disorders, hemophagocytic lymphohistiocytosis, histiocytosis, immunologic disorders and infectious disorders, primary immunodeficiency disease, rare disease, virus-associated hemophagocytic syndrome
- Medical subject headings
- Chediak-Higashi Syndrome, Graft vs Host Disease, Lymphoproliferative Disorders, Lymphohistiocytosis, Hemophagocytic, Genetic Diseases, Inborn, Hematologic Diseases, Histiocytosis, Immune System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-thymocyte globulinDrug
busulfanDrug
cyclophosphamideDrug
cyclosporineDrug
etoposideDrug
filgrastimDrug
methotrexateDrug
allogeneic hematopoietic stem cell transplantationProcedure or surgery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jul 2000 (estimated)
- Study startMar 2000
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chediak-Higashi SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Enrolment
- 40 participants
Sponsor and investigators
- Lead sponsor
- Fairview University Medical Center
- Sponsor type
- University, hospital or other
- Investigators
- K. Scott Baker, Study chair, Fairview University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00006056
- Sponsor's protocol ID
- 199/15106
- Other IDs
- UMN-MT-1997-08, UMN-MT-9708
- Status last verified
- Oct 2003
- First submitted
- 5 Jul 2000
- Last updated
- 24 Jun 2005
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2005. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders
- ClinicalTrials.gov ID
- NCT00006056
- Acronym / sponsor ID
- 199/15106
- Conditions
- Chediak-Higashi Syndrome, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, Virus-Associated Hemophagocytic Syndrome
- Keywords
- Chediak-Higashi syndrome, X-linked lymphoproliferative syndrome, disease-related problem/condition, familial erythrophagocytic lymphohistiocytosis, genetic diseases and dysmorphic syndromes, graft versus host disease, hematologic disorders, hemophagocytic lymphohistiocytosis
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants
- Lead sponsor
- Fairview University Medical CenterUniversity, hospital or other
- Study start
- Mar 2000
- First posted
- 6 Jul 2000 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 0 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| anti-thymocyte globulin | Drug | – |
| busulfan | Drug | – |
| cyclophosphamide | Drug | – |
| cyclosporine | Drug | – |
| etoposide | Drug | – |
| filgrastim | Drug | – |
| methotrexate | Drug | – |
| allogeneic hematopoietic stem cell transplantation | Procedure or surgery | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2005.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
K. Scott Baker study chair
Fairview University Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing anti-thymocyte globulin, busulfan and cyclophosphamide in people with Chediak-Higashi Syndrome and Graft Versus Host Disease, run by Fairview University Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 24 Jun 2005. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jun 2005.