NeutropeniaNCT00005893
Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders
A study testing anti-thymocyte globulin, busulfan and cyclophosphamide in people with Neutropenia and Sickle Cell Anemia, run by Fairview University Medical Center at 1 site in United States.
Trial summary
OBJECTIVES: I. Determine the efficacy of bone marrow transplantation using matched related donors in patients with nonmalignant hematologic disorders. II. Determine the quality of life, absence of adverse effects (e.g., graft versus host disease and B cell lymphoproliferative disease), and completeness of recovery of… Read more about this study
- Who is it for?
- Ages 0 years and older
- Conditions the study is for
- Neutropenia, Sickle Cell Anemia, Thalassemia Major
- What is being tested?
- anti-thymocyte globulin, busulfan and cyclophosphamide
- Type of treatment
- Surgery or procedure
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Jun 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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Severe hemoglobinopathy including, but not limited to the following:
Homozygous beta thalassemia Other beta chain mutation as demonstrated by hemoglobin electrophoresis Sickle cell anemia (age 16 to 50 years) Evidence of one or more the following prior complications: Stage I-II sickle cell lung disease Sickle cell nephropathy (moderate to severe proteinuria or glomerular filtration rate of 30-50% predicted normal for age) Acute chest syndrome requiring blood transfusions More than 3 debilitating pain episodes per year for 3 years lasting more than 4 hours each Any combination of episodes of acute chest syndrome and painful episodes adding up to 3 episodes per year for 3 consecutive years Requirement for chronic transfusions with alloimmunization (more than 2 antibodies) Sickle cell anemia (age under 16 years) Evidence of one or more the following prior complications: Prior stroke or hemorrhage Any neurologic event lasting more than 24 hours Abnormal cerebral MRI and cerebral arteriogram MRI angiographic impaired neuropsychologic testing Stage I-II sickle cell lung disease Sickle cell nephropathy (moderate to severe proteinuria or glomerular filtration rate of 30-50% predicted normal for age) Significant visual impairment in at least one eye with bilateral proliferative retinopathy Acute chest syndrome with history of recurrent hospitalizations or exchange transfusions Osteonecrosis of multiple joints with destructive changes More than 3 debilitating pain episodes per year or priapism Requirement for chronic transfusions with alloimmunization
OR
Bone marrow failure syndrome unresponsive to therapy, including but not limited to the following: Congenital pure red cell aplasia (Diamond Blackfan anemia) Confirmed by bone marrow aspirate More than 6 transfusions per year despite steroid therapy Kostmann's neutropenia Confirmed by bone marrow aspirate Unable to maintain absolute neutrophil count greater than 750/mm3 or recurrent life threatening infections or more than 4 hospitalizations per year despite therapy with filgrastim (G-CSF) No myelodysplasia or aplastic anemia
Must have a related donor with at least a 5 out of 6 HLA antigen match
--Patient Characteristics--
Age: Sickle cell anemia patients 0 to 50; All other patients under 18
Performance status: Karnofsky 70-100%
Hepatic: Bilirubin no greater than 3.0 mg/dL with a direct fraction no greater than 2.0 mg/dL ALT no greater than 150 U/L No moderate or severe portal fibrosis No active hepatitis
Renal: Glomerular filtration rate at least 30% predicted (except with sickle cell anemia) No renal dysfunction
Cardiovascular: Left ejection fraction at least 50% No cardiac compromise
Other: No severe, stable neurologic impairment HIV negative
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 bone marrow transplantation using matched related donors in patients with nonmalignant hematologic disorders.
II. Determine the quality of life, absence of adverse effects (e.g., graft versus host disease and B cell lymphoproliferative disease), and completeness of recovery of their underlying condition in these patients with this treatment regimen.
Read the detailed description
PROTOCOL OUTLINE: Patients receive IV or oral busulfan every 6 hours on days -9 to -6; cyclophosphamide IV on days -5 to -2; anti-thymocyte globulin IV on days -4 to -2; and allogeneic bone marrow transplantation (BMT) on day 0.
Patients with class 3 thalassemia (liver edge greater than 2 cm below costal margin, a history of inconsistent chelation, and portal fibrosis) receive a less intensive conditioning regimen consisting of oral busulfan every 6 hours on days -7 to -4; anti-thymocyte globulin IV on days -5 to -1 and days 1-5; cyclophosphamide IV on days -3 to -2; and allogeneic BMT on day 0.
Patients are followed at day 28, and then at 3, 6, 12, and 24 months.
- Being tested
- anti-thymocyte globulin, busulfan, cyclophosphamide, Bone marrow transplantation
- Keywords
- chronic congenital neutropenia, chronic neutropenia, congenital pure red cell aplasia, disease-related problem/condition, genetic diseases and dysmorphic syndromes, hematologic disorders, neutropenia, pure red cell aplasia, rare disease, sickle cell anemia, thalassemia major
- Medical subject headings
- Neutropenia, Anemia, Sickle Cell, beta-Thalassemia, Red-Cell Aplasia, Pure, Anemia, Diamond-Blackfan, Genetic Diseases, Inborn, Hematologic Diseases, Rare 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
Bone marrow 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 posted5 Jun 2000 (estimated)
- Study startJun 2000
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeutropeniaStudies 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
Sponsor and investigators
- Lead sponsor
- Fairview University Medical Center
- Sponsor type
- University, hospital or other
- Investigators
- Paul J. Orchard, Study chair, Fairview University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00005893
- Sponsor's protocol ID
- 199/15101
- Other IDs
- UMN-MT-1994-18, UMN-MT-9418
- Status last verified
- Jul 2004
- First submitted
- 2 Jun 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
- Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders
- ClinicalTrials.gov ID
- NCT00005893
- Acronym / sponsor ID
- 199/15101
- Keywords
- chronic congenital neutropenia, chronic neutropenia, congenital pure red cell aplasia, disease-related problem/condition, genetic diseases and dysmorphic syndromes, hematologic disorders, neutropenia, pure red cell aplasia
- Study type
- Interventional
- Recruitment status
- Completed
- Lead sponsor
- Fairview University Medical CenterUniversity, hospital or other
- Study start
- Jun 2000
- First posted
- 5 Jun 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 bone marrow transplantation using matched related donors in patients with nonmalignant hematologic disorders.
II. Determine the quality of life, absence of adverse effects (e.g., graft versus host disease and B cell lymphoproliferative disease), and completeness of recovery of their underlying condition in these patients with this treatment regimen.
Detailed description
PROTOCOL OUTLINE: Patients receive IV or oral busulfan every 6 hours on days -9 to -6; cyclophosphamide IV on days -5 to -2; anti-thymocyte globulin IV on days -4 to -2; and allogeneic bone marrow transplantation (BMT) on day 0.
Patients with class 3 thalassemia (liver edge greater than 2 cm below costal margin, a history of inconsistent chelation, and portal fibrosis) receive a less intensive conditioning regimen consisting of oral busulfan every 6 hours on days -7 to -4; anti-thymocyte globulin IV on days -5 to -1 and days 1-5; cyclophosphamide IV on days -3 to -2; and allogeneic BMT on day 0.
Patients are followed at day 28, and then at 3, 6, 12, and 24 months.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 0 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older 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 | – |
| Bone marrow transplantation | Procedure or surgery | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2005.
Related trials for Neutropenia
Enrolling by invitationObservational study
Screening for Hematology Branch Protocols
Hematologic Disease and Disorders, Donors
RecruitingObservational study
Pivotal Study to Validate a Novel System to Non-Invasively Detect Severe Neutropenia
Diffuse Large B Cell Lymphoma, Multiple Myeloma
RecruitingPhase 4
Adjustment of Antibiotic Dosage in Pediatric Oncology Patients With Febrile Neutropenia and Augmented Renal Clearance
Febrile Neutropenia
Not yet recruitingPhase 2
A Study to Investigate the Safety and Tolerability of Granulocyte-colony Stimulating Factor (G-CSF) Dosage Reduction in Mavorixafor-treated Participants With Acquired Primary Autoimmune and Idiopathic Chronic Neutropenia Disorders
Acquired Primary Autoimmune and Idiopathic Chronic Neutropenia Disorders, Chronic Neutropenia
Not yet recruiting
Diagnostic Accuracy, Clinical Utility and Implementation of Point-of-Care Absolute Neutrophil Count in Suspected Neutropenic Fever (DUCATIS) - Diagnostic Accuracy (S1)
Suspected Neutropenic Fever, Confirmed Neutropenic Fever
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.
Paul J. Orchard 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 Neutropenia and Sickle Cell Anemia, 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: finished, 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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.