TumorsNCT00179764
Immunoablative Mini Transplant (Hematopoietic Peripheral Blood Stem Cell Transplant [HPBSC])
A study testing Immunoablative Hematopoietic PBSC Transplant, Busulfan pharmacokinetics and Central Nervous System (CNS) prophylaxis radiation in people with Tumors and Malignant Melanoma, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
Trial summary
The purpose of this research study is to evaluate the effectiveness of transplantation of high doses of peripheral blood stem cells (stem cells are special cells found in the blood and bone marrow that produce new blood cells) after treatment with non-myeloablative chemotherapy (not toxic to the bone marrow). Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Tumors, Malignant Melanoma, Hematological Malignancies
- What is being tested?
- Immunoablative Hematopoietic PBSC Transplant, Busulfan pharmacokinetics and Central Nervous System (CNS) prophylaxis radiation
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 68 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Mar 2000to 24 Jan 2014
- 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 Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients with recurrent solid tumors
- Patients with malignant melanoma
- Patients with hematological malignancies.
- Chronic myelogenous leukemia in chronic or accelerated phase, to include chronic myelomonocytic leukemia (juvenile chronic myelogenous leukemia (JCML) or CMML).
- Acute lymphoblastic leukemia (ALL)
- First remission high-risk ALL (Ph+ with initial high white blood cell (WBC)t (4-11) in infants less than 1 year and CALLA negative)
Show all 25Show fewer
- Second or subsequent remission ALL or isolated extramedullary disease on or off therapy.
- Acute non-lymphocytic leukemia (ANLL)
- Patients with ANLL in first remission who have a matched sibling donor.
- ANLL in second remission, or patients who only achieve an initial partial remission < 15% blasts, or early relapse.
- Myelodysplastic syndromes (MDS): refractory anemia (RA), refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T) and CMML/JCML.
- Selected immunodeficiencies:
- Wiskott-Aldrich syndrome.
- Severe combined immunodeficiency variants that require ablation.
- Hyper-Immunoglobulin M (IgM) syndrome.
- Other immune deficiencies after approval from the medical director.
- Bone marrow failure syndromes (single or multiple hematopoietic lines)
- Venous access: A double lumen central vascular access device or its equivalent will be required for all patients entered on the protocol.
- Informed consent: The donor and the patient and/or the patient's legally authorized guardian must acknowledge in writing that consent to become a study subject has been obtained in accordance with the institutional policy approved by the United States (U.S.) Department of Health and Human Services.
- Patient organ function requirements:
- Adequate renal function: serum creatinine < 2 x normal, or creatinine clearance calculated by Schwartz formula, of glomerular filtration rate (GFR) > 40 ml/min/1.73m2, or an equivalent GFR as determined by the institutional normal range.
- Adequate liver function: total bilirubin </= 2 x normal; and Aspartate aminotransferase (AST) or Alanine aminotransferase (ALT) </= 4 x normal.
- Adequate cardiac function: shortening fraction of > 24% by echocardiogram, or ejection fraction of > 30% by radionuclide angiogram.
- Adequate pulmonary function: Diffusion Lung Capacity Carbon Monoxide (DLCO), Forced Expiratory Volume in 1 second (FEV1) / Forced Vital Capacity (FVC) > 30% by pulmonary function test. For children who are uncooperative for pulmonary function tests and have no evidence of dyspnea at rest or exercise intolerance, pulse oximetry > 94% on room air is considered acceptable.
- Performance status: Lansky Score >/= 60% for children </= 16 years of age; or Karnofsky > 60% status for those > 16 years of age.
Who cannot join
- Patients who are pregnant
- Inability to find a suitable donor for the patient
- Patient is HIV-positive
- Patient has active Hepatitis B
- Disease progression or relapse prior to HPC infusion
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Immunoablative Protocol for Allogeneic Related and Unrelated Hematopoietic Peripheral Blood Stem Cell Transplant (HPBSC)
The purpose of this research study is to evaluate the effectiveness of transplantation of high doses of peripheral blood stem cells (stem cells are special cells found in the blood and bone marrow that produce new blood cells) after treatment with non-myeloablative chemotherapy (not toxic to the bone marrow). In addition, this study will assess the side effects of the transplant.
Read the detailed description
The standard treatment in many disorders of the bone marrow is high dose chemotherapy and whole-body radiation treatment followed by the stem cell transplant. This type of transplant not only suppresses or kills off the immune system, but is very toxic to the bone marrow. This study uses a chemotherapy regimen that will suppress the patient's immune system; however, it is non-myeloablative (not toxic to the bone marrow). It does not use whole-body radiation treatment. This approach can minimize the short- and long-term effects of transplantation. Other studies have shown that using chemotherapy followed by bone marrow transplantation without whole-body radiation can produce similar results as treatment with whole-body radiation.
Patients will be given chemotherapy with Fludarabine and Busulfan prior to the stem cell transplant. This treatment not only destroys diseased cells, but it also kills normal bone marrow cells. Following this experimental treatment, the patient will be given the stem cells through a central venous catheter (tube inserted in a vein). When the healthy stem cells are given to the patient, they will replace the destroyed bone marrow cells and produce new blood cells. The Allogeneic (not one's own) stem cells used in this experimental transplant will be obtained from a related matched donor or from an unrelated matched donor located through the National Marrow Donor Program.
- Conditions
- Tumors, Malignant Melanoma, Hematological Malignancies, Myelogenous Leukemia, Chronic, Leukemia, Lymphoblastic, Acute
- Being tested
- Immunoablative Hematopoietic PBSC Transplant, Busulfan pharmacokinetics, Central Nervous System (CNS) prophylaxis radiation
- Keywords
- Patients with recurrent solid tumors, Patients with malignant melanoma, Patients with hematological malignancies, Acute lymphoblastic leukemia (ALL), Acute myelogenous leukemia (AML)
- Medical subject headings
- Neoplasms, Melanoma, Hematologic Neoplasms, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Immunoablative Hematopoietic PBSC TransplantProcedure or surgery
Immunoablative conditioning chemotherapy regimen, followed by transplantation of peripheral blood stem cells on Day 0 of the conditioning regimen.
Busulfan pharmacokineticsProcedure or surgery
Pharmacokinetics of once-a-day dosing of intravenous busulfan as a 3-hour infusion
Central Nervous System (CNS) prophylaxis radiationRadiation
- Patients diagnosed with ALL over 1 year of age and without prior CNS disease will receive CNS prophylaxis radiation to the whole brain prior to transplant.
- Patients diagnosed with ALL with prior CNS disease over the age of 1 year will be treated with prophylaxis radiation to the whole brain and spine prior to transplant.
| Study group | Role | What they receive |
|---|---|---|
| Reduced Intensity Conditioning Regimen | Other | Not described Receives: Procedure: Immunoablative Hematopoietic PBSC Transplant; Procedure: Busulfan pharmacokinetics; Radiation: Central Nervous System (CNS) prophylaxis radiation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate the morbidity and mortality of matched related and unrelated hematopoietic progenitor cell (HPC) transplantation at Children's Memorial Hospital using high dose CD34+ HPCs after a reduced intensity conditioning regimen.
Measured: To study end
Determine the toxicity of a reduced intensity conditioning regimen consisting of Fludarabine and Busulfan.
Measured: To study end
Secondary outcomes (4)
Validate the pharmacokinetics of once-a-day dosing of intravenous Busulfan given as a 3-hour infusion, using a limited number of samples.
Measured: To study end
Assess chimeric engraftment utilizing this regimen in malignant and non-malignant disorders.
Measured: To study end
Assess the relapse rate of patients transplanted with this reduced intensity regimen.
Measured: To study end
Determine the incidence of acute and chronic Graft vs. Host Disease (GVHD) using prophylaxis with Cyclosporine A and mycophenolate mofetil following this reduced intensity regimen.
Measured: To study end
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2005 (estimated)
- Study start10 Mar 2000
- Primary completion24 Jan 2014
- Study completion24 Jan 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for TumorsStudies 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
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 68 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Reggie E Duerst, MD, Ann & Robert H Lurie Children's Hospital of Chicago
- Investigators
- Morris Kletzel, M.D., Principal investigator, Ann & Robert H Lurie Children's Hospital of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00179764
- Sponsor's protocol ID
- BMT 0300 Mini
- Status last verified
- Aug 2019
- First submitted
- 10 Sep 2005
- Last updated
- 30 Aug 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Aug 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Immunoablative Protocol for Allogeneic Related and Unrelated Hematopoietic Peripheral Blood Stem Cell Transplant (HPBSC)
- ClinicalTrials.gov ID
- NCT00179764
- Acronym / sponsor ID
- BMT 0300 Mini
- Conditions
- Tumors, Malignant Melanoma, Hematological Malignancies, Myelogenous Leukemia, Chronic, Leukemia, Lymphoblastic, Acute
- Keywords
- Patients with recurrent solid tumors, Patients with malignant melanoma, Patients with hematological malignancies, Acute lymphoblastic leukemia (ALL), Acute myelogenous leukemia (AML)
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 68 participants (actual)
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of ChicagoUniversity, hospital or other
- Study start
- 10 Mar 2000 (actual)
- Primary completion
- 24 Jan 2014 (actual)
- Study completion
- 24 Jan 2014 (actual)
- First posted
- 16 Sep 2005 (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.
The purpose of this research study is to evaluate the effectiveness of transplantation of high doses of peripheral blood stem cells (stem cells are special cells found in the blood and bone marrow that produce new blood cells) after treatment with non-myeloablative chemotherapy (not toxic to the bone marrow). In addition, this study will assess the side effects of the transplant.
Detailed description
The standard treatment in many disorders of the bone marrow is high dose chemotherapy and whole-body radiation treatment followed by the stem cell transplant. This type of transplant not only suppresses or kills off the immune system, but is very toxic to the bone marrow. This study uses a chemotherapy regimen that will suppress the patient's immune system; however, it is non-myeloablative (not toxic to the bone marrow). It does not use whole-body radiation treatment. This approach can minimize the short- and long-term effects of transplantation. Other studies have shown that using chemotherapy followed by bone marrow transplantation without whole-body radiation can produce similar results as treatment with whole-body radiation.
Patients will be given chemotherapy with Fludarabine and Busulfan prior to the stem cell transplant. This treatment not only destroys diseased cells, but it also kills normal bone marrow cells. Following this experimental treatment, the patient will be given the stem cells through a central venous catheter (tube inserted in a vein). When the healthy stem cells are given to the patient, they will replace the destroyed bone marrow cells and produce new blood cells. The Allogeneic (not one's own) stem cells used in this experimental transplant will be obtained from a related matched donor or from an unrelated matched donor located through the National Marrow Donor Program.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Reduced Intensity Conditioning Regimen | Other | Procedure: Immunoablative Hematopoietic PBSC Transplant, Procedure: Busulfan pharmacokinetics, Radiation: Central Nervous System (CNS) prophylaxis radiation |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (25 listed)
- Patients with recurrent solid tumors
- Patients with malignant melanoma
- Patients with hematological malignancies.
- Selected immunodeficiencies:
- Bone marrow failure syndromes (single or multiple hematopoietic lines)
Key exclusion criteria (5 listed)
- Patients who are pregnant
- Inability to find a suitable donor for the patient
- Patient is HIV-positive
- Patient has active Hepatitis B
- Disease progression or relapse prior to HPC infusion
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 |
|---|---|---|
| Immunoablative Hematopoietic PBSC TransplantImmunoablative conditioning chemotherapy regimen, followed by transplantation of peripheral blood stem cells on Day 0 of the conditioning regimen. | Procedure or surgery | Reduced Intensity Conditioning Regimen |
| Busulfan pharmacokineticsPharmacokinetics of once-a-day dosing of intravenous busulfan as a 3-hour infusion | Procedure or surgery | Reduced Intensity Conditioning Regimen |
| Central Nervous System (CNS) prophylaxis radiation* Patients diagnosed with ALL over 1 year of age and without prior CNS disease will receive CNS prophylaxis radiation to the whole brain prior to transplant. * Patients diagnosed with ALL with prior CNS disease over the age of 1 year will be treated with prophylaxis radiation to the whole brain and spine prior to transplant. | Radiation | Reduced Intensity Conditioning Regimen |
Primary outcome
Evaluate the morbidity and mortality of matched related and unrelated hematopoietic progenitor cell (HPC) transplantation at Children's Memorial Hospital using high dose CD34+ HPCs after a reduced intensity conditioning regimen.
Time frame: To study end
Determine the toxicity of a reduced intensity conditioning regimen consisting of Fludarabine and Busulfan.
Time frame: To study end
4 secondary outcomes
Validate the pharmacokinetics of once-a-day dosing of intravenous Busulfan given as a 3-hour infusion, using a limited number of samples.
Time frame: To study end
Assess chimeric engraftment utilizing this regimen in malignant and non-malignant disorders.
Time frame: To study end
Assess the relapse rate of patients transplanted with this reduced intensity regimen.
Time frame: To study end
Determine the incidence of acute and chronic Graft vs. Host Disease (GVHD) using prophylaxis with Cyclosporine A and mycophenolate mofetil following this reduced intensity regimen.
Time frame: To study end
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 30 Aug 2019.
Related trials for Tumors
Not yet recruitingPhase 1
Hepatic Artery Infusion of Carfilzomib in Participants With Liver Metastatic Disease Previously Treated With Hepatic Artery Infusion Pump Therapy
Colorectal Neoplasms, Neoplasms
RecruitingPhase 2
Tafasitamab With Acalabrutinib and Venetoclax for the Treatment of Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
Chronic Lymphocytic Leukemia, Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
Enrolling by invitationPhase 2
A Single-Arm Study of Nimotuzumab Combined With NALIRIFOX as First-Line Treatment for Pancreatic Cancer
Pancreatic Cancer Metastatic
Recruiting
Methylation Profiling in Self-Samples for Identification of (Epi)Genetic Biomarkers for Gynecological Cancers
Gynaecological Cancers, Ovarian Cancer
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.
Morris Kletzel, M.D. principal investigator
Ann & Robert H Lurie Children's Hospital of Chicago
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Immunoablative Hematopoietic PBSC Transplant, Busulfan pharmacokinetics and Central Nervous System (CNS) prophylaxis radiation in people with Tumors and Malignant Melanoma, run by Ann & Robert H Lurie Children's Hospital of Chicago 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?
The sponsor lists 25 inclusion and 5 exclusion criteria. 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 30 Aug 2019. 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.
What does the primary outcome measure?
Evaluate the morbidity and mortality of matched related and unrelated hematopoietic progenitor cell (HPC) transplantation at Children's Memorial Hospital using high dose CD34+ HPCs after a reduced intensity conditioning regimen. (time frame: To study end). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
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 30 Aug 2019.