Clinical Trials Logo

Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00179764
Other study ID # BMT 0300 Mini
Secondary ID
Status Completed
Phase N/A
First received
Last updated
Start date March 10, 2000
Est. completion date January 24, 2014

Study information

Verified date August 2019
Source Ann & Robert H Lurie Children's Hospital of Chicago
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical 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). In addition, this study will assess the side effects of the transplant.


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.


Recruitment information / eligibility

Status Completed
Enrollment 68
Est. completion date January 24, 2014
Est. primary completion date January 24, 2014
Accepts healthy volunteers No
Gender All
Age group N/A to 21 Years
Eligibility Inclusion Criteria:

- 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)

- 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.

Exclusion Criteria:

- 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

Study Design


Related Conditions & MeSH terms


Intervention

Procedure:
Immunoablative Hematopoietic PBSC Transplant
Immunoablative conditioning chemotherapy regimen, followed by transplantation of peripheral blood stem cells on Day 0 of the conditioning regimen.
Busulfan pharmacokinetics
Pharmacokinetics of once-a-day dosing of intravenous busulfan as a 3-hour infusion
Radiation:
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.

Locations

Country Name City State
United States Lurie Children's Hospital Chicago Illinois

Sponsors (1)

Lead Sponsor Collaborator
Ann & Robert H Lurie Children's Hospital of Chicago

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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. To study end
Primary Determine the toxicity of a reduced intensity conditioning regimen consisting of Fludarabine and Busulfan. To study end
Secondary Validate the pharmacokinetics of once-a-day dosing of intravenous Busulfan given as a 3-hour infusion, using a limited number of samples. To study end
Secondary Assess chimeric engraftment utilizing this regimen in malignant and non-malignant disorders. To study end
Secondary Assess the relapse rate of patients transplanted with this reduced intensity regimen. To study end
Secondary 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. To study end
See also
  Status Clinical Trial Phase
Completed NCT04229277 - Fast Track Diagnosis of Skin Cancer by Advanced Imaging N/A
Completed NCT03653819 - High Intensity Interval Training (HIIT) for Patients With Cancer-related Lymphedema in the Lower Limbs N/A
Active, not recruiting NCT04074096 - Binimetinib Encorafenib Pembrolizumab +/- Stereotactic Radiosurgery in BRAFV600 Melanoma With Brain Metastasis Phase 2
Completed NCT02935790 - Selective HDAC6 Inhibitor ACY-241 in Combination With Ipilimumab and Nivolumab Phase 1
Recruiting NCT05478876 - Carbon Ion Radiation Therapy in the Treatment of Mucous Melanomas of the Female Lower Genital Tract N/A
Completed NCT01211262 - Study to Assess the Tolerability of a Bispecific Targeted Biologic IMCgp100 in Malignant Melanoma Phase 1
Recruiting NCT03649529 - Treatment of Malignant Melanoma With GPA-TriMAR-T Cell Therapy Early Phase 1
Completed NCT03278665 - 4SC-202 in Combination With Pembrolizumab in Patients Primary Refractory/Non-responding to Prior Anti-PD-1 Therapy Phase 1/Phase 2
Completed NCT04452214 - A Study of the Safety and Tolerance of CAN04 and Pembrolizumab in Combination With and Without Carboplatin and Pemetrexed in Subjects With Solid Tumors Phase 1
Terminated NCT02709889 - Rovalpituzumab Tesirine in Delta-Like Protein 3-Expressing Advanced Solid Tumors Phase 1/Phase 2
Completed NCT01455259 - Phase I/IIa AdCD40L Immunogene Therapy for Malignant Melanoma and Other Solid Tumors Phase 1/Phase 2
Completed NCT00978913 - Transfected Dendritic Cell Based Therapy for Patients With Breast Cancer or Malignant Melanoma Phase 1
Completed NCT00232726 - Clinical Study of Previously Untreated Patients With Malignant Melanoma Phase 2
Completed NCT00350597 - GM-CSF as Adjuvant Therapy of Melanoma Phase 2
Completed NCT00336986 - Efficacy Study of IL-21 to Treat Metastatic Melanoma Phase 2
Completed NCT02523313 - Immunotherapy With Nivolumab or Nivolumab Plus Ipilimumab vs. Double Placebo for Stage IV Melanoma w. NED Phase 2
Completed NCT03545334 - Lymph Node Identification in Skin Malignancy Using ICG Transcutaneously Study N/A
Completed NCT04253574 - Comparison of PET/CT and Ultrasound in Staging of Malignant Melanoma
Completed NCT00179608 - Study of the Combination of Lenalidomide and DTIC (Dacarbazine) in Patients With Metastatic Malignant Melanoma Previously Untreated With Systemic Chemotherapy Phase 1
Terminated NCT00104884 - FR901228 in Treating Patients With Unresectable Stage III or Stage IV Malignant Melanoma Phase 2