NeuroblastomaPhase 1NCT00112645
Donor Stem Cell Transplant in Treating Young Patients With Relapsed or Refractory Solid Tumors
A Phase 1 study testing anti-thymocyte globulin, busulfan and cyclosporine in people with Neuroblastoma and Sarcoma, run by Milton S. Hershey Medical Center at 1 site in United States.
Trial summary
RATIONALE: A peripheral stem cell, bone marrow, or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy and radiation therapy. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Read more about this study
- Who is it for?
- Ages Up to 30 years
- Conditions the study is for
- Neuroblastoma, Sarcoma, Unspecified Childhood Solid Tumor, Protocol Specific
- What is being tested?
- anti-thymocyte globulin, busulfan and cyclosporine
- Type of treatment
- Surgery or procedure
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2005to Feb 2011
- Phase
- Phase 1
- 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 30 years
- All sexes
- No healthy volunteers
- Child, Adult
DISEASE CHARACTERISTICS:
- Diagnosis of relapsed or refractory solid tumor, including, but not limited to, relapsed neuroblastoma, relapsed Ewing's sarcoma, and relapsed rhabdomyosarcoma
- No isolated local disease recurrence at the site of the primary tumor > 1 year after completion of prior therapy
- No brain tumor or brain metastases
- Related or unrelated hematopoietic stem cell donor available, meeting 1 of the following criteria:
- Matched for HLA-A, -B, -C, -DR, and -DQ (9/10 or 10/10 allelles) (marrow or peripheral blood)
- Matched for HLA-A, -B, and -DR (5/6 or 6/6 allelles) (cord blood)
- Cord blood specimen must contain ≥ 2 x 10 ^7 nucleated cells/kg body weight
PATIENT CHARACTERISTICS:
Age
- 30 and under
Performance status
- ECOG 0-1 OR
- Lansky 80-100%
Life expectancy
- Not specified
Hematopoietic
- Not specified
Hepatic
- Bilirubin ≤ 3.0 mg/dL
Renal
- Creatinine clearance ≥ 50 mL/min
Cardiovascular
- Ejection fraction ≥ 50%
Pulmonary
- DLCO ≥ 70% OR
- O_2 saturation ≥ 95% on room air
Other
- Not pregnant
- Negative pregnancy test
- Fertile patients must use effective contraception
- HIV negative
PRIOR CONCURRENT THERAPY:
Biologic therapy
- Prior autologous stem cell transplantation allowed
Chemotherapy
- Not specified
Endocrine therapy
- Not specified
Radiotherapy
- Not specified
Surgery
- Not specified
Trial locations and contacts
1 site in 1 country.
United States1 site
Penn State Hershey Cancer Institute at Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033-0850
About this study
Official title: A Phase I Study to Examine the Toxicity of Allogeneic Stem Cell Transplantation for Pediatric Solid Tumors With Relapsed or Therapy Refractory Disease
RATIONALE: A peripheral stem cell, bone marrow, or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy and radiation therapy. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving busulfan and melphalan with or without antithymocyte globulin before transplant and cyclosporine with methylprednisolone or methotrexate after transplant may stop this from happening.
PURPOSE: This phase I trial is studying the side effects of donor stem cell transplant in treating young patients with relapsed or refractory solid tumors.
Read the detailed description
OBJECTIVES:
- Determine the toxicity of allogeneic hematopoietic stem cell transplantation, in terms of the incidence of grade 3-4 acute graft-versus-host disease, in young patients with relapsed or refractory solid tumors.
- Determine the incidence of transplant-related mortality at 100 days post-transplantation in these patients.
OUTLINE:
- Conditioning: Patients receive busulfan IV or orally 4 times daily on days -8 to -5 (a total of 16 doses) and melphalan IV over 15-20 minutes on days -4 to -2. Patients with an unrelated donor also receive anti-thymocyte globulin IV on days -4 to -2.
- Allogeneic hematopoietic stem cell transplantation (SCT): Patients undergo allogeneic hematopoietic SCT on day 0.
- Post-transplant graft-versus-host disease (GVHD) prophylaxis: Patients who undergo cord blood SCT receive cyclosporine and methylprednisolone for graft-versus-host disease (GVHD) prophylaxis. Patients who undergo peripheral blood or bone marrow SCT receive cyclosporine and methotrexate (short course) for GVHD prophylaxis.
After completion of study treatment, patients are followed at 1, 3, 6, and 12 months and then annually thereafter.
PROJECTED ACCRUAL: A total of 10 patients will be accrued for this study within 4 years.
- Being tested
- anti-thymocyte globulin, busulfan, cyclosporine, melphalan, methotrexate
- Keywords
- unspecified childhood solid tumor, protocol specific, recurrent neuroblastoma, recurrent Ewing sarcoma/peripheral primitive neuroectodermal tumor, recurrent childhood rhabdomyosarcoma
- Medical subject headings
- Neuroblastoma, Sarcoma, Neuroectodermal Tumors, Primitive, Peripheral
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-thymocyte globulinBiological
busulfanDrug
cyclosporineDrug
melphalanDrug
methotrexateDrug
allogeneic bone marrow transplantationProcedure or surgery
peripheral blood stem cell transplantationProcedure or surgery
umbilical cord blood 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 posted3 Jun 2005 (estimated)
- Study startApr 2005
- Primary completionApr 2010
- Study completionFeb 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies of the same condition, open and under way.
- More trials run by Milton S. Hershey Medical CenterThe sponsor's other registered studies.
- 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 1
- Primary purpose
- Treatment
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Milton S. Hershey Medical Center
- Sponsor type
- University, hospital or other
- Investigators
- Kenneth G. Lucas, MD, Principal investigator, Milton S. Hershey Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00112645
- Sponsor's protocol ID
- PSCI-20615
- Other IDs
- CDR0000430441
- Status last verified
- Feb 2011
- First submitted
- 2 Jun 2005
- Last updated
- 18 Jul 2013
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jul 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study to Examine the Toxicity of Allogeneic Stem Cell Transplantation for Pediatric Solid Tumors With Relapsed or Therapy Refractory Disease
- ClinicalTrials.gov ID
- NCT00112645
- Acronym / sponsor ID
- PSCI-20615
- Keywords
- unspecified childhood solid tumor, protocol specific, recurrent neuroblastoma, recurrent Ewing sarcoma/peripheral primitive neuroectodermal tumor, recurrent childhood rhabdomyosarcoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Milton S. Hershey Medical CenterUniversity, hospital or other
- Study start
- Apr 2005
- Primary completion
- Apr 2010 (actual)
- Study completion
- Feb 2011 (actual)
- First posted
- 3 Jun 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.
RATIONALE: A peripheral stem cell, bone marrow, or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy and radiation therapy. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving busulfan and melphalan with or without antithymocyte globulin before transplant and cyclosporine with methylprednisolone or methotrexate after transplant may stop this from happening.
PURPOSE: This phase I trial is studying the side effects of donor stem cell transplant in treating young patients with relapsed or refractory solid tumors.
Detailed description
OBJECTIVES:
- Determine the toxicity of allogeneic hematopoietic stem cell transplantation, in terms of the incidence of grade 3-4 acute graft-versus-host disease, in young patients with relapsed or refractory solid tumors.
- Determine the incidence of transplant-related mortality at 100 days post-transplantation in these patients.
OUTLINE:
- Conditioning: Patients receive busulfan IV or orally 4 times daily on days -8 to -5 (a total of 16 doses) and melphalan IV over 15-20 minutes on days -4 to -2. Patients with an unrelated donor also receive anti-thymocyte globulin IV on days -4 to -2.
- Allogeneic hematopoietic stem cell transplantation (SCT): Patients undergo allogeneic hematopoietic SCT on day 0.
- Post-transplant graft-versus-host disease (GVHD) prophylaxis: Patients who undergo cord blood SCT receive cyclosporine and methylprednisolone for graft-versus-host disease (GVHD) prophylaxis. Patients who undergo peripheral blood or bone marrow SCT receive cyclosporine and methotrexate (short course) for GVHD prophylaxis.
After completion of study treatment, patients are followed at 1, 3, 6, and 12 months and then annually thereafter.
PROJECTED ACCRUAL: A total of 10 patients will be accrued for this study within 4 years.
Study design
- Primary purpose
- Treatment
- Masking
- None (open label)
Who may qualify
- Ages
- Up to 30 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 | Biological | – |
| busulfan | Drug | – |
| cyclosporine | Drug | – |
| melphalan | Drug | – |
| methotrexate | Drug | – |
| methylprednisolone | Drug | – |
| allogeneic bone marrow transplantation | Procedure or surgery | – |
| peripheral blood stem cell transplantation | Procedure or surgery | – |
| umbilical cord blood 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 18 Jul 2013.
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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.
Kenneth G. Lucas, MD principal investigator
Milton S. Hershey Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing anti-thymocyte globulin, busulfan and cyclosporine in people with Neuroblastoma and Sarcoma, run by Milton S. Hershey 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 18 Jul 2013. 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 18 Jul 2013.