Unspecified Childhood Solid Tumor, Protocol SpecificPhase 1NCT00436761
Busulfan, Melphalan, and Antithymocyte Globulin Followed By Umbilical Cord Blood Transplant in Treating Young Patients With Refractory or Relapsed Malignant Solid Tumors
A Phase 1 study testing anti-thymocyte globulin, graft-versus-tumor induction therapy and sargramostim in people with Unspecified Childhood Solid Tumor, Protocol Specific, run by Milton S. Hershey Medical Center at 1 site in United States.
Trial summary
RATIONALE: Giving chemotherapy before a donor umbilical cord blood stem cell transplant helps stop the growth of tumor cells. It also helps stop the patient's immune system from rejecting the donor's stem cells when they do not exactly match the patient's blood. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Unspecified Childhood Solid Tumor, Protocol Specific
- What is being tested?
- anti-thymocyte globulin, graft-versus-tumor induction therapy and sargramostim
- Type of treatment
- Surgery or procedure
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- May 2004to not given
- 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 21 years
- All sexes
- No healthy volunteers
- Child, Adult
DISEASE CHARACTERISTICS:
- Diagnosis of malignant solid tumor
- Relapsed or refractory disease
- No isolated local recurrence of disease (in the site of the primary tumor) > 1 year after completing therapy
- No brain tumors or brain metastases
- Unrelated cord blood donor available
- May be HLA 6/6 matched (HLA-A, -B, -DR) OR mismatched for 1, 2, or 3 of these HLA loci, but must be mismatched for HLA-C group as indicated by their following killer cell immunoglobulin-like receptor (KIR) group specificity:
- KIR2DL1
- Cw 2
- Cw 0307
- Cw 4, 5, 6
- Cw 0707, 0709
- Cw 1204, 1205
- All other Cw 15 alleles
- Cw 1602
- Cw 17
- Cw 18
- KIR2DL2
- Cw 1
- All other Cw 3 alleles
- All other Cw 7 alleles
- Cw 8
- Cw 1202, 1203, 1206
- Cw 1301
- Cw 1402, 1403
- Cw 1507
- Cw 1601, 1604
- Cord blood specimen must have ≥ 1 x 10^7 nucleated cells/kg patient ideal body weight
PATIENT CHARACTERISTICS:
- ECOG performance status (PS) 0-2 OR Lansky PS 70-100%
- Cardiac ejection fraction ≥ 50%
- Creatinine clearance ≥ 50%
- Bilirubin ≤ 3.0 mg/dL
- DLCO ≥ 70% OR O_2 saturation ≥ 95% on room air
PRIOR CONCURRENT THERAPY:
- Prior autologous stem cell transplantation allowed
Trial locations and contacts
1 site in 1 country.
United States1 site
Penn State 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 Killer IG-Like Receptor (KIR) Mismatched Umbilical Cord Blood for Pediatric Patients With Malignant Solid Tumors
RATIONALE: Giving chemotherapy before a donor umbilical cord blood stem cell transplant helps stop the growth of tumor cells. It also helps stop the patient's immune system from rejecting the donor's stem cells when they do not exactly match the patient's blood. The donated stem cells may replace the patient's immune cells and help destroy any remaining tumor cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and methylprednisolone after the transplant may stop this from happening.
PURPOSE: This phase I trial is studying the side effects of busulfan, melphalan, and antithymocyte globulin followed by umbilical cord blood transplant in treating young patients with refractory or relapsed malignant solid tumors.
Read the detailed description
OBJECTIVES:
- Examine the impact of the use of killer cell immunoglobulin-like receptor (KIR)-mismatched umbilical cord blood as a source of hematopoietic stem cells, after busulfan, melphalan, and anti-thymocyte globulin in pediatric patients with relapsed or refractory solid tumors.
- Determine the toxicity of this regimen, in terms of incidence of grade 3-4 acute graft-versus-host disease, donor/host chimerism, and cellular immunity against tumor cell lines, in these patients.
OUTLINE:
- Transplantation: Patients receive busulfan orally or IV every 6 hours on days -8 to -5, anti-thymocyte globulin IV over 6 hours on days -4 to -1, and melphalan IV over 15-20 minutes on days -4 to -2. Patients undergo allogeneic umbilical cord blood stem cell infusion on day 0. Patients receive sargramostim (GM-CSF) subcutaneously beginning on day 7 and continuing until blood counts recover.
- Graft-vs-host disease prophylaxis: Patients receive cyclosporine IV over 1 hour or orally twice daily on days -1 to 180 and methylprednisolone IV or orally once or twice daily on days 5 - 49.
Blood samples are collected periodically for immunophenotyping and flow cytometric analysis (including interferon gamma and other TH1 and TH2 cytokines).
After completion of study treatment, patients are followed periodically.
PROJECTED ACCRUAL: A total of 20 patients will be accrued for this study.
- Being tested
- anti-thymocyte globulin, graft-versus-tumor induction therapy, sargramostim, busulfan, cyclosporine
- Keywords
- unspecified childhood solid tumor, protocol specific
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-thymocyte globulinBiological
graft-versus-tumor induction therapyBiological
sargramostimBiological
busulfanDrug
cyclosporineDrug
melphalanDrug
flow cytometryOther
immunologic techniqueOther
laboratory biomarker analysisOther
allogeneic hematopoietic stem cell transplantationProcedure or surgery
umbilical cord blood transplantationProcedure or surgery
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety
Incidence of graft-versus-host disease
Secondary outcomes (2)
Donor/host chimerism status
Immune function post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Feb 2007 (estimated)
- Study startMay 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Unspecified Childhood Solid Tumor, Protocol SpecificStudies 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 1
- Primary purpose
- Treatment
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Milton S. Hershey Medical Center
- Sponsor type
- University, hospital or other
- Investigators
- Kenneth G. Lucas, MD, Study chair, Milton S. Hershey Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00436761
- Sponsor's protocol ID
- CDR0000529361
- Other IDs
- PSCI-18589
- Status last verified
- Jun 2007
- First submitted
- 15 Feb 2007
- Last updated
- 18 Dec 2013
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Dec 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 Killer IG-Like Receptor (KIR) Mismatched Umbilical Cord Blood for Pediatric Patients With Malignant Solid Tumors
- ClinicalTrials.gov ID
- NCT00436761
- Acronym / sponsor ID
- CDR0000529361
- Keywords
- unspecified childhood solid tumor, protocol specific
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Milton S. Hershey Medical CenterUniversity, hospital or other
- Study start
- May 2004
- First posted
- 19 Feb 2007 (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: Giving chemotherapy before a donor umbilical cord blood stem cell transplant helps stop the growth of tumor cells. It also helps stop the patient's immune system from rejecting the donor's stem cells when they do not exactly match the patient's blood. The donated stem cells may replace the patient's immune cells and help destroy any remaining tumor cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and methylprednisolone after the transplant may stop this from happening.
PURPOSE: This phase I trial is studying the side effects of busulfan, melphalan, and antithymocyte globulin followed by umbilical cord blood transplant in treating young patients with refractory or relapsed malignant solid tumors.
Detailed description
OBJECTIVES:
- Examine the impact of the use of killer cell immunoglobulin-like receptor (KIR)-mismatched umbilical cord blood as a source of hematopoietic stem cells, after busulfan, melphalan, and anti-thymocyte globulin in pediatric patients with relapsed or refractory solid tumors.
- Determine the toxicity of this regimen, in terms of incidence of grade 3-4 acute graft-versus-host disease, donor/host chimerism, and cellular immunity against tumor cell lines, in these patients.
OUTLINE:
- Transplantation: Patients receive busulfan orally or IV every 6 hours on days -8 to -5, anti-thymocyte globulin IV over 6 hours on days -4 to -1, and melphalan IV over 15-20 minutes on days -4 to -2. Patients undergo allogeneic umbilical cord blood stem cell infusion on day 0. Patients receive sargramostim (GM-CSF) subcutaneously beginning on day 7 and continuing until blood counts recover.
- Graft-vs-host disease prophylaxis: Patients receive cyclosporine IV over 1 hour or orally twice daily on days -1 to 180 and methylprednisolone IV or orally once or twice daily on days 5 - 49.
Blood samples are collected periodically for immunophenotyping and flow cytometric analysis (including interferon gamma and other TH1 and TH2 cytokines).
After completion of study treatment, patients are followed periodically.
PROJECTED ACCRUAL: A total of 20 patients will be accrued for this study.
Study design
- Primary purpose
- Treatment
- Masking
- None (open label)
Who may qualify
- Ages
- Up to 21 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 | – |
| graft-versus-tumor induction therapy | Biological | – |
| sargramostim | Biological | – |
| busulfan | Drug | – |
| cyclosporine | Drug | – |
| melphalan | Drug | – |
| methylprednisolone | Drug | – |
| flow cytometry | Other | – |
| immunologic technique | Other | – |
| laboratory biomarker analysis | Other | – |
| allogeneic hematopoietic stem cell transplantation | Procedure or surgery | – |
| umbilical cord blood transplantation | Procedure or surgery | – |
Primary outcome
Safety
Incidence of graft-versus-host disease
2 secondary outcomes
Donor/host chimerism status
Immune function post-transplant
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Dec 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 study chair
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, graft-versus-tumor induction therapy and sargramostim in people with Unspecified Childhood Solid Tumor, Protocol Specific, 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: status not confirmed, as of the sponsor's last update on 18 Dec 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?
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.
What does the primary outcome measure?
Safety. 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 18 Dec 2013.