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Stem Cell Transplant for High-Risk Solid Cancers in Youth

Phase 2
Recruiting
Led By Dristhi Ragoonanan, MD
Research Sponsored by M.D. Anderson Cancer Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
DONOR: Matched related donor bone marrow (10 of 10 HLA alleles [HLA-A, B, C, DR, and DQ]. Matched related donor peripheral blood stem cell (PBSC) is allowed only if collection of bone marrow (BM) is not available or refused by parents/donor
Patients must have chemo-responsive disease, defined as 30% or greater decrease in the tumor target lesions when compared to its pre-treatment evaluation. Patients with complete response will be eligible to participate
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at 1 year post transplant
Awards & highlights

Study Summary

This trial is testing side effects and how well stem cell transplant after chemo works for kids and young adults with high-risk solid tumors.

Who is the study for?
This trial is for pediatric and young adult patients with high-risk solid tumors that have returned or are not responding to treatment. Eligible participants must show a positive response to chemotherapy, have proper liver and kidney function, suitable donors for stem cell transplant, and normal lung function or adequate oxygen levels without assistance.Check my eligibility
What is being tested?
The trial tests the effectiveness of donor stem cell transplants following chemotherapy in treating recurrent or refractory solid tumors. Chemotherapy includes drugs like fludarabine, thiotepa, etoposide, melphalan, and rabbit anti-thymocyte globulin which aim to kill or stop cancer cells from growing before introducing healthy donor stem cells.See study design
What are the potential side effects?
Potential side effects may include reactions to the infusion of new stem cells; damage to organs due to powerful chemotherapy drugs; increased risk of infections; blood-forming complications; potential graft-versus-host disease where donated cells attack the patient's body.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My donor is a family member who matches me perfectly in 10 out of 10 HLA markers.
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My cancer responded well to chemotherapy, shrinking by 30% or more.
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I have a specific type of cancer that is either new or has come back.
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My donor's umbilical cord blood matches mine at least 4/6 for transplant.
Select...
My donor's umbilical cord blood matches mine at least 4/6 for transplant.
Select...
My kidneys work well enough, and I don't need dialysis.
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My liver tests are within the required limits, except for high bilirubin due to Gilbert's syndrome.
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My lung function test shows I can breathe well enough, or my oxygen level is at least 92% without extra help.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at 1 year post transplant
This trial's timeline: 3 weeks for screening, Varies for treatment, and at 1 year post transplant for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Rate of grade III or higher organ toxicity attributable to conditioning
Transplant-related mortality (TRM)
Secondary outcome measures
Incidence of acute graft versus host disease (aGVHD)
Incidence of chronic GVHD
Incidence of relapse
+8 more

Side effects data

From 2019 Phase 2 trial • 77 Patients • NCT01251575
5%
Hypoxia
5%
Febrile neutropenia
5%
Acute kidney injury
4%
Blood bilirubin increased
4%
Diarrhea
4%
Creatinine increased
4%
Sepsis
3%
Hypotension
3%
Left ventricular systolic dysfunction
3%
Bronchopulmonary hemorrhage
3%
Chronic kidney disease
3%
Thromboembolic event
3%
Lung infection
1%
Atrial fibrillation
1%
Atrial flutter
1%
Hemolysis
1%
Hemolytic uremic syndrome
1%
Ejection fraction decreased
1%
Encephalitis infection
1%
Gastric hemorrhage
1%
Gastritis
1%
Heart failure
1%
Mucositis oral
1%
Multi-organ failure
1%
Myalgia
1%
Pleural effusion
1%
Respiratory failure
1%
Small intestine infection
1%
Syncope
1%
Treatment related secondary malignancy
1%
Typhlitis
1%
Fever
1%
Paroxysmal atrial tachycardia
1%
Ascites
100%
80%
60%
40%
20%
0%
Study treatment Arm
Treatment (Fludarabine, Transplant, Immunosuppression)

Trial Design

1Treatment groups
Experimental Treatment
Group I: Treatment (conditioning regimen, HSCT)Experimental Treatment9 Interventions
CONDITIONING REGIMEN: Patients receive thiotepa IV over 2-4 hours, etoposide IV over 60 minutes on days -8 to -6, melphalan IV over 20 minutes on days -5 and -4, and fludarabine phosphate IV over 1 hour on days -5 to -3 in the absence of disease progression or unacceptable toxicity. Patients receiving umbilical cord transplant also receive rabbit anti-thymocyte globulin IV on days -3 and -4. TRANSPLANT: Patients undergo HSCT on day 0. GVHD PROPHYLAXIS: Beginning day -2, patients receive tacrolimus or cyclosporine IV continuously until able to receive PO. Patients continue tacrolimus or cyclosporine PO to day 60 and tapered to day 100. Patients also receive mycophenolate mofetil PO or IV every 8 hours until day 40 and tapered to day 90.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Tacrolimus
2011
Completed Phase 4
~4740
Thiotepa
2008
Completed Phase 3
~2150
Melphalan
2008
Completed Phase 3
~1500
Mycophenolate Mofetil
1997
Completed Phase 4
~2380
Fludarabine Phosphate
1997
Completed Phase 3
~2390
Allogeneic Hematopoietic Stem Cell Transplantation
2012
Completed Phase 2
~1200
Cyclosporine
1997
Completed Phase 3
~1830
Etoposide
2010
Completed Phase 3
~2440

Find a Location

Who is running the clinical trial?

M.D. Anderson Cancer CenterLead Sponsor
2,962 Previous Clinical Trials
1,803,325 Total Patients Enrolled
Dristhi Ragoonanan, MDPrincipal InvestigatorM.D. Anderson Cancer Center
Kris M MahadeoPrincipal InvestigatorM.D. Anderson Cancer Center
2 Previous Clinical Trials

Media Library

Allogeneic Hematopoietic Stem Cell Transplantation Clinical Trial Eligibility Overview. Trial Name: NCT04530487 — Phase 2
Solid Tumors Research Study Groups: Treatment (conditioning regimen, HSCT)
Solid Tumors Clinical Trial 2023: Allogeneic Hematopoietic Stem Cell Transplantation Highlights & Side Effects. Trial Name: NCT04530487 — Phase 2
Allogeneic Hematopoietic Stem Cell Transplantation 2023 Treatment Timeline for Medical Study. Trial Name: NCT04530487 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there currently opportunities to participate in this trial?

"Data found on clinicaltrials.gov affirms that this medical trial is recruiting participants at the moment. The study was listed initially on August 19th 2020 and its most recent edit occured September 22nd 2022."

Answered by AI

To what health issues does Allogeneic Hematopoietic Stem Cell Transplantation lend itself as a potential remedy?

"Allogeneic Hematopoietic Stem Cell Transplantation can be an effective therapy for patients who are non-responsive to other treatments, suffer from vernal keratoconjunctivitis, or have adenocarcinoma."

Answered by AI

What is the population size of this research endeavor?

"Affirmative, according to information on clinicaltrials.gov recruitment for this medical trial is currently underway. Initially published 8/19/2020 and most recently altered 9/22/2022, the study seeks 40 participants from a single site."

Answered by AI

Has Allogeneic Hematopoietic Stem Cell Transplantation been tested as part of any other clinical trials?

"Currently, 870 active trials are examiing the potential of Allogeneic Hematopoietic Stem Cell Transplantation with 150 being in Phase 3. Moreover, 20613 clinical sites across the globe are running studies related to this topic; Philadelphia, Pennsylvania is home to several such tests."

Answered by AI

Has Allogeneic Hematopoietic Stem Cell Transplantation been given authorization by the FDA?

"Allogeneic Hematopoietic Stem Cell Transplantation has been deemed to be of intermediate safety, scoring a 2 on our organisation's scale. This is due to its Phase 2 status, suggesting that while there are some data points supporting its safety profile, none exist in regards to efficacy."

Answered by AI

What are the principal aims of this research endeavor?

"Evaluated over a month-long period, the primary objective of this medical trial is to assess grade III or higher organ toxicity from conditioning. Additional goals include measuring one year rates of chronic GVHD with competing risk analysis as outlined by Gooley, determining time until platelet and neutrophil engraftment using Kaplan and Meier's method, and calculating 100 day rates of acute graft versus host disease (aGVHD) with its own competing relapse rate according to Gooley's technique."

Answered by AI
~9 spots leftby May 2025