Trial Outcomes & Findings for Donor Stem Cell Transplant After Chemotherapy for the Treatment of Recurrent or Refractory High-Risk Solid Tumors in Pediatric and Adolescent-Young Adults (NCT NCT04530487)
NCT ID: NCT04530487
Last Updated: 2025-06-29
Results Overview
To assess tolerability of allogeneic HCT for patients with chemo-responsive recurrent/refractory solid tumors as defined by transplant-related mortality (TRM) at day 30
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
1 participants
Primary outcome timeframe
By day +30 after allogeneic HCT infusion
Results posted on
2025-06-29
Participant Flow
This study was open to accrual from 8/19/2020 to 7/23/2024. Participants were referred for the study both internally and externally.
Participant milestones
| Measure |
Allogeneic Stem Cell Transplant in High Risk Solid Tumors
Matched Allogeneic Umbilical Cord Donor - thiotepa, etoposide, mephalan, rabbit ATG conditioning; tacrolimus/cyclosporine and MMF - GVHD prophylaxis
|
|---|---|
|
Overall Study
STARTED
|
1
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Allogeneic Stem Cell Transplant in High Risk Solid Tumors
Matched Allogeneic Umbilical Cord Donor - thiotepa, etoposide, mephalan, rabbit ATG conditioning; tacrolimus/cyclosporine and MMF - GVHD prophylaxis
|
|---|---|
|
Overall Study
Death
|
1
|
Baseline Characteristics
Donor Stem Cell Transplant After Chemotherapy for the Treatment of Recurrent or Refractory High-Risk Solid Tumors in Pediatric and Adolescent-Young Adults
Baseline characteristics by cohort
| Measure |
Allogeneic Stem Cell Transplant in High Risk Solid Tumors
n=1 Participants
Matched Allogeneic Umbilical Cord Donor - thiotepa, etoposide, mephalan, rabbit ATG conditioning; tacrolimus/cyclosporine and MMF - GVHD prophylaxis
|
|---|---|
|
Age, Categorical
<=18 years
|
1 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
12 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: By day +30 after allogeneic HCT infusionPopulation: No analysis was performed as insufficient patients were enrolled for statistically significant conclusions.
To assess tolerability of allogeneic HCT for patients with chemo-responsive recurrent/refractory solid tumors as defined by transplant-related mortality (TRM) at day 30
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: By day +30 after allogeneic HCT infusionPopulation: No analysis was performed as insufficient patients were enrolled for statistically significant conclusions.
The rate of grade III or higher organ toxicity (Bearman Regimen-Related Toxicities Scale)\[1\] attributable to conditioning occurring within 30 days.
Outcome measures
Outcome data not reported
Adverse Events
Allogeneic Stem Cell Transplant in High Risk Solid Tumors
Serious events: 0 serious events
Other events: 1 other events
Deaths: 1 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Allogeneic Stem Cell Transplant in High Risk Solid Tumors
n=1 participants at risk
Matched Allogeneic Umbilical Cord Donor - thiotepa, etoposide, mephalan, rabbit ATG conditioning; tacrolimus/cyclosporine and MMF - GVHD prophylaxis
|
|---|---|
|
Investigations
Alanine aminotransferase increased
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Metabolism and nutrition disorders
Anorexia
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Investigations
Aspartate aminotransferase increased
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Investigations
Blood lactate dehydrogenase increased
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Investigations
Creatinine Increase
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Gastrointestinal disorders
Diarrhea
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
General disorders
Fatigue
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
General disorders
Fever
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
General disorders
Generalized Edema
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Vascular disorders
Hypertension
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Infections and infestations
Infections and infestions-HHV6
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Investigations
Lymphocyte count decreased
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Gastrointestinal disorders
Mucositis oral
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Gastrointestinal disorders
Nausea
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Investigations
Neutrophil count decreased
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Cardiac disorders
Pericardial effusion
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Investigations
Platelet count decrease
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Infections and infestations
Lung infection - Pneumonia
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders-tachypnea
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Cardiac disorders
Sinus Tachycardia- intermittent
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Gastrointestinal disorders
Vomiting
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
|
Investigations
Weight loss
|
100.0%
1/1 • Number of events 1 • From the start of preparative regimen up to D+100 the collection of adverse events will reflect the onset and resolution date and maximum grade
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place