The information contained in this Clinical Trials Resource Guide includes actively recruiting clinical studies focused on pediatric leukemia. These studies highlight quality-of-life reporting; neutropenia management; combination chemotherapy; chimeric antigen receptor (CAR) T-cell therapy and some of the PLAT trials; risk-directed therapy; and more. All of the studies are listed on the National Institutes of Health website at ClinicalTrials.gov.
OBSERVATIONAL
Study Title: Home or Away From Home: Comparing Patient- and Caregiver-Reported Quality of Life and Other Patient-Centered Outcomes for Inpatient vs Outpatient Management of Neutropenia in Children With Acute Myeloid Leukemia (AML)
Study Type: Observational
Study Sponsor and Collaborators: Children’s Hospital of Philadelphia; see ClinicalTrials.gov for complete list of -collaborators.
Purpose: To gather clinical data to inform whether management of neutropenia post-AML chemotherapy should occur in an outpatient or inpatient setting
Primary Outcome Measures: Compare differences in health-related quality-of-life scores between outpatient vs inpatient management [time frame: June 2016 to December 2018]
Ages Eligible for Study: 1–18 years
Principal Investigator: Richard Aplenc, MD, PhD, Children’s Hospital of Philadelphia; aplenc@chop.edu
ClinicalTrials.gov Identifier: NCT02777021
PHASE I
Study Title: A Phase I and Expansion Cohort Study of Venetoclax in Combination With Chemotherapy in Pediatric Patients With Refractory or Relapsed AML
Study Type: Phase I/interventional/single-group assignment
Study Sponsor and Collaborators: St. Jude Children’s Research Hospital, Gateway for Cancer Research
Purpose: To test the safety of venetoclax and to find the highest dose of venetoclax that can be given safely to patients with pediatric leukemia when it is combined with cytarabine or cytarabine and idarubicin
Primary Outcome Measures: Maximum tolerated combination [time frame: 28 days after start of therapy]
Ages Eligible for Study: 2–20 years
Principal Investigator: Richard Aplenc, MD, PhD, Children’s Hospital of Philadelphia; aplenc@chop.edu
ClinicalTrials.gov Identifier: NCT02777021
Study Title: Pediatric and Young Adult Leukemia Adoptive Therapy (PLAT)-05: A Phase I Feasibility and Safety Study of Dual-Specificity CD19 and CD22 CAR T-Cell Immunotherapy for CD19-Positive/CD22-Positive Leukemia and Lymphoma
Study Type: Phase I/interventional/single-group assignment
Study Sponsor and Collaborators: Seattle Children’s Hospital
Purpose: To use T cells obtained directly from the patient for treatment, which can be genetically modified to express two CARs. One is to recognize CD19 and the other is to recognize CD22, both of which are proteins expressed on the surface of the leukemic cell in patients with CD19-positive/CD22-positive leukemia.
Primary Outcome Measures: The adverse events associated with one or multiple CAR T-cell product infusions [time frame: 30 days]; the number of successfully and unsuccessfully manufactured and infused CAR T-cell products [time frame: 28 days]
Ages Eligible for Study: 1–26 years
Principal Investigator: Rebecca Gardner, MD, Seattle Children’s Hospital; 206-987-2106
ClinicalTrials.gov Identifier: NCT03330691
Phase I/II
Study Title: Pediatric and Young Adult Leukemia Adoptive Therapy (PLAT)-02: A Phase I/II Feasibility and Safety Study of CD19–CAR T-Cell Immunotherapy for CD19-Positive Leukemia
Study Type: Interventional/nonrandomized/single-group assignment
Study Sponsor and Collaborators: Seattle Children’s Hospital
Purpose: To determine the maximum tolerated dose and efficacy of the CAR-positive T cells. The phase I cohort is restricted to patients who have already had an allogeneic hematopoietic cell transplant (HCT). Phase II is open to all patients regardless of having a history of HCT.
Primary Outcome Measures: Number of participants with adverse events [time frame: 30 days]; number of participants with a minimal residual disease–negative complete remission after T-cell infusion [time frame: 63 days]; number of participants who have a releasable cell product generated [time frame: 28 days]
Ages Eligible for Study: 1–26 years
Principal Investigator: Rebecca Gardner, MD, Seattle Children’s Hospital; 206-987-2106
ClinicalTrials.gov Identifier: NCT02028455
Phase II
Study Title: A Phase II Study of Therapy for Pediatric Relapsed or Refractory Precursor B-Cell Acute Lymphoblastic Leukemia and Lymphoma
Study Type: Interventional/nonrandomized/parallel -assignment
Study Sponsor and Collaborators: St. Jude Children’s Research Hospital, Cookies for Kids’ Cancer, Assisi Foundation
Purpose: To study risk-directed therapy for B-cell lymphoblastic leukemia or lymphoma in first relapse. Standard-risk and high-risk participants will receive different therapy; treatment will consist of chemotherapy for standard-risk participants, and chemotherapy followed by hematopoietic stem cell transplant for high-risk participants in first relapse.
Primary Outcome Measures: 3-year overall survival rate of patients with relapsed acute lymphoblastic leukemia or lymphoma; 3-year event-free survival rates in patients with relapsed acute lymphoblastic leukemia or lymphoma [time frame: At 3 years of follow-up since the on-study date of the last enrolled patient]
Ages Eligible for Study: Up to 21 years
Principal Investigator: Sima Jeha, MD, St. Jude Children’s Research Hospital; (866) 278-5833, referralinfo@stjude.org
ClinicalTrials.gov Identifier: NCT01700946
Editor’s Note: The clinical trials presented here do not represent all the trials listed on ClinicalTrials.gov. For the complete list, go to ClinicalTrials.gov. ■
