Trial Outcomes & Findings for Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML) (NCT NCT00167219)

NCT ID: NCT00167219

Last Updated: 2023-11-07

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 15 participants
• Primary outcome timeframe: at 1 year after transplant
• Results posted on: 2023-11-07

Participant Flow

Participant milestones

Measure	Intent-to-Treat Patients receiving study regimen. Stem Cell Transplant: Transplantation on Day 0. Preparative Regimen: - Busulfan * Cyclophosphamide * Mesna * Melphalan * Anti-thymocyte Globulin (ATG)
Overall Study STARTED	15
Overall Study COMPLETED	15
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)

Baseline characteristics by cohort

Measure	Intent-to-Treat n=15 Participants Patients receiving study regimen. Stem Cell Transplant: Transplantation on Day 0. Preparative Regimen: - Busulfan * Cyclophosphamide * Mesna * Melphalan * Anti-thymocyte Globulin (ATG)
Age, Categorical <=18 years	15 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
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	9 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 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	1 Participants n=5 Participants
Race (NIH/OMB) White	9 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	4 Participants n=5 Participants
Region of Enrollment United States	15 participants n=5 Participants

PRIMARY outcome

Timeframe: at 1 year after transplant

Outcome measures

Measure	Intent-to-Treat n=15 Participants Patients receiving study regimen. Stem Cell Transplant: Transplantation on Day 0. Preparative Regimen: - Busulfan * Cyclophosphamide * Mesna * Melphalan * Anti-thymocyte Globulin (ATG)
Determine Probability of Long-term Disease Free Survival in JMML	67 Percentage of participants Interval 38.0 to 85.0

SECONDARY outcome

Timeframe: at 1 year after transplant

Measured by AGVHD Grades 2 - 4.

Outcome measures

Measure	Intent-to-Treat n=15 Participants Patients receiving study regimen. Stem Cell Transplant: Transplantation on Day 0. Preparative Regimen: - Busulfan * Cyclophosphamide * Mesna * Melphalan * Anti-thymocyte Globulin (ATG)
Combined Incidence of Neutrophil Engraftment, Graft-versus-host Disease (GVHD), Regimen-related Toxicity, and Relapse.	33 Percentage of participants Interval 10.0 to 57.0

Adverse Events

Intent-to-Treat

Serious events: 5 serious events

Other events: 15 other events

Deaths: 4 deaths

Serious adverse events

Measure	Intent-to-Treat n=15 participants at risk Patients receiving study regimen. Stem Cell Transplant: Transplantation on Day 0. Preparative Regimen: - Busulfan * Cyclophosphamide * Mesna * Melphalan * Anti-thymocyte Globulin (ATG)
Infections and infestations Aspergillus Fumigatus	6.7% 1/15 • Number of events 1 • 1 year
Renal and urinary disorders Acute Renal Failure	6.7% 1/15 • Number of events 1 • 1 year
Infections and infestations Sepsis	6.7% 1/15 • Number of events 1 • 1 year
General disorders Hepatomegaly	13.3% 2/15 • Number of events 5 • 1 year
General disorders Seizure	6.7% 1/15 • Number of events 1 • 1 year

Other adverse events

Measure	Intent-to-Treat n=15 participants at risk Patients receiving study regimen. Stem Cell Transplant: Transplantation on Day 0. Preparative Regimen: - Busulfan * Cyclophosphamide * Mesna * Melphalan * Anti-thymocyte Globulin (ATG)
Vascular disorders Hypertension	26.7% 4/15 • Number of events 6 • 1 year
Cardiac disorders Pericardial Effusion	26.7% 4/15 • Number of events 4 • 1 year
Gastrointestinal disorders Blood in Stool	26.7% 4/15 • Number of events 5 • 1 year
Gastrointestinal disorders Blood in Urine	6.7% 1/15 • Number of events 1 • 1 year
Gastrointestinal disorders Rectal Bleed	6.7% 1/15 • Number of events 1 • 1 year
Gastrointestinal disorders Bowel Perferation	6.7% 1/15 • Number of events 1 • 1 year
Gastrointestinal disorders Colitis	13.3% 2/15 • Number of events 2 • 1 year
Gastrointestinal disorders Ischemia	6.7% 1/15 • Number of events 1 • 1 year
Gastrointestinal disorders Ascites	13.3% 2/15 • Number of events 2 • 1 year
Gastrointestinal disorders Vomiting	6.7% 1/15 • Number of events 1 • 1 year
Investigations Platelet count decreased	6.7% 1/15 • Number of events 1 • 1 year
Investigations Blood bilirubin increased	20.0% 3/15 • Number of events 3 • 1 year
Infections and infestations Hickman Line Infection	6.7% 1/15 • Number of events 1 • 1 year
Ear and labyrinth disorders Ear Infection	26.7% 4/15 • Number of events 5 • 1 year
Infections and infestations Upper respiratory infection	33.3% 5/15 • Number of events 6 • 1 year
Infections and infestations Lung Infection	26.7% 4/15 • Number of events 5 • 1 year
Reproductive system and breast disorders Pneumonia	33.3% 5/15 • Number of events 7 • 1 year
Skin and subcutaneous tissue disorders Dermititis	6.7% 1/15 • Number of events 1 • 1 year

Additional Information

Dr. Margaret MacMillan, MD

University of Minnesota, Masonic Cancer Center

Phone: +1 612-626-2961

Email: macmi002@umn.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place