Trial Outcomes & Findings for Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma (NCT NCT00601003)
NCT ID: NCT00601003
Last Updated: 2024-08-06
Results Overview
Test the safety of nifurtimox in children with relapsed or refractory neuroblastoma or medulloblastoma in combination with cyclophosphamide/topotecan
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
112 participants
Primary outcome timeframe
2 years
Results posted on
2024-08-06
Participant Flow
Participant milestones
| Measure |
Nifurtimox
Nifurtimox: 30mg/kg/day PO divided into TID dosing q day
Cyclophosphamide: 250 mg/m2/dose in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
Topotecan: 0.75mg/m2/dose, in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
|
|---|---|
|
Overall Study
STARTED
|
112
|
|
Overall Study
COMPLETED
|
76
|
|
Overall Study
NOT COMPLETED
|
36
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma
Baseline characteristics by cohort
| Measure |
Nifurtimox
n=112 Participants
Nifurtimox: 30mg/kg/day PO divided into TID dosing q day
Cyclophosphamide: 250 mg/m2/dose in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
Topotecan: 0.75mg/m2/dose, in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
|
|---|---|
|
Age, Continuous
|
5.5 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
39 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
73 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
16 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
93 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
11 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
89 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2 yearsTest the safety of nifurtimox in children with relapsed or refractory neuroblastoma or medulloblastoma in combination with cyclophosphamide/topotecan
Outcome measures
| Measure |
Nifurtimox
n=110 Participants
Nifurtimox: 30mg/kg/day PO divided into TID dosing q day
Cyclophosphamide: 250 mg/m2/dose in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
Topotecan: 0.75mg/m2/dose, in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
|
|---|---|
|
Number of Participants With Related Adverse Events as a Measure of Safety and Tolerability
|
45 Participants
|
PRIMARY outcome
Timeframe: 2 yearsTest the efficacy of nifurtimox in children with relapsed or refractory neuroblastoma or medulloblastoma in combination with cyclophosphamide/topotecan Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions, Overall Best Response assessed by CT or MRI, MIBG, and Bone Marrow: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions, bone marrow with CR, and MIBG with CR/PR. Overall Response (OR) = CR + PR.
Outcome measures
| Measure |
Nifurtimox
n=76 Participants
Nifurtimox: 30mg/kg/day PO divided into TID dosing q day
Cyclophosphamide: 250 mg/m2/dose in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
Topotecan: 0.75mg/m2/dose, in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
|
|---|---|
|
Best Radiological Response in Participants Using the RECIST Criteria
Complete Response
|
7 Participants
|
|
Best Radiological Response in Participants Using the RECIST Criteria
Partial Response
|
11 Participants
|
|
Best Radiological Response in Participants Using the RECIST Criteria
Stable Disease
|
35 Participants
|
|
Best Radiological Response in Participants Using the RECIST Criteria
Progressive Disease
|
23 Participants
|
Adverse Events
Nifurtimox
Serious events: 25 serious events
Other events: 45 other events
Deaths: 19 deaths
Serious adverse events
| Measure |
Nifurtimox
n=110 participants at risk
Nifurtimox: 30mg/kg/day PO divided into TID dosing q day
Cyclophosphamide: 250 mg/m2/dose in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
Topotecan: 0.75mg/m2/dose, in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
|
|---|---|
|
Gastrointestinal disorders
Anorexia
|
0.91%
1/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Other (Progressive Disease)
|
15.5%
17/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Infections and infestations
Sepsis
|
1.8%
2/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Gastrointestinal disorders
Nausea and vomiting
|
0.91%
1/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Renal and urinary disorders
Bladder Thrombosis
|
0.91%
1/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
1.8%
2/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
General disorders
Tooth Pain
|
0.91%
1/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Nervous system disorders
Seizure
|
2.7%
3/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Distress
|
0.91%
1/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration pneumonia
|
0.91%
1/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Vascular disorders
Hypotension
|
0.91%
1/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Metabolism and nutrition disorders
Dehydration
|
0.91%
1/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Surgical and medical procedures
Appendicitis
|
0.91%
1/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Vascular disorders
Brain hemorrhage
|
0.91%
1/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Nervous system disorders
Aphasia
|
0.91%
1/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Nervous system disorders
Gait Disturbance and Confusion
|
0.91%
1/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Nervous system disorders
confusion/psychosis and dysphagia
|
0.91%
1/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
Other adverse events
| Measure |
Nifurtimox
n=110 participants at risk
Nifurtimox: 30mg/kg/day PO divided into TID dosing q day
Cyclophosphamide: 250 mg/m2/dose in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
Topotecan: 0.75mg/m2/dose, in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
8.2%
9/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Blood and lymphatic system disorders
Leukopenia
|
6.4%
7/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Blood and lymphatic system disorders
Neutropenia
|
6.4%
7/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
6.4%
7/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Nervous system disorders
Ataxia
|
9.1%
10/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Nervous system disorders
Confusion
|
5.5%
6/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Nervous system disorders
Memory Impairment
|
2.7%
3/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Nervous system disorders
Motor Neuropathy/Weakness
|
6.4%
7/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Nervous system disorders
Peripheral Sensory Neuropathy
|
3.6%
4/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Nervous system disorders
Seizures
|
4.5%
5/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Investigations
ALT elevation
|
2.7%
3/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Gastrointestinal disorders
Anorexia
|
6.4%
7/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Blood and lymphatic system disorders
Febrile Neutropenia
|
4.5%
5/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Gastrointestinal disorders
Nausea
|
5.5%
6/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
General disorders
Pain
|
3.6%
4/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
|
Gastrointestinal disorders
Vomiting
|
5.5%
6/110 • From first dose of Nifurtimox through to 30 days after last dose, an average of 8 months. Additionally all events deemed related to Nifurtimox were followed past the 30 days to resolution or baseline.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place