Trial Outcomes & Findings for Use of Rituximab in Opsoclonus-Myoclonus in Children With Neuroblastoma (NCT NCT00202930)
NCT ID: NCT00202930
Last Updated: 2021-01-22
Results Overview
To evaluate the feasibility of using 4 weekly Rituximab infusions in the treatment of children with neuroblastoma associated opsoclonus-myoclonus syndrome (OMS). The first infusion involved a slow up titration from an initial rate of 0.5 mg/kg/hour to a maximum of 400 mg/hour. If well tolerated, the subsequent infusions were administered at a starting rate of 1 mg/kg/hour to a maximum of 100 mg/hour for the first hour. In the absence of adverse reaction doses were escalated by 1 mg/kg/hour (maximum 100 mg increase per hour) every 30 minutes to a maximum rate of 400 mg/hour. If hypersensitivity or infusion related events occurred, the infusion was temporarily interrupted and resumed at 50% of the previous rate if reaction resolves. The number of participants for whom the study dosing was feasible is reported.
TERMINATED
PHASE2
4 participants
4 weeks
2021-01-22
Participant Flow
Participant milestones
| Measure |
Rituximab
Participants received 4 weekly doses of IV rituxan at 375 mg/m\^2 on days 1, 8, 15 and 22
|
|---|---|
|
Overall Study
STARTED
|
4
|
|
Overall Study
COMPLETED
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Use of Rituximab in Opsoclonus-Myoclonus in Children With Neuroblastoma
Baseline characteristics by cohort
| Measure |
Rituximab
n=4 Participants
Participants received 4 weekly doses of IV rituxan at 375 mg/m\^2 on days 1, 8, 15 and 22
|
|---|---|
|
Age, Categorical
<=18 years
|
4 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
4 participants
n=99 Participants
|
|
OMS Evaluation Scale of Motor-Performance.
|
NA units on a scale
n=99 Participants
|
PRIMARY outcome
Timeframe: 4 weeksTo evaluate the feasibility of using 4 weekly Rituximab infusions in the treatment of children with neuroblastoma associated opsoclonus-myoclonus syndrome (OMS). The first infusion involved a slow up titration from an initial rate of 0.5 mg/kg/hour to a maximum of 400 mg/hour. If well tolerated, the subsequent infusions were administered at a starting rate of 1 mg/kg/hour to a maximum of 100 mg/hour for the first hour. In the absence of adverse reaction doses were escalated by 1 mg/kg/hour (maximum 100 mg increase per hour) every 30 minutes to a maximum rate of 400 mg/hour. If hypersensitivity or infusion related events occurred, the infusion was temporarily interrupted and resumed at 50% of the previous rate if reaction resolves. The number of participants for whom the study dosing was feasible is reported.
Outcome measures
| Measure |
Rituximab
n=4 Participants
Participants received 4 weekly doses of IV rituxan at 375 mg/m\^2 on days 1, 8, 15 and 22
|
|---|---|
|
Feasibility of Using 4 Weekly Rituximab Infusions
|
4 Participants
|
PRIMARY outcome
Timeframe: Individuals were followed for adverse events until day 270The trial was to be terminated early for any grade 3 or 4 toxicity that did not resolve in 2 of the first 5 patients treated, or for any infusion related death. The number of participants who experienced these events \[grade 3 or 4 toxicity that did not resolve in 2 of the first 5 patients treated, or for any infusion related death\] is reported.
Outcome measures
| Measure |
Rituximab
n=4 Participants
Participants received 4 weekly doses of IV rituxan at 375 mg/m\^2 on days 1, 8, 15 and 22
|
|---|---|
|
Toxicity of Rituximab
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline, following the four infusions, at months 3, 6, 12, 18 and 24 following the first infusion.OMS Evaluation Scale of Motor-Performance This scoring system utilizes a scale of 0 to 3, with 0 being unaffected by OMS, and 3 representing those with severe impairment. OMS testing will be scored by two independent scorers who have special expertise in the evaluation and management of children with neurologic disorders. The mean of these scores will be obtained, and a standard error of the mean reported.
Outcome measures
| Measure |
Rituximab
n=4 Participants
Participants received 4 weekly doses of IV rituxan at 375 mg/m\^2 on days 1, 8, 15 and 22
|
|---|---|
|
OMS Evaluation Scale of Motor-Performance
|
NA score on a scale
The raw data for OMS scoring is at the NIH in the offices of Dr. Nina Schor, MD PhD. She was the responsible neurology investigator in this study. The trial closed early due to low accrual and did not enroll adequate numbers to conduct detailed statistical analysis of the raw data. Due to the pandemic and discontinuation of in person activities, a decision was made by the NIH to undertake extensive renovations of the facility. Dr. Schor is unable to access them due to construction.
|
SECONDARY outcome
Timeframe: Baseline; 3, 6, 9 months post treatmentPopulation: These studies were unable to be performed due to withdraw of funding from the study sponsor. 0 of 4 patients participated.
Blood samples to be evaluated for the occurrence of antibody formation and potential effect on pharmacokinetic profiles.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 yearsB cell depletion was measured through analysis of serum IgG levels. The number of participants who experienced B cell depletion is reported.
Outcome measures
| Measure |
Rituximab
n=4 Participants
Participants received 4 weekly doses of IV rituxan at 375 mg/m\^2 on days 1, 8, 15 and 22
|
|---|---|
|
Peripheral B Cell Depletion
|
0 Participants
|
Adverse Events
Rituximab
Serious adverse events
| Measure |
Rituximab
n=4 participants at risk
Participants received 4 weekly doses of IV rituxan at 375 mg/m\^2 on days 1, 8, 15 and 22
|
|---|---|
|
General disorders
Febrile illness with upper respiratory symptoms and dehydration
|
25.0%
1/4 • Number of events 1 • In addition to outlined in-office evaluations, telephone contact to inquire regarding hospitalizations and adverse event questioning will be performed at days 60, 120 and 270 due to the length of time between scheduled testing.
|
Other adverse events
Adverse event data not reported
Additional Information
Jean M. Tersak, M.D.
UPMC Children's Hospital of Pittsburgh
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place