Use of Rituximab in Opsoclonus-Myoclonus in Children With Neuroblastoma

NCT00202930 | Terminated Phase 2 Results DMC

• 1 other identifier: IRB0405652
• Study Type: interventional
• Target: 4
• Locations: 0 countries
• Sites: N/A

Brief Summary

The purpose of this study is to evaluate the feasibility of giving four weekly doses of Rituximab (anti-CD20 antibody) in the treatment of children with refractory neuroblastoma associated opsoclonus-myoclonus. Patients must have continued symptoms of opsoclonus, myoclonus and or ataxia despite surgical resection and a minimum of one month of steroid therapy. Evaluations include clinical symptoms of opsoclonus-myoclonus and ataxia as well as detailed evaluation of learning and development.

Conditions

Neuroblastoma; Opsoclonus-myoclonus

Keywords

neuroblastoma; Opsoclonus-myoclonus; rituximab

Outcome Measures

Primary Outcomes (2)

• Feasibility of Using 4 Weekly Rituximab Infusions

  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.

  4 weeks

• Toxicity of Rituximab

  The 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.

  Individuals were followed for adverse events until day 270

Secondary Outcomes (3)

• OMS Evaluation Scale of Motor-Performance

  Baseline, following the four infusions, at months 3, 6, 12, 18 and 24 following the first infusion.

• Human-anti-chimeric Antibody (HACA) Development

  Baseline; 3, 6, 9 months post treatment

• Peripheral B Cell Depletion

  2 years

Study Arms (1)

Rituximab

OTHER

Single Arm

Drug: anti-CD20 (Rituximab)

Interventions

anti-CD20 (Rituximab) DRUG

4 weekly doses of IV rituxan at 375 mg/m2 on days 1, 8, 15 and 22

Also known as: Rituxan

Rituximab

Eligibility Criteria

• Age: 2 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Pathologic confirmation of diagnosis of neuroblastoma Surgical resection of primary tumor Symptoms of OMS despite a minimum of one month of steroid therapy Must meet all laboratory criteria to demonstrate adequate organ function -

You may not qualify if:

• Patients currently receiving systemic chemotherapy for treatment of neuroblastoma Patients with documented active infection Patients who are HIV, Hep B or Hep C positive Organ toxicity from any prior therapy or surgical intervention must be resolved prior to study entry

Sponsors & Collaborators

• Jean M. Tersak, M.D.: lead
• Genentech, Inc.: collaborator

MeSH Terms

Conditions

Neuroblastoma; Opsoclonus-Myoclonus Syndrome

Interventions

Rituximab

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Paraneoplastic Syndromes; Ocular Motility Disorders; Central Nervous System Diseases; Nervous System Diseases; Cranial Nerve Diseases; Neurodegenerative Diseases; Myoclonus; Dyskinesias; Neurologic Manifestations; Eye Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Murine-Derived; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Results Point of Contact

• Title: Jean M. Tersak, M.D.
• Organization: UPMC Children's Hospital of Pittsburgh

jean.tersak@chp.edu

412-692-8570

Study Officials

• Jean M Tersak, M.D.

  Children's Hospital of Pittsburgh Department of Hematology Oncology and BMT

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: September 12, 2005
• First Posted: September 20, 2005
• Study Start: July 1, 2005
• Primary Completion: December 1, 2008
• Study Completion: February 5, 2009
• Last Updated: January 22, 2021
• Results First Posted: January 22, 2021

Record last verified: 2021-01