Use of Rituximab in Opsoclonus-Myoclonus in Children With Neuroblastoma

The recruitment status of this study is unknown because the information has not been verified recently.
Verified September 2006 by Tersak, Jean M., M.D..
Recruitment status was  Recruiting
Sponsor:
Collaborator:
Genentech
Information provided by:
Tersak, Jean M., M.D.
ClinicalTrials.gov Identifier:
NCT00202930
First received: September 12, 2005
Last updated: September 19, 2006
Last verified: September 2006
  Purpose

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.


Condition Intervention Phase
Neuroblastoma
Opsoclonus-Myoclonus
Drug: anti-CD20 (Rituximab)
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Use of Rituximab in Opsoclonus-Myoclonus in Children With Neuroblastoma

Resource links provided by NLM:


Further study details as provided by Tersak, Jean M., M.D.:

Primary Outcome Measures:
  • Feasibility and toxicity
  • Response

Secondary Outcome Measures:
  • Pharmacokinetic data and HACA development in the pediatric population

Estimated Enrollment: 10
Study Start Date: July 2005
Estimated Study Completion Date: July 2008
Detailed Description:

Opsoclonus-myoclonus ataxia syndrome (OMS) is a rare immune mediated paraneoplastic syndrome that occurs in approximately 2 to 3% of children with neuroblastoma. Children with neuroblastoma associated opsoclonus-myoclonus tend to have a favorable prognosis from the standpoint of the cure of their cancer. Unfortunately,approximately two-thirds of this subgroup of patients are left with long term sequellae of the syndrome, including residual symptoms of opsoclonus, myoclonus, ataxia, learning difficulties and disturbance of sleep and mood.

Multiple lines of evidence indicate an immune mechanism to this rare disorder. This includes occurence of OMS in the post-infectious state, aggressive lymphocytic infiltration of the tumor in children with OMS, and documented responses to therapries that act through suppression of the immune system.

The current study utilizes four weekly doses of anti-CD 20 antibody (rituximab) to treat children with refractory OMS. Refractory disease is defined as continued symptoms of OMS despite surgical resection of the tumor and a minimum of one month of steroid therapy.

All patients have baseline OMS evaluation and detailed neurocognitive testing with all studies being repeated at the completion of the four weekly infusions. OMS testing is repeated at Month 3. OMS testing and detailed neurocognitive testing is conducted at 6 months intervals until 2 years from the initial infusion.

The goal of the study is to utilize this novel therapy to improve long term neurologic and neurodevelopmental outcome in children with refratory neuroblastoma associated opsoclonus-myoclonus.

  Eligibility

Ages Eligible for Study:   2 Months to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

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 -

Exclusion Criteria:

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

-

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00202930

Contacts
Contact: Jean M. Tersak, MD 412-692-5055 jean.tersak@chp.edu

Locations
United States, Pennsylvania
Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15213
Contact: Jean M Tersak, MD    412-692-5055    jean.tersak@chp.edu   
Contact: Nina F Schor, MD, PhD    412-692-5528    nina.schor@chp.edu   
Principal Investigator: Jean M Tersak, MD         
Sponsors and Collaborators
Tersak, Jean M., M.D.
Genentech
Investigators
Principal Investigator: Jean M Tersak, M.D. Children's Hospital of Pittsburgh Department of Hematology Oncology and BMT
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00202930     History of Changes
Other Study ID Numbers: IRB# 0405652
Study First Received: September 12, 2005
Last Updated: September 19, 2006
Health Authority: United States: Food and Drug Administration

Keywords provided by Tersak, Jean M., M.D.:
neuroblastoma
Opsoclonus-myoclonus
rituximab

Additional relevant MeSH terms:
Myoclonus
Opsoclonus-Myoclonus Syndrome
Neuroblastoma
Ocular Motility Disorders
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
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
Central Nervous System Diseases
Cranial Nerve Diseases
Eye Diseases
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
Neoplasms by Site
Paraneoplastic Syndromes
Neurodegenerative Diseases
Rituximab
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions
Immunologic Factors

ClinicalTrials.gov processed this record on August 18, 2014