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Trial record 6 of 6 for:    Opsoclonus Myoclonus

Immunotherapy of the Paraneoplastic Syndromes

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ClinicalTrials.gov Identifier: NCT00378326
Recruitment Status : Completed
First Posted : September 20, 2006
Results First Posted : February 23, 2016
Last Update Posted : February 23, 2016
Sponsor:
Information provided by (Responsible Party):
Rockefeller University

Brief Summary:
We treat a subset of patients with paraneoplastic neurologic disorders, including those with Yo-mediated paraneoplastic cerebellar degeneration (PCD), the Hu syndrome, which is most commonly associated with small cell lung cancer (SCLC) - paraneoplastic subacute sensory neuropathy, encephalomyelitis, limbic encephalopathy, autonomic neuropathy - and the Ri Syndrome (a.k.a. Paraneoplastic Opsoclonus-Myoclonus Ataxia), as well as those patients suspected to have a paraneoplastic neurologic disorder but in whom a characteristic antibody has not yet been identified. Our treatment protocol consists of immune suppression therapy using tacrolimus (FK506), a potent inhibitor of lymphocyte proliferation that is commonly used to prevent organ transplant rejection.

Condition or disease Intervention/treatment Phase
Paraneoplastic Syndromes Drug: Tacrolimus Not Applicable

Detailed Description:

Patients may stay either in-hospital while being treated with Tacrolimus, receive treatment as an outpatient, or a combination of the two. Additionally, patients who are too sick to be treated at Rockefeller University (RU) (eg. patients actively seizing), but are in need of urgent treatment, may be treated at either Memorial Sloan-Kettering Cancer Center or New York-Presbyterian Hospital. During treatment, patients will undergo blood draws, at set intervals (see section g below), clinical evaluation, possibly repeat leukapheresis or large volume blood draw, and lumbar puncture (see below). Since many patients live far away from New York, some of these procedures may be performed by RU staff or in conjunction with their local MDs.

Patients who are terminated from Tacrolimus treatment after 7-21 days will be followed up as outpatients for evaluation of their neurologic and medical status. Wherever possible, these patients will be seen on days 3 and 10 post treatment termination, and then on a biweekly basis for two months. Since many patients live far away from New York, they may instead be monitored in conjunction with their local MDs. Patients who show a definite clinical response to Tacrolimus may be maintained on a therapeutic dose for up to one year, and will be followed as outpatients. For patients receiving retreatment, they may be treated as inpatients or on an outpatient basis, at the discretion of the PI, on the same schedule as patients being treated initially. Long term improvement or decline in neurologic function will be objectively assessed by neurologic exam, which will be quantified by use of the Karnofsky scale (a measure of functional neurologic status). Since the vast majority of Hu patients decline over a 6-12 month period following diagnosis, a stable or improved Karnofsky score over such a time period will be taken as a measure of successful treatment. Repeat lumbar puncture (up to eight per year) and leukapheresis or large volume blood draw (approx. 100 cc) may be performed (up to four of each per year), especially in the setting of neurologic change, to assess the immune responses to the medications.


Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 26 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Immunotherapy of the Paraneoplastic Syndromes
Study Start Date : April 2006
Actual Primary Completion Date : May 2014
Actual Study Completion Date : May 2014

Resource links provided by the National Library of Medicine

Drug Information available for: Tacrolimus

Arm Intervention/treatment
Experimental: Tacrolimus
Tacrolimus at doses of 0.15- 0.3mg/kg/day in two divided oral doses, in conjunction with, initially, up to 60mg/day of oral prednisone
Drug: Tacrolimus
Tacrolimus at doses of 0.15- 0.3mg/kg/day in two divided oral doses, in conjunction with, initially, up to 60mg/day of oral prednisone
Other Name: FK506




Primary Outcome Measures :
  1. Survival of Patients With Paraneoplastic Disease Who Are Treated With Tacrolimus [ Time Frame: through study completion, median 3 years of follow up ]
    Survival in patients with paraneoplastic disease who are treated with Tacrolimus, from time of tacrolimus treatment


Secondary Outcome Measures :
  1. Cerebrospinal Fluid (CSF) Pleocytosis [ Time Frame: White blood cell count in CSF was measured at two time points, pre- and post-treatment ]


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Ages Eligible for Study:   14 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients diagnosed with Paraneoplastic Disorder

Exclusion Criteria:

  • Metastasis (spread) of cancer to brain, History of additional active malignancy other than non-melanoma skin cancer, History of Hepatitis B, Hepatitis C, HIV or Syphilis.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00378326


Locations
United States, New York
Rockefeller University Hospital
New York, New York, United States, 10021
Sponsors and Collaborators
Rockefeller University
Investigators
Principal Investigator: Robert Darnell, MD, PhD Rockefeller University

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Rockefeller University
ClinicalTrials.gov Identifier: NCT00378326     History of Changes
Other Study ID Numbers: RDA-0572
First Posted: September 20, 2006    Key Record Dates
Results First Posted: February 23, 2016
Last Update Posted: February 23, 2016
Last Verified: January 2016

Keywords provided by Rockefeller University:
paraneoplastic syndrome

Additional relevant MeSH terms:
Syndrome
Paraneoplastic Syndromes
Disease
Pathologic Processes
Neoplasms
Tacrolimus
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Calcineurin Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action