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| Sponsor: | Rockefeller University |
|---|---|
| Information provided by (Responsible Party): | Rockefeller University |
| ClinicalTrials.gov Identifier: | NCT00378326 |
Purpose
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. POMA - 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 | Intervention |
|---|---|
|
Paraneoplastic Syndromes |
Drug: Tacrolimus (FK506) |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Immunotherapy of the Paraneoplastic Syndromes |
| Estimated Enrollment: | 100 |
| Study Start Date: | April 2006 |
| Estimated Study Completion Date: | May 2015 |
| Estimated Primary Completion Date: | May 2015 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
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 (FK506)
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
|
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 (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 in conjunction with MDs there. Dr. Robert Darnell, being credentialed at both institutions, will be the primary person responsible for monitoring all portions of the study performed at MSKCC and 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 NY, 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 NY, 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 (see 10.g.). 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.
Eligibility| Ages Eligible for Study: | 16 Years to 75 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| Contact: Mayu Frank, NP | 212-327-7443 | frankm@rockefeller.edu |
| United States, New York | |
| Rockefeller University Hospital | Recruiting |
| New York, New York, United States, 10021 | |
| Principal Investigator: Robert Darnell, MD, PhD | |
| Principal Investigator: | Robert Darnell, MD, PhD | Rockefeller University |
More Information
| Responsible Party: | Rockefeller University |
| ClinicalTrials.gov Identifier: | NCT00378326 History of Changes |
| Other Study ID Numbers: | RDA-0572 |
| Study First Received: | September 18, 2006 |
| Last Updated: | January 10, 2012 |
| Health Authority: | United States: Institutional Review Board United States: Food and Drug Administration |
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paraneoplastic syndrome |
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Paraneoplastic Syndromes Neoplasms Tacrolimus Immunosuppressive Agents |
Immunologic Factors Physiological Effects of Drugs Pharmacologic Actions |