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(RDA-0572) Immunotherapy of the Paraneoplastic Syndromes
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Immunotherapy of the Paraneoplastic Syndromes (RDA-0572)
Principal Investigator:
Robert B. Darnell M.D., Ph.D.
Investigators:
Mayu Frank N.P.
Contact Information:
Principal Investigator:
Robert B. Darnell M.D., Ph.D.
Investigators:
Mayu Frank N.P.
Contact Information:
Mayu Frank, ANP
Rockefeller University
1230 York Avenue
New Y ork, NY 10065
Rockefeller University
1230 York Avenue
New Y ork, NY 10065
Telephone: 212-327-7443
Email: frankm@rockefeller.edu
Email: frankm@rockefeller.edu
Enrollment Status:
Open to Enrollment
Brief Summary of Protocol:
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.
Detailed Description of Protocol:
Patients typically spend two to three weeks as in-patients at the Rockefeller University Hospital. Patients will have a complete history as well as complete general and neurologic examinations, and will be assessed for baseline neurologic, hepatic, hematopoietic, cardiovascular, pulmonary, and renal function (including 24 hour creatinine clearance). Eligible patients will then have a lumbar puncture (spinal tap) and a leukapheresis, a procedure like blood donation during which white blood cells are removed but red blood cells are returned. Tacrolimus will be given in two divided oral doses, in conjunction with oral prednisone. Patients will be treated for up to three weeks, at which time they will be discontinued from treatment with tacrolimus and prednisone unless they have shown definite signs of a beneficial clinical response. Patients will have electrolyte, metabolic, and hematologic measurements, peak and trough serum tacrolimus levels, renal function, and neurologic status serially monitored, requiring approximately one tablespoon of blood per assay. Patients who cannot tolerate tacrolimus without renal dysfunction will be terminated from protocol. Repeat leukapheresis and/or large volume blood draw, and lumbar puncture may be performed either during or after the in-patient stay, with the patient’s consent. Patients will be followed after leaving the hospital to monitor for clinical changes and for drug side-effects.
What specifically makes a person eligible for the study?
You may be eligible to enter this study:
All patients with paraneoplastic neurologic disorders may be eligible, including patients with:
The Hu syndrome (paraneoplastic subacute sensory neuropathy/encephalomyelitis, limbic encephalitis, autonomic neuropathy)
Yo-mediated paraneoplastic cerebellar degeneration (PCD)
Ri-mediated paraneoplastic opsoclonus-myoclonus ataxia (POMA)
Uncharacterized neurologic syndromes which are thought to be paraneoplastic
Gender:
Both
Age(s):
16-75 years old
Children permitted to participate:
No
Potential Benefits.....
There is a possible direct benefit to patients for participating in this study. Given the reasonable probability of T lymphocyte involvement in these disorders, patients may hope to experience clinical remission or a halting of progression with the proposed treatment protocol. Furthermore, patient participation may lead to other novel forms of treatment.
Compensation:
There is no cost to you for being in this research study
Open to Enrollment
Brief Summary of Protocol:
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.
Detailed Description of Protocol:
Patients typically spend two to three weeks as in-patients at the Rockefeller University Hospital. Patients will have a complete history as well as complete general and neurologic examinations, and will be assessed for baseline neurologic, hepatic, hematopoietic, cardiovascular, pulmonary, and renal function (including 24 hour creatinine clearance). Eligible patients will then have a lumbar puncture (spinal tap) and a leukapheresis, a procedure like blood donation during which white blood cells are removed but red blood cells are returned. Tacrolimus will be given in two divided oral doses, in conjunction with oral prednisone. Patients will be treated for up to three weeks, at which time they will be discontinued from treatment with tacrolimus and prednisone unless they have shown definite signs of a beneficial clinical response. Patients will have electrolyte, metabolic, and hematologic measurements, peak and trough serum tacrolimus levels, renal function, and neurologic status serially monitored, requiring approximately one tablespoon of blood per assay. Patients who cannot tolerate tacrolimus without renal dysfunction will be terminated from protocol. Repeat leukapheresis and/or large volume blood draw, and lumbar puncture may be performed either during or after the in-patient stay, with the patient’s consent. Patients will be followed after leaving the hospital to monitor for clinical changes and for drug side-effects.
What specifically makes a person eligible for the study?
You may be eligible to enter this study:
All patients with paraneoplastic neurologic disorders may be eligible, including patients with:
The Hu syndrome (paraneoplastic subacute sensory neuropathy/encephalomyelitis, limbic encephalitis, autonomic neuropathy)
Yo-mediated paraneoplastic cerebellar degeneration (PCD)
Ri-mediated paraneoplastic opsoclonus-myoclonus ataxia (POMA)
Uncharacterized neurologic syndromes which are thought to be paraneoplastic
Gender:
Both
Age(s):
16-75 years old
Children permitted to participate:
No
Potential Benefits.....
There is a possible direct benefit to patients for participating in this study. Given the reasonable probability of T lymphocyte involvement in these disorders, patients may hope to experience clinical remission or a halting of progression with the proposed treatment protocol. Furthermore, patient participation may lead to other novel forms of treatment.
Compensation:
There is no cost to you for being in this research study
