Clinical Trial Details
— Status: Completed
Administrative data
NCT number |
NCT00378326 |
Other study ID # |
RDA-0572 |
Secondary ID |
|
Status |
Completed |
Phase |
N/A
|
First received |
|
Last updated |
|
Start date |
April 2006 |
Est. completion date |
May 2014 |
Study information
Verified date |
August 2023 |
Source |
Rockefeller University |
Contact |
n/a |
Is FDA regulated |
No |
Health authority |
|
Study type |
Interventional
|
Clinical Trial 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.
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.