Dr. Ranjan Dohil
Dr. Ranjan Dohil
The purpose of this study is to obtain data that will ultimately help
to create a controlled-release preparation of cysteamine, which can hopefully
be taken without the unwanted gastrointestinal side-effects.
We would
like to enroll 6 patients with cystinosis to this study. Volunteers should be12
years and over, and will be flown to San Diego along with a one
parent.
A specially designed soft tube (about one-tenth of an inch in
diameter) has been constructed in Australia specifically for this study. The
tube will be inserted into the nose and slowly passed into the stomach and
intestine. Cysteamine will then be administered into different sites of the
intestine. Blood samples will be measured to determine the absorption / effect
of cysteamine when it is administered into different sites of the intestine. We
will also measure blood gastrin levels, the hormone that is normally
responsible for producing stomach acid and causing gastrointestinal symptoms,
following a dose of cysteamine.
Patients will receive generous
compensation for this study.
Information from this study may be useful
in helping us to develop a more effective way to deliver cysteamine to the
body, which in turn may allow cysteamine to be given less than 4 times a day
without diminishing its effect.
This is a follow-on from the Prilosec study. Nexium is the new
generation of Prilosec and is thought to be more effective in suppressing acid
production (which we now know is a major factor in the causation of
gastrointestinal symptoms suffered by patients with cystinosis).
We
would like to enroll 12 patients with cystinosis to this study. Volunteers
should be one year and over, and will be flown to San Diego along with a one
parent. Patients will undergo upper gastrointestinal endoscopy with biopsies;
the latter will be examined for inflammation and cystine crystals. Acid
collection studies will be performed and samples will be taken from either a
nasal tube or a gastrostomy tube (if already in place). Blood samples will be
measured to determine gastrin levels and cysteamine levels in the body after a
single dose of cysteamine has been given. The acid and blood studies will be
repeated following 4 months of treatment with Nexium. Patients will receive
compensation for this study. Patients who took part in the Prilosec study are
encouraged to enroll.
This study will be particularly useful as it will
allow us to (1) better understand gastrointestinal disease associated with
cystinosis, (2) determine whether stronger acid suppression with Nexium will
further diminish GI symptoms and improve quality of life, and (3) determine
whether cysteamine is better absorbed from the intestine when stomach acid
production is reduced. The latter may have implications for the long term
dosing and frequency of cysteamine ingestion.
Meredith Fidler, PhD. will be the study coordinator and approximately
September 15th 2003 will begin interviewing patients for their possible
participation in both of these studies. Dr. Fidler will be able to answer your
questions, send you copies of the protocol, the consent forms, and make all
appropriate arrangements. Dr. Fidler's contact information will be available
shortly, in the meantime if any patient or parents have any questions regarding
either of these studies they can contact Meredith by email:
mfidler@ucsd.edu or phone:
(619) 543-2049.