http://www.cystinosisregistry.org () —
The recently launched Cure Cystinosis International Registry
(www.cystinosisregistry.org (http://www.cystinosisregistry.org) ) has
been translated into Spanish, dramatically increasing the
accessibility of the CCIR, which is the first global effort to link
cystinosis patients with scientists and researchers who are developing
new treatments. The rare metabolic and fatal disorder afflicts about
2,000 persons, mostly children, worldwide.
"We're working diligently to reach cystinosis patients worldwide so
they can take advantage of the benefits of the CCIR and help advance
efforts to develop a cure," said Nancy Stack, President and co-founder
of the Cystinosis Research Foundation, one of the registry's key
partners.
Plans are to translate the registry into French within the next two
months. Cystinosis patients from 25 countries have enrolled in the
CCIR, Stack said.
"Patients and families on six continents have registered in the four
months since the CCIR was activated. We're very encouraged with the
response. Top cystinosis researchers are encouraging cystinosis
patients to register with CCIR which is the only international patient
registry in the world dedicated to the cystinosis community," Stack
said.
"Our goal is to have every cystinosis patient worldwide register with
the CCIR. The benefits of CCIR enrollment will be lifesaving and life
changing," Stack said.
The purpose of the CCIR is to identify people with cystinosis globally
and collect their medical histories and information to share
(anonymously) with the cystinosis research community. This information
will allow clinicians, researchers and pharmaceutical companies to
accelerate novel treatments and a cure for cystinosis.
Researchers Worldwide Enrolling With CCIR
Many researchers among the 48 teams of scientists currently funded by
the Cystinosis Research Foundation have registered with the CCIR.
Stack said this clearly demonstrates the importance of the patients'
information, particularly to researchers doing advanced bench and
clinical studies.
The CCIR is a collaboration of 12 cystinosis advocate foundations
worldwide. The countries in which patients are enrolled, thus far,
are: the United States, Canada, Belgium, England, Scotland, France,
Italy, Portugal, Ireland, Germany, Norway, Sweden, Switzerland The
Netherlands, Australia, Russia, Iran, South Africa, Argentina, Chile,
Bolivia, Brazil, Costa Rica, St. Barthelemy and The Bahamas.
Patient Information Is Private, Protected
The CCIR is the only registry created specifically for individuals
with cystinosis and will contain current information regarding
cystinosis clinical trials and studies. All patient information is
de-identified (anonymous) and held in a secure data base accessible
only by the CCIR curator. Information that could identify participants
and their family members will not be shared without their expressed
written approval. Participants will also be able to view aggregate
data allowing them to view how they fit within the larger cystinosis
community.
CCIR officials say recruitment for clinical trials can be a lengthy
process, especially for a rare disease like cystinosis. Participation
in this registry will help speed up the recruitment process and
facilitate and expedite clinical trials, officials say.
In patients with cystinosis, the amino acid cystine accumulates in the
tissue due to the inability of the body to transport cystine out of
one of the compartments of the cell. Cystinosis is a metabolic disease
that slowly destroys every organ in the body, including the liver,
kidneys, eyes, muscles, thyroid and brain. There is a medicine that
prolongs the children's lives, but there is no cure. Most cystinosis
sufferers succumb to the disease or its complications by age 40.
For more information about the Cure Cystinosis International Registry,
contact Betty Cabrera at curator@cystinosisregistry.org
(mailto:curator@cystinosisregistry.org) .