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A phase I clinical trial of tiopronin, a putative neuroprotective agent, in aneurysmal subarachnoid hemorrhage.
[cystinuria]
The
neurotoxic
aldehyde
3
-
aminopropanal
(
3
-
AP
)
contributes
to
brain
injury
following
cerebral
ischemia
.
Tiopronin
(
N-
2
-
mercaptopropionyl-glycine
[
N-
2
-
MPG
]
)
is
a
US
Food
and
Drug
Administration
(
FDA
)
-
approved
drug
for
the
treatment
of
cystinuria
and
a
putative
neuroprotective
agent
that
has
been
shown
to
bind
and
neutralize
3
-
AP
and
reduce
infarct
volumes
.
The
objective
of
this
trial
was
to
establish
the
safety
of
tiopronin
administration
in
patients
with
aneurysmal
subarachnoid
hemorrhage
(
aSAH
)
in
preparation
for
further
trials
of
its
efficacy
as
a
neuroprotective
agent
in
this
disease
process
.
This
Phase
I
dose-escalation
trial
enrolled
three
-patient
cohorts
using
a
conventional
"
3
+
3
"
study
design
.
Tiopronin
dose
began
at
1
g
/
d
until
aSAH
Day
14
.
Each
subsequent
cohort
received
a
dose
of
tiopronin
based
on
predetermined
guidelines
.
A
maximum
dose
of
3
g
/
d
was
selected
,
because
this
is
the
maximum
FDA-approved
dose
for
long
-term
cystinuria
treatment
.
Subjects
were
monitored
for
known
side
effects
of
tiopronin
.
Nine
patients
were
enrolled
,
the
minimum
number
required
based
on
the
study
design
.
None
of
these
patients
experienced
serious
side
effects
attributable
to
tiopronin
,
and
no
adverse
events
were
noted
that
could
not
be
attributed
to
the
pathophysiology
of
aSAH
.
The
administration
of
3
g
/
d
of
tiopronin
following
aSAH
for
up
to
14
days
appears
to
be
safe
and
without
the
side
effects
associated
with
long
-term
use
.
Plans
for
a
randomized
,
placebo-controlled
Phase
II
trial
of
tiopronin
for
neuroprotection
following
aSAH
are
underway
.
Diseases
Validation
Diseases presenting
"a putative neuroprotective agent that has been shown to bind and neutralize 3"
symptom
cystinuria
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