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Oral liarozole in the treatment of patients with moderate/severe lamellar ichthyosis: results of a randomized, double-blind, multinational, placebo-controlled phase II/III trial.
[lamellar ichthyosis]
Oral
liarozole
,
a
retinoic
acid
metabolism-blocking
agent
,
may
be
an
alternative
to
systemic
retinoid
therapy
in
patients
with
lamellar
ichthyosis
.
To
demonstrate
the
efficacy
and
safety
of
once-daily
oral
liarozole
in
the
treatment
of
moderate
/
severe
lamellar
ichthyosis
.
This
was
a
double
-blind
,
multinational
,
parallel
phase
II
/
III
trial
(
NCT
00282724
)
.
Patients
aged
≥
14
years
with
moderate
/
severe
lamellar
ichthyosis
[
Investigator
's
Global
Assessment
(
IGA
)
score
≥
3
]
were
randomized
3
:
3
:
1
to
receive
oral
liarozole
(
75
or
150
mg
)
or
placebo
once
daily
for
12
weeks
.
Assessments
included
:
IGA
;
a
five
-point
scale
for
erythema
,
scaling
and
pruritus
severity
;
Short
Form-
36
health
survey
;
Dermatology
Life
Quality
Index
(
DLQI
)
;
and
safety
parameters
.
The
primary
efficacy
variable
was
response
rate
at
week
12
(
responder
:
≥
2
-
point
decrease
in
IGA
from
baseline
)
.
Sixty
-
four
patients
were
enrolled
.
At
week
12
,
11
/
27
(
41
%
;
liarozole
75
mg
)
,
14
/
28
(
50
%
;
liarozole
150
mg
)
and
one
out
of
nine
(
11
%
;
placebo
)
patients
were
responders
;
the
difference
between
groups
(
liarozole
150
mg
vs
.
placebo
)
was
not
significant
(
P
=
0
.
056
)
.
Mean
IGA
and
scaling
scores
decreased
from
baseline
in
both
liarozole
groups
at
weeks
8
and
12
vs
.
placebo
;
erythema
and
pruritus
scores
were
similar
between
treatment
groups
.
Improvement
in
DLQI
score
was
observed
in
both
liarozole
groups
.
Treatment
with
liarozole
for
12
weeks
was
well
tolerated
.
The
primary
efficacy
variable
did
not
reach
statistical
significance
,
possibly
owing
to
the
small
sample
size
following
premature
termination
.
However
,
once-daily
oral
liarozole
,
75
and
150
mg
,
improved
scaling
and
DLQI
and
was
well
tolerated
in
patients
with
moderate
/
severe
lamellar
ichthyosis
.
Diseases
Validation
Diseases presenting
"small sample size"
symptom
lamellar ichthyosis
proteus syndrome
systemic capillary leak syndrome
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