ACADIA and Neuren have both re-announced the onset of the DAFFODIL trial for children with Rett syndrome, ages 2-5. Per ACADIA’s announcement:
Acadia will be conducting an additional clinical study of trofinetide. DAFFODIL (TM)
will be a 12-week, multicenter, open-label safety, tolerability and
pharmacokinetics study in girls with diagnosed Rett syndrome, 2 to 5 years of
age, followed by a possible open-label extension for up to an additional 21
months.
What does all THAT mean?
There will be more than one site. Everyone will get the real thing, no placebo. The trial is to assess safety and tolerability and MAY have an open-label extension for POSSIBLY up to 21 additional months for a POSSIBLE total of 24 months.
Please recognize that the trial design criteria for DAFFODIL(TM) is limited to a small
number (approximately 10 total) of female participants in specified weight ranges,
to enable measurements of the blood levels of trofinetide, an investigational drug,
and safety assessments across this age group.
What does that mean?
10 girls will be divided into groups based on weight and age to present an even distribution of participants that will provide equitable information throughout this age range.
The contact for the study is:
| Contact: Maile Krumpschmidt | 442-232-2001 | rettsyndomestudies@precisionformedicine.com |
You can read the full announcement on rettsyndrome.org
You can view inclusion/exclusion criteria at www.clinicaltrials.gov
The next update will include the trial sites, which have yet to be determined.
I would like to end with this, one of my favorite poems.
I Wandered Lonely as a Cloud
I wandered lonely as a cloud
That floats on high o’er vales and hills,
When all at once I saw a crowd,
A host, of golden daffodils;
Beside the lake, beneath the trees,
Fluttering and dancing in the breeze.
Continuous as the stars that shine
And twinkle on the milky way,
They stretched in never-ending line
Along the margin of a bay:
Ten thousand saw I at a glance,
Tossing their heads in sprightly dance.
The waves beside them danced; but they
Out-did the sparkling waves in glee:
A poet could not but be gay,
In such a jocund company:
I gazed—and gazed—but little thought
What wealth the show to me had brought:
For oft, when on my couch I lie
In vacant or in pensive mood,
They flash upon that inward eye
Which is the bliss of solitude;
And then my heart with pleasure fills,
And dances with the daffodils.
William Wordsworth
One day, that is what we’ll see- all those little babies dancing like flowers in the breeze and our hearts will fill with pleasure and dance along.
Trail to a Texas Trial 