from the Food and Drug Administration Office of Pediatric Therapeutics
and Division of Pediatric and Maternal
;e Food and Drug Administration (FDA) has determined that a
two-year study using a totally im-plantable central venous access device
(TICVAD) to administer an investigational drug or placebo to children
with Duchenne muscular dystrophy
(DMD) may proceed.
An institutional review board (IRB)
asked the FDA to determine if use of
the TICVAD for infusion of placebo
would be acceptable after a subject’s
parent complained about problems
with the use of peripheral intravenous
The randomized, double-blind,
placebo-controlled study will proceed
under criteria established by Additional Safeguards for Children in Clinical Investigations ( 21 CFR part 50
subpart D). Under these regulations,
placement of a TICVAD for children
in the active treatment arm would be
ethically acceptable since they are offered a prospect of direct clinical benefit. A “direct clinical benefit” may
improve the health or well-being of
the individual child enrolled in the
research and results from the intervention being studied (and not from
other clinical interventions included
in the protocol).
Since patients randomized to pla-
cebo do not have a prospect of direct
clinical benefit as they receive no ac-
tive treatment, the IRB sought formal
review by the FDA Pediatric Advi-
sory Committee (PAC), including
an opportunity for public comment.
;e PAC voted unanimously on May
18 to recommend use of a TICVAD
at the discretion of the parent or
guardian in consultation with the
investigator and consulting surgeon.
In addition, adequately informed
and voluntary parent or guardian
permission and child assent must be
obtained. ;e FDA determination
was issued May 25.
The FDA determined that a
TICVAD should be used over other
forms of central venous access, unless
contraindicated, due to its lower incidence of infection, thrombosis and
The meeting materials and FDA determination letter are posted on the FDA
website at http://bit.ly/2ueEYBb.
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