A Study of Norepinephrine in Patients With Congenital Insensitivity to Pain and Anhidrosis
Status:
Withdrawn
Trial end date:
2019-01-01
Target enrollment:
Participant gender:
Summary
The aim of this study is to increase norepinephrine levels in a population of young adults
where NE levels are very low or undetectable. In order to achieve this, the optimal dose will
be determined in a titration step. In the titration step, different doses of L-DOPS will be
tested in order to find the optimal and safest dose suitable for each individual enrolled in
the study. Because L-DOPS has never been used in the US in children or young adults, with
this titration step investigators will also determine the safest dose for this population.
Currently, L-DOPS is being used in our center to treat othostatic hypotension in autonomic
failure. The titration step for this study starts with the dose of 100 mg and increases in an
escalating manner up to a maximum of 600 mg a day (see investigational brochure attached).
L-DOPS has been developed in capsules for oral used and all the previous safety data has been
performed using this route. Oral route is the one that will used during study.
Carbidopa is well tolerated, safe in children and it has been used in this population in the
US without severe adverse effects.
Phase:
Phase 2
Details
Lead Sponsor:
New York University School of Medicine NYU Langone Health