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A Study of Norepinephrine in Patients With Congenital Insensitivity to Pain and Anhidrosis

NCT02624310 · Status: WITHDRAWN · Phase: PHASE2 · Type: INTERVENTIONAL

Last updated 2016-09-12

No results posted yet for this study

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.

Conditions

  • HSAN Type IV

Interventions

DRUG

Droxidopa (L-DOPS)

Patients will be given their initial 100 mg of L-DOPS (visit 2a). The dose of L-DOPS will be increased in escalation in the following visits. Each day the dose will be increased 100 mg. The escalating doses of L-DOPS will be 200 mg (visit 2b), 300 mg (visit 2c), 400 mg (visit 2d), 500 mg (visit 2e) and 600 mg (visit 2f). Following this titration step, L-DOPS will be withdrawn from the subjects for a minimum of two weeks, when the subject will return for visit 3.Visit 3a,b,c,d,e,f: Carbidopa open-label dose titration. This titration step will be similar than L-DOPS titration. Carbidopa starting dose will be 100 mg (visit 3a) and will be increased 100 mg each visit up to a maximum of 600 mg (visit 3f). Following carbidopa titration step, patients will be withdrawn from the drug for minimum of two weeks, then the subject will return for the next titration step (visit 4).

DRUG

Placebo

Sponsors & Collaborators

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
CROSSOVER

Eligibility

Min Age
15 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-01-31
Primary Completion
2019-01-31
Completion
2019-01-31

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02624310 on ClinicalTrials.gov