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

• Status: Withdrawn
• Trial end date: 2019-01-01
• Target enrollment: 0
• Participant gender: All

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
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: New York University School of Medicine; NYU Langone Health
• Treatments: Droxidopa; Norepinephrine

Criteria

Inclusion Criteria:

• Male or female 15 years old or older

• Patient carrying the genetic mutation for the NTRK1 gene

• Patients (or guardian if patient it is underage) have signed a written consent to participate in the study after being fully informed about the procedure of the study and their right to withdraw at anytime during it.

Exclusion Criteria:

• Patients taking amphetamines, norepinephrine reuptake inhibitors.

• Patients taking any medication that can interact with the study drug L- DOPS.

• Patients with previous severe hypertension (systolic blood pressure >170 mmHg)

• Patients with arrhythmias or any other cardiac condition.

• Women who are pregnant or breast feeding

• Have a renal or hepatic disease