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Trial record 2 of 2 for:    "Autonomic Neuropathy" | "Sympathomimetics"

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

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ClinicalTrials.gov Identifier: NCT02624310
Recruitment Status : Withdrawn
First Posted : December 8, 2015
Last Update Posted : September 12, 2016
Sponsor:
Information provided by (Responsible Party):
NYU Langone Health

Brief 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.


Condition or disease Intervention/treatment Phase
HSAN Type IV Drug: Droxidopa (L-DOPS) Drug: Placebo Phase 2

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase II, Randomized, Double Blind, Cross-over, Placebo-controlled Study on Norepinephrine Replenishment Therapy Using L-DOPS in Congenital Insensitivity to Pain With Anhidrosis Patients
Study Start Date : January 2016
Estimated Primary Completion Date : January 2019
Estimated Study Completion Date : January 2019


Arm Intervention/treatment
Experimental: Droxidopa First, Placebo Second
Participants in this arm will receive droxidopa first, then cross over and receive placebo
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).
Other Name: Northera

Drug: Placebo
Experimental: Placebo First, Droxidopa Second
Participants in this arm will receive placebo first, then cross over and receive droxidopa
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).
Other Name: Northera

Drug: Placebo



Primary Outcome Measures :
  1. Incidence of Treatment-Emergent Adverse Events [ Time Frame: 8 weeks ]
    Safety and tolerability will be assessed using general physical and neurological examinations, vital signs including blood pressure and heart rate, temperature and body weight, blood chemistries including serum creatinine, electrolytes, transaminases and liver function tests, 12 lead electrocardiograms and adverse events monitoring.



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Ages Eligible for Study:   15 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

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Responsible Party: NYU Langone Health
ClinicalTrials.gov Identifier: NCT02624310     History of Changes
Other Study ID Numbers: 14-01774
First Posted: December 8, 2015    Key Record Dates
Last Update Posted: September 12, 2016
Last Verified: September 2016
Additional relevant MeSH terms:
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Hereditary Sensory and Autonomic Neuropathies
Sympathomimetics
Pain Insensitivity, Congenital
Hypohidrosis
Sweat Gland Diseases
Skin Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Nervous System Malformations
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Polyneuropathies
Congenital Abnormalities
Norepinephrine
Droxidopa
Adrenergic alpha-Agonists
Adrenergic Agonists
Adrenergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Autonomic Agents
Peripheral Nervous System Agents
Vasoconstrictor Agents
Antiparkinson Agents
Anti-Dyskinesia Agents