Telemedicine Clinical Trial for Cognitive Behavioral Therapy in Familial Dysautonomia

A Single-Blind Placebo-Controlled Telemedicine Clinical Trial for Cognitive Behavioral Therapy in Familial Dysautonomia

This is a single-blind, placebo-controlled, telemedicine clinical trial to assess the efficacy of cognitive behavioral therapy (CBT) in adult patients 18 years and older with familial dysautonomia (FD) and anxiety and/or depression and/or obsessive compulsive or related disorders. The trial will enroll 20 adult patients each with FD who have anxiety and/or depression and/or obsessive compulsive or related disorders by the DSM V criteria.

Enrolled participants will be allocated to receive, in a non-randomized fashion, weekly 5-10 min talking sessions (i.e., placebo) for 8 weeks, followed by weekly 30-60 min CBT sessions during 8 weeks. Although investigators will be un-blinded to the intervention, participants will be blinded to the expected effects of each intervention.

Both the talking sessions (i.e., placebo) and CBT sessions will be performed via telemedicine either via a HIPAA secure telemedicine platform or the telephone based on the preference of the individual patient. If a patient specifically requests talking or CBT sessions to be performed in person, this will be accommodated. The use of telemedicine is to accommodate disability and potential physical limitations of this unique patient population. The CBT sessions will be supervised by Lily Armstrong, certified mental health therapist and Dr. Thomas Boes, NYU Clinical Assistant Professor in the Departments of Psychiatric and Neurology.

Study Overview

• Status: Withdrawn
• Conditions: Familial Dysautonomia
• Intervention / Treatment: Behavioral: CBT sessions; Behavioral: Talking Sessions

• Study Type: Interventional
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • New York
    • New York, New York, United States, 10016
      • NYU Langone Health

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Diagnosis of familial dysautonomia (FD).
• DSM-V criteria of major depressive disorder OR anxiety disorder OR obsessive-compulsive behaviors.
• STAI score ≥ 25 OR a PHQ-9 depression scale score ≥ 5 or greater.
• Willing and able to complete 8 sessions of placebo and 8 sessions of CBT.
• If taking an antidepressant it will be maintained at a constant dose throughout study and concurrent talk therapy from another therapist will not be initiated during the study.
• MoCA score of 14 or greater.

Exclusion Criteria:

• Current suicidal attempt or suicidal ideation (subjects with a past history of suicidal ideation or attempt are eligible to participate).
• Patients starting psychoactive medications within 3-months prior to screening.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cognitive behavioral therapy (CBT)	Behavioral: CBT sessions; weekly 20-40 min CBT sessions during 8 weeks; Both the talking sessions (i.e., placebo) and CBT sessions will be performed via telemedicine either via a HIPAA secure telemedicine platform or the telephone based on the preference of the individual patient.
Active Comparator: Placebo Talking Sessions	Behavioral: Talking Sessions; 20-40 min talking sessions (i.e., placebo) for 8 weeks; The placebo talking sessions will be performed by an acute care and palliative care board certified, New York State licensed nurse practitioner at the NYU Dysautonomia Center who is familiar with each FD subject enrolling in the study and an expert in familial dysautonomia. The NP will focus the 20-40 minute conversation on typical medical questions and concerns that the FD patient routinely calls the Center to discuss.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
STAI anxiety score	The range of possible scores for form Y of the STAI varies from a minimum score of 20 to a maximum score of 80 on both the STAI-T and STAI-S subscales. STAI scores are commonly classified as "no or low anxiety" (20-37), "moderate anxiety" (38-44), and "high anxiety" (45-80).	Baseline
STAI anxiety score	The range of possible scores for form Y of the STAI varies from a minimum score of 20 to a maximum score of 80 on both the STAI-T and STAI-S subscales. STAI scores are commonly classified as "no or low anxiety" (20-37), "moderate anxiety" (38-44), and "high anxiety" (45-80).	Week 2
STAI anxiety score	The range of possible scores for form Y of the STAI varies from a minimum score of 20 to a maximum score of 80 on both the STAI-T and STAI-S subscales. STAI scores are commonly classified as "no or low anxiety" (20-37), "moderate anxiety" (38-44), and "high anxiety" (45-80).	Week 9
STAI anxiety score	The range of possible scores for form Y of the STAI varies from a minimum score of 20 to a maximum score of 80 on both the STAI-T and STAI-S subscales. STAI scores are commonly classified as "no or low anxiety" (20-37), "moderate anxiety" (38-44), and "high anxiety" (45-80).	Week 17
PHQ-9 depression score	9 item depression scale of the patient health questionnaire.The total sum of the responses suggests varying levels of depression. Scores range from 0 to 27. In general, a total of 10 or above is suggestive of the presence of depression.	Baseline
PHQ-9 depression score	9 item depression scale of the patient health questionnaire.The total sum of the responses suggests varying levels of depression. Scores range from 0 to 27. In general, a total of 10 or above is suggestive of the presence of depression.	Week 2
PHQ-9 depression score	9 item depression scale of the patient health questionnaire.The total sum of the responses suggests varying levels of depression. Scores range from 0 to 27. In general, a total of 10 or above is suggestive of the presence of depression.	Week 9
PHQ-9 depression score	9 item depression scale of the patient health questionnaire.The total sum of the responses suggests varying levels of depression. Scores range from 0 to 27. In general, a total of 10 or above is suggestive of the presence of depression.	Week 17

Collaborators and Investigators

• Sponsor: NYU Langone Health
• Investigators: Principal Investigator: Horacio Kaufmann, New York Langone Medical Center

Study record dates

Study Major Dates

• Study Start (Anticipated): April 1, 2019
• Primary Completion (Anticipated): April 1, 2021
• Study Completion (Anticipated): April 1, 2021

Study Registration Dates

• First Submitted: March 28, 2019
• First Submitted That Met QC Criteria: April 9, 2019
• First Posted (Actual): April 10, 2019

Study Record Updates

• Last Update Posted (Actual): April 28, 2020
• Last Update Submitted That Met QC Criteria: April 24, 2020
• Last Verified: April 1, 2020

More Information

Terms related to this study

• Keywords: Telemedicine
• Additional Relevant MeSH Terms: Nervous System Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Neuromuscular Diseases; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Nervous System Malformations; Polyneuropathies; Hereditary Sensory and Autonomic Neuropathies; Primary Dysautonomias; Autonomic Nervous System Diseases; Dysautonomia, Familial
• Other Study ID Numbers: 18-01152

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No