A Trial of Cognitive Behavioral Therapy in Familial Dysautonomia

An Open-Label Pilot Trial of Cognitive Behavioral Therapy in Familial Dysautonomia

To determine the effect of cognitive behavioral therapy (CBT) in the severity of anxiety and depression in adult patients with familial dysautonomia. Patients will be enrolled in an 8-week CBT program. All CBT sessions will be done either in person at the NYU Dysautonomia Center or over the phone to help accommodate disability and potential physical limitations of our patient population.

Study Overview

• Status: Completed
• Conditions: Anxiety Disorders; Familial Dysautonomia; Dysthymia; Paroxysmal Hypertension; Autosomal Recessive Disease
• Intervention / Treatment: Behavioral: 8-week CBT Program

Detailed Description

Cognitive behavioral therapy (CBT), defined as a program of interventions that utilize education to teach relaxation, healthy coping skills, stress management, assertiveness training in order to help the individual identify and correct maladaptive beliefs in combination with education to help practice symptom reduction and improve quality of life and function. Patients will participate in eight forty-five minute sessions of CBT with a mental health therapist in order to help treat generalized anxiety disorder, anxiety disorder due to another medical condition (familial dysautonomia), major depressive disorder, persistent depressive disorder (dysthymia), substance/medication- induced depressive disorder, depressive disorder due to another medical condition.

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • New York
    • New York, New York, United States, 10016
      • New York University School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Diagnosis of FD (genetically confirmed)
• DSM-V criteria of major depressive disorder OR anxiety disorder
• STAI score ≥ 25 OR a PHQ-9 depression scale score ≥ 5 or greater
• Willing and able to complete 8 CBT sessions
• Maintain constant psychoactive medication through out study and no concurrent talk therapy from another therapist.

Exclusion Criteria:

• Currently suicidal or having current suicidal ideations
• Currently under psychiatric treatment for depression or anxiety
• Have started any psychoactive medication within 3 months prior to screening

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

EXPERIMENTAL: Cognitive behavioral therapy (CBT); The patient would participate in eight forty-five minute sessions of CBT with a mental health therapist. Cognitive behavioral therapy (CBT), defined as a program of interventions that utilize education to teach relaxation, healthy coping skills, stress management, assertiveness training in order to help the individual identify and correct maladaptive beliefs in combination with education to help practice symptom reduction and improve quality of life and function.

Behavioral: 8-week CBT Program

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Patient Health Questionnaire (PHQ-9) depression scale	Short, but comprehensive scale that consists specifically to the criteria for diagnosis in the DSM-V. The PHQ-9 is effective and sensitive as an instrument in establishing tentative depressive disorder diagnoses while also assessing severity of depressive symptoms. The brevity and criterion validity of the scale make it an appropriate, dual purpose instrument for assessing the severity of depressive disorder within a clinical trial.	8 Weeks
Rosenberg Self-Esteem Scale	10-item questionnaire that objectively measures global self-worth. The Rosenberg Self-Esteem scale is a 10-item questionnaire that objectively measures global self-worth. The Rosenberg self-esteem scale is a 10-item scale scored using a four-point response. The scale has extensive and acceptable reliability and validity, both convergent and discriminant	8 Weeks
State-Trait Anxiety Inventory (STAI)	The STAI has 40 items, 20 items for each of the S-Anxiety and T-Anxiety sub scales. The STAI was chosen for brevity while also providing a broad coverage of DSM V diagnostic criteria for anxiety. The STAI has proven efficacy in measuring valid self-reports of anxiety symptoms and propensity for anxiety.	8 Weeks

Collaborators and Investigators

• Sponsor: NYU Langone Health

Study record dates

Study Major Dates

• Study Start (ACTUAL): December 6, 2016
• Primary Completion (ACTUAL): July 20, 2018
• Study Completion (ACTUAL): July 20, 2018

Study Registration Dates

• First Submitted: January 5, 2017
• First Submitted That Met QC Criteria: January 5, 2017
• First Posted (ESTIMATE): January 6, 2017

Study Record Updates

• Last Update Posted (ACTUAL): January 29, 2019
• Last Update Submitted That Met QC Criteria: January 25, 2019
• Last Verified: January 1, 2019

More Information

Terms related to this study

• Keywords: Familial dysautonomia
• Additional Relevant MeSH Terms: Mental Disorders; Nervous System Diseases; Mood Disorders; Congenital Abnormalities; Genetic Diseases, Inborn; Neuromuscular Diseases; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Depressive Disorder; Nervous System Malformations; Polyneuropathies; Hereditary Sensory and Autonomic Neuropathies; Anxiety Disorders; Primary Dysautonomias; Autonomic Nervous System Diseases; Dysthymic Disorder; Dysautonomia, Familial
• Other Study ID Numbers: 16-01823

Drug and device information, study documents

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