<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT03013777</url>
  </required_header>
  <id_info>
    <org_study_id>16-01823</org_study_id>
    <nct_id>NCT03013777</nct_id>
  </id_info>
  <brief_title>A Trial of Cognitive Behavioral Therapy in Familial Dysautonomia</brief_title>
  <official_title>An Open-Label Pilot Trial of Cognitive Behavioral Therapy in Familial Dysautonomia</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>NYU Langone Health</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>NYU Langone Health</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      To determine the effect of cognitive behavioral therapy (CBT) in the severity of anxiety and&#xD;
      depression in adult patients with familial dysautonomia. Patients will be enrolled in an&#xD;
      8-week CBT program. All CBT sessions will be done either in person at the NYU Dysautonomia&#xD;
      Center or over the phone to help accommodate disability and potential physical limitations of&#xD;
      our patient population.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Cognitive behavioral therapy (CBT), defined as a program of interventions that utilize&#xD;
      education to teach relaxation, healthy coping skills, stress management, assertiveness&#xD;
      training in order to help the individual identify and correct maladaptive beliefs in&#xD;
      combination with education to help practice symptom reduction and improve quality of life and&#xD;
      function. Patients will participate in eight forty-five minute sessions of CBT with a mental&#xD;
      health therapist in order to help treat generalized anxiety disorder, anxiety disorder due to&#xD;
      another medical condition (familial dysautonomia), major depressive disorder, persistent&#xD;
      depressive disorder (dysthymia), substance/medication- induced depressive disorder,&#xD;
      depressive disorder due to another medical condition.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">December 6, 2016</start_date>
  <completion_date type="Actual">July 20, 2018</completion_date>
  <primary_completion_date type="Actual">July 20, 2018</primary_completion_date>
  <phase>N/A</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Patient Health Questionnaire (PHQ-9) depression scale</measure>
    <time_frame>8 Weeks</time_frame>
    <description>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.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Rosenberg Self-Esteem Scale</measure>
    <time_frame>8 Weeks</time_frame>
    <description>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</description>
  </primary_outcome>
  <primary_outcome>
    <measure>State-Trait Anxiety Inventory (STAI)</measure>
    <time_frame>8 Weeks</time_frame>
    <description>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.</description>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">15</enrollment>
  <condition>Dysthymia</condition>
  <condition>Anxiety Disorders</condition>
  <condition>Familial Dysautonomia</condition>
  <condition>Paroxysmal Hypertension</condition>
  <condition>Autosomal Recessive Disease</condition>
  <arm_group>
    <arm_group_label>Cognitive behavioral therapy (CBT)</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>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.</description>
  </arm_group>
  <intervention>
    <intervention_type>Behavioral</intervention_type>
    <intervention_name>8-week CBT Program</intervention_name>
    <arm_group_label>Cognitive behavioral therapy (CBT)</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Diagnosis of FD (genetically confirmed)&#xD;
&#xD;
          -  DSM-V criteria of major depressive disorder OR anxiety disorder&#xD;
&#xD;
          -  STAI score ≥ 25 OR a PHQ-9 depression scale score ≥ 5 or greater&#xD;
&#xD;
          -  Willing and able to complete 8 CBT sessions&#xD;
&#xD;
          -  Maintain constant psychoactive medication through out study and no concurrent talk&#xD;
             therapy from another therapist.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Currently suicidal or having current suicidal ideations&#xD;
&#xD;
          -  Currently under psychiatric treatment for depression or anxiety&#xD;
&#xD;
          -  Have started any psychoactive medication within 3 months prior to screening&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Horacio Kaufmann, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>NYU Langone Health</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>New York University School of Medicine</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10016</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>January 2019</verification_date>
  <study_first_submitted>January 5, 2017</study_first_submitted>
  <study_first_submitted_qc>January 5, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">January 6, 2017</study_first_posted>
  <last_update_submitted>January 25, 2019</last_update_submitted>
  <last_update_submitted_qc>January 25, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">January 29, 2019</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Familial dysautonomia</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Primary Dysautonomias</mesh_term>
    <mesh_term>Autonomic Nervous System Diseases</mesh_term>
    <mesh_term>Dysautonomia, Familial</mesh_term>
    <mesh_term>Anxiety Disorders</mesh_term>
    <mesh_term>Dysthymic Disorder</mesh_term>
  </condition_browse>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03013777
org study id: 16-01823
nct id: NCT03013777
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
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.
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.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Patient Health Questionnaire (PHQ-9) depression scale
time frame: 8 Weeks
description: 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.
measure: Rosenberg Self-Esteem Scale
time frame: 8 Weeks
description: 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
measure: State-Trait Anxiety Inventory (STAI)
time frame: 8 Weeks
description: 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.
arm group label: Cognitive behavioral therapy (CBT)
arm group type: Experimental
description: 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.
intervention type: Behavioral
intervention name: 8-week CBT Program
arm group label: Cognitive behavioral therapy (CBT)
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Horacio Kaufmann, MD
role: Principal Investigator
affiliation: NYU Langone Health
facility: 
country: United States
responsible party type: Sponsor
mesh term: Primary Dysautonomias
mesh term: Autonomic Nervous System Diseases
mesh term: Dysautonomia, Familial
mesh term: Anxiety Disorders
mesh term: Dysthymic Disorder