Telemedicine Clinical Trial for Cognitive Behavioral Therapy in Familial Dysautonomia

NCT03911063 | Withdrawn

• 1 other identifier: 18-01152
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Familial Dysautonomia

Keywords

Telemedicine

Outcome Measures

Primary Outcomes (8)

• 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

Study Arms (2)

Cognitive behavioral therapy (CBT)

EXPERIMENTAL

Behavioral: CBT sessions

Placebo Talking Sessions

ACTIVE COMPARATOR

Behavioral: Talking Sessions

Interventions

CBT sessions BEHAVIORAL

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.

Cognitive behavioral therapy (CBT)

Talking Sessions BEHAVIORAL

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.

Placebo Talking Sessions

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• NYU Langone Health: lead

Study Sites (1)

NYU Langone Health

New York, New York, 10016, United States

Location

MeSH Terms

Conditions

Dysautonomia, Familial

Condition Hierarchy (Ancestors)

Primary Dysautonomias; Autonomic Nervous System Diseases; Nervous System Diseases; Hereditary Sensory and Autonomic Neuropathies; Nervous System Malformations; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Study Officials

• Horacio Kaufmann

  New York Langone Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 28, 2019
• First Posted: April 10, 2019
• Study Start: April 1, 2019
• Primary Completion: April 1, 2021
• Study Completion: April 1, 2021
• Last Updated: April 28, 2020

Record last verified: 2020-04

Locations

US (1)