Familial DysautonomiaNCT03911063
Telemedicine Clinical Trial for Cognitive Behavioral Therapy in Familial Dysautonomia
A study testing CBT sessions and Talking Sessions in people with Familial Dysautonomia, run by NYU Langone Health at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Familial Dysautonomia
- What is being tested?
- CBT sessions and Talking Sessions
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Apr 2019to 1 Apr 2021
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
- Conditions
- Familial Dysautonomia
- Being tested
- CBT sessions, Talking Sessions
- Keywords
- Telemedicine
- Medical subject headings
- Dysautonomia, Familial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CBT sessionsBehavioural
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.
Talking SessionsBehavioural
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.
| Study group | Role | What they receive |
|---|---|---|
| Cognitive behavioral therapy (CBT) | Experimental | Not described Receives: Behavioral: CBT sessions |
| Placebo Talking Sessions | Active comparator | Not described Receives: Behavioral: Talking Sessions |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Week 17
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Apr 2019
- Study start1 Apr 2019 (estimated)
- Primary completion1 Apr 2021 (estimated)
- Study completion1 Apr 2021 (estimated)
Why it stopped: Personnel unavailable to run the study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Familial DysautonomiaStudies of the same condition, open and under way.
- More trials run by NYU Langone HealthThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- NYU Langone Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Horacio Kaufmann, Principal investigator, New York Langone Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03911063
- Sponsor's protocol ID
- 18-01152
- Status last verified
- Apr 2020
- First submitted
- 28 Mar 2019
- Last updated
- 28 Apr 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-Blind Placebo-Controlled Telemedicine Clinical Trial for Cognitive Behavioral Therapy in Familial Dysautonomia
- ClinicalTrials.gov ID
- NCT03911063
- Acronym / sponsor ID
- 18-01152
- Conditions
- Familial Dysautonomia
- Keywords
- Telemedicine
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Personnel unavailable to run the study
- Enrolment
- 0 participants (actual)
- Lead sponsor
- NYU Langone HealthUniversity, hospital or other
- Study start
- 1 Apr 2019 (estimated)
- Primary completion
- 1 Apr 2021 (estimated)
- Study completion
- 1 Apr 2021 (estimated)
- First posted
- 10 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cognitive behavioral therapy (CBT) | Experimental | Behavioral: CBT sessions |
| Placebo Talking Sessions | Active comparator | Behavioral: Talking Sessions |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CBT sessionsweekly 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. | Behavioural | Cognitive behavioral therapy (CBT) |
| Talking Sessions20-40 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. | Behavioural | Placebo Talking Sessions |
Primary outcome
STAI anxiety score
Time frame: Baseline
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).
STAI anxiety score
Time frame: Week 2
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).
STAI anxiety score
Time frame: Week 9
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).
STAI anxiety score
Time frame: Week 17
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).
PHQ-9 depression score
Time frame: Baseline
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.
PHQ-9 depression score
Time frame: Week 2
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.
PHQ-9 depression score
Time frame: Week 9
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.
PHQ-9 depression score
Time frame: Week 17
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.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2020.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Horacio Kaufmann principal investigator
New York Langone Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing CBT sessions and Talking Sessions in people with Familial Dysautonomia, run by NYU Langone Health at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 28 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
STAI anxiety score (time frame: Baseline). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Apr 2020.