Recruiting

Observational Study

Sponsor:

Dufresne, Craig, MD, PC

Code:

NCT05419245

Conditions

Freeman-Burian Syndrome

Freeman-Sheldon Syndrome

Whistling Face Syndrome

Sheldon-Hall Syndrome

Freeman-Sheldon Syndrome Variant

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Interventions

Guided Health History for Freeman-Burian Syndrome Questionnaire

Freeman-Burian syndrome Semi-Structured Quality of Life Interview

Medical Records Review

PTSD Checklist for DSM-5

Modified Flanagan Quality of Life Scale

Study Details

Brief summary:

The purpose of this study is to evaluate the difference in diagnosis accuracy, treatment outcomes, patient perspectives, facial function and walking ability, emotional and social health, and respiratory complications between Freeman-Burian syndrome (formerly, Freeman-Sheldon or whistling face syndrome), Sheldon-Hall syndrome, and distal arthrogryposis types 3, and 1.

The approximate cumulative total time for study-related activities will be 3 hours, including email communication, survey completion, and a medical interview.

The study will involve completing 6 short ½ to 1-page surveys and participating in a medical interview. Participants may be asked to provide medical records for review. All study-related activities will take place remotely, and no travel is required.

Conditions

Freeman-Burian Syndrome

Freeman-Sheldon Syndrome

Whistling Face Syndrome

Sheldon-Hall Syndrome

Freeman-Sheldon Syndrome Variant

Study ID

NCT05419245

Start date

Jun 1, 2022

Status verified date

Jul, 2022

Completion date

Dec, 2023

Anticipated

Primary completion date

Dec, 2023

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Patients who are able to provide informed consent or have a legally appointed representative who is able to provide informed consent
  • Patients who have Freeman-Burian syndrome (formerly, Freeman-Sheldon syndrome), Sheldon-Hall syndrome, distal arthrogryposis type 1, or distal arthrogryposis type 3
  • Persons who speak and read English, German, Russian, or Czech

Exclusion Criteria:

  • Patients who are not able to provide informed consent or do not have a legally appointed representative who is able to provide informed consent
  • Patients who do not have Freeman-Burian syndrome (formerly, Freeman-Sheldon syndrome), Sheldon-Hall syndrome, distal arthrogryposis type 1, or distal arthrogryposis type 3
  • Persons who do not speak or read English, German, Russian, or Czech

Study Design

Enrollment

20 participants

Anticipated

Interventions and Outcome Measures

Arms

Freeman-Sheldon syndrome Classic Type

Patients who have all features required by the Stevenson criteria, including: very small mouth (microstomia); whistling-face appearance (pursed lips); "H" or "V" shaped chin dimple; very obvious down-slanting crease from the nostril to the corners of the mouth (nasolabial creases); and restricted movement in joints (contractures) of two or more body areas, often hands and feet, with fingers and toes frequently overlapping.

Freeman-Sheldon syndrome Craniofacial Type

Patients who have only the face and skull physical findings required by the Stevenson criteria, including: very small mouth (microstomia), whistling-face appearance (pursed lips), "H" or "V" shaped chin dimple, very obvious down-slanting crease from the nostril to the corners of the mouth (nasolabial creases).

Freeman-Sheldon syndrome Mixed Type

Patients who have the face and skull physical findings required by the Stevenson criteria and some but not all required joint problems.

Sheldon-Hall syndrome

Patients who have all features required by the Stevenson criteria, including: small mouth (not microstomia); neck webbing (pterygium colli); small but prominent chin; very obvious down-slanting crease from the nostril to the corners of the mouth (nasolabial creases); and restricted movement in joints (contractures) of two or more body areas, often hands and feet, with fingers and toes frequently overlapping.

Distal Arthrogryposis Type 1

Patients with features consistent with this diagnosis, including restricted movement in joints (contractures) of two or more body areas, often hands and feet, with fingers and toes frequently overlapping.

Distal Arthrogryposis Type 3

Patients with features consistent with this diagnosis, including: gap in the roof of the mouth (cleft palate); drooping eyelid (blepharoptosis); and spine curve problems; and restricted movement in joints (contractures) of two or more body areas, often hands and feet, with fingers and toes frequently overlapping.

Interventions

Guided Health History for Freeman-Burian Syndrome Questionnaire

Completed during the interview. The Guided Health History for Freeman-Burian Syndrome Questionnaire is a guided interview form that will be used to assess diagnosis, problems, treatments, and outcomes.

Freeman-Burian syndrome Semi-Structured Quality of Life Interview

Completed during the interview. Freeman-Burian syndrome Semi-Structured Quality of Life Interview is a quality of life interview for Freeman-Burian syndrome, taking into consideration individual's total health.

Medical Records Review

Review of medical records will be used, along with Guided Health History for Freeman-Burian Syndrome Questionnaire to assess patient histories and outcomes.

PTSD Checklist for DSM-5

Completed by patients before the interview. The PTSD Checklist for DSM-5 is a 20-item questionnaire. Each item is rated 0-4, with 0 being "Not at all" and 4 being "Extremely". Lower scores suggest the person may be experiencing fewer symptoms.

Modified Flanagan Quality of Life Scale

Completed by patients before the interview. The Modified Flanagan Quality of Life Scale is a 16-item survey designed for use in persons with chronic illness. Each item is rated 1-7, with 1 being "Terrible" and 7 being "Delighted". Higher scores suggest the person may be experiencing a better quality of life.

Center for Epidemiologic Studies Depression Scale

Completed by patients before the interview. The Center for Epidemiologic Studies Depression Scale is a 20-item survey that asks about depressive feelings and behaviours in the past week. Each item is rated 0-3, with 0 being "Rarely or none of the time (less than 1 day)" and 3 being "Most or all of the time (5-7 days)". Lower scores suggest the person may be experiencing fewer symptoms.

Review of Systems

Completed during the interview. A review of systems is a checklist of medical problems.

FACE-Q | Craniofacial - Appearance of the Face

Completed by patients. The FACE-Q \| Craniofacial - Appearance of the Face is a 9-item questionnaire. Each item is rated 1-4, with 1 being "Not at All" and 4 being "Very Much". Lower scores suggest the person may be less pleased with the appearance of their face.

FACE-Q | Craniofacial - Appearance Distress

Completed by patients. The FACE-Q \| Craniofacial - Appearance Distress is an 8-item questionnaire. Each item is rated 1-4, with 1 being "Always" and 4 being "Never". Lower scores suggest the person may be experiencing more distress related to the appearance of their face.

FACE-Q | Craniofacial - Facial Function

Completed by patients. The FACE-Q \| Craniofacial - Facial Function is a 10-item questionnaire. Each item is rated 1-3, with 1 being "I cannot do this" and 3 being "I can do this". Lower scores suggest the person may be experiencing more problems with facial muscle functioning.

Primary outcome measure

  • Difference in intervention-related outcome rates between groups [ Time Frame: During a single study interview, which lasts 1-2 hours ]
  • Difference in diagnostic accuracy between groups [ Time Frame: During a single study interview, which lasts 1-2 hours ]

Central Contacts and Locations

Central contacts

Locations

Office of Craig R Dufresne, MD, PC

Recruiting

Fairfax, Virginia, United States, 22031

Contacts

Principal Investigator:

Craig R Dufresne, MD

More Information

Sponsor

Dufresne, Craig, MD, PC

Last update posted

Jul 19, 2022

Last verified

Jul, 2022

Keywords

  • craniofacial abnormalities
  • arthrogryposis
  • rehabilitation
  • surgery
  • therapy
  • health status
  • quality of life
  • patient reported outcome
  • health-related quality of life
  • depressive symptoms

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Dufresne, Craig, MD, PC on 2022-07-19.