ASSESSING EMERALD AND MC10 BIOSTAMP nPOINT BIOSENSORS FOR RETT SYNDROME

• ClinicalTrials.gov Identifier: NCT04514549
• Recruitment Status: Completed
• First Posted: August 17, 2020
• Last Update Posted: March 28, 2023
• Sponsor: Rett Syndrome Research Trust
• Information provided by (Responsible Party): Rett Syndrome Research Trust

Study Description

Brief Summary:

This is a pilot study of the Emerald device in Rett syndrome patients diagnosed with a confirmed MECP2 mutation. MC10 BioStamp nPoint patches will also be assessed with the goal to develop Rett-specific breathing algorithms

Condition or disease	Intervention/treatment
Rett Syndrome	Device: Emerald

Detailed Description:

The study consists of two sequential cohorts with a total of approximately 20 patients enrolled. Patients in each cohort may participate for up to 4 weeks. Each participant will have up to 2 Emerald devices installed in their home for monitoring sleep, breathing and movement, and will use between 3 and 9 nPoint patches for determining proper patch placement for detecting breathing signals.

The study will consist of a Screening visit, an Observation period, and a Follow-up phone call. The Screening Period will be one day in clinic, the Observation period will be up to 4 weeks at home and the Follow up phone call will be performed at the completion of the Observation Period.

Study Design

• Study Type: Observational
• Actual Enrollment: 21 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Assessing Emerald and MC10 nPoint Biosensors for Rett Syndrome
• Actual Study Start Date: November 23, 2020
• Actual Primary Completion Date: March 1, 2023
• Actual Study Completion Date: March 1, 2023

Groups and Cohorts

Group/Cohort	Intervention/treatment
Cohort 1; will enroll approximately 5 patients with pronounced respiratory dysfunction. Patients may be enrolled with tremors and or seizures. All patients will be observed via Emerald to capture sleep staging, movement and breathing for up to approximately 4 weeks. Caregivers will keep a daily questionnaire diary of the patient's anxiety, sleep and seizures. MC10 nPoint data will be captured for at least two 24-hour periods in each of the 4 weeks to assess patch placements for breathing detection. Ongoing assessment of MC10 nPoint data may lead to the use of more or less sensors, changes in duration of sensor data collection, or placement as Cohort 1 progresses.Preliminary results from Cohort 1 will determine if Emerald will continue to be evaluated and will inform on MC10 nPoint optimizations for Cohort 2. If preliminary results indicate Emerald is not an effective device, then Emerald will be discontinued	Device: Emerald; The Emerald is a wireless sensor that can track the motion, breathing, and sleep of subjects without touching or requiring any interaction with the subjects, allowing them to go about their normal lives. The physical device is roughly a 30 x 35 x 5 cm box that contains directional antennas and a motherboard to process these signals. In typical operation, the device is mounted onto a wall within a clinic or home setting using peel-away (reversible) mount strips.; Other Name: MC 10
Cohort 2; will enroll approximately 15 patients. Patients with pronounced respiratory dysfunction, tremors, seizures, and/or other expanded features of Rett syndrome deemed appropriate may be enrolled. If Emerald is continued, all patients are observed via Emerald to capture sleep staging, movement and breathing up to for approximately 4 weeks. Caregivers will keep a daily questionnaire diary of the patient's anxiety, sleep and seizures. Ongoing assessment of MC10 nPoint data may lead to the use of more or less sensors, changes in duration of sensor data collection, or placement as Cohort 2 progresses.

Outcome Measures

Primary Outcome Measures :

1. Emerald respiratory function in Rett patients [ Time Frame: 4 weeks ]
   Suitability of Emerald technology to assess sleep staging and

Secondary Outcome Measures :

1. MC10 [ Time Frame: 8 weeks ]
   Determination of MC10 BioStamp nPoint patch placement for detecting breathing

Eligibility Criteria

• Ages Eligible for Study: Child, Adult, Older Adult
• Sexes Eligible for Study: Female
• Gender Based Eligibility: Yes
• Gender Eligibility Description: Female
• Accepts Healthy Volunteers: No
• Sampling Method: Probability Sample

Study Population

Female patients with Diagnosis of typical Rett Syndrome according to the revised Clinical Diagnostic Criteria and presence of a disease-causing MECP2 genetic mutation.

Criteria

Inclusion Criteria:

1. Prior to the conduct of any study-specific procedures, the parent/caregiver/LAR must provide written informed consent. If the caregiver attending the clinic visits is not the parent or LAR, written consent must be obtained from the parent or LAR for the caregiver's participation in the study.
2. Diagnosis of typical Rett Syndrome according to the revised Clinical Diagnostic Criteria1 and presence of a disease causing MECP2 genetic mutation.
3. The patient's parent/caregiver/LAR must be able to understand the nature of the study and to allow for the completion of study assessments. The same parent/caregiver/LAR must be capable of providing reliable information about the patient's condition.
4. Live within approximately 50 to 60 miles of MIT.
5. Primary language English.
6. Approximately 4 people or less living in the home. This does not include visiting caregivers.
7. Must have home access to Wi-Fi.

Exclusion Criteria:

1. Inability of patient to sleep alone in their own room.
2. Pets in the home, or caregivers unwilling to keep pets out of the bedroom monitored by Emerald during the Observation Period.
3. Plans to take a prolonged vacation or to otherwise be out of the home during the Observation Period.
4. Participation in another device study that could interfere with this study.
5. Active implantable devices such as pacemakers or defibrillators.
6. Known allergies or hypersensitivities to adhesives.
7. Patients with any condition that, in the opinion of the principal investigator, might interfere with the conduct of the study, confound interpretation of the study results, endanger their own well-being, or who may otherwise not be suitable for the study.

Contacts and Locations

Locations

United States, Massachusetts

Boston Children's Hospital

Boston, Massachusetts, United States, 02115

Sponsors and Collaborators

Rett Syndrome Research Trust

Investigators

• Principal Investigator: David Lieberman, MD; Boston Children's Hospital

More Information

• Responsible Party: Rett Syndrome Research Trust
• ClinicalTrials.gov Identifier: NCT04514549
• Other Study ID Numbers: SENS-101-RSRT
• First Posted: August 17, 2020
• Last Update Posted: March 28, 2023
• Last Verified: March 2023

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Rett Syndrome; Syndrome; Disease; Pathologic Processes; Mental Retardation, X-Linked; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Heredodegenerative Disorders, Nervous System