ASSESSING EMERALD AND MC10 BIOSTAMP nPOINT BIOSENSORS FOR RETT SYNDROME

Assessing Emerald and MC10 nPoint Biosensors for Rett Syndrome

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

Study Overview

• Status: Completed
• Conditions: Rett Syndrome
• Intervention / Treatment: 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 Type: Observational
• Enrollment (Actual): 21

Contacts and Locations

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• 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.

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

2

Cohorts and Interventions

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 Names: 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Emerald respiratory function in Rett patients	Suitability of Emerald technology to assess sleep staging and	4 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
MC10	Determination of MC10 BioStamp nPoint patch placement for detecting breathing	8 weeks

Collaborators and Investigators

• Sponsor: Rett Syndrome Research Trust
• Investigators: Principal Investigator: David Lieberman, MD, Boston Children's Hospital

Study record dates

Study Major Dates

• Study Start (Actual): November 23, 2020
• Primary Completion (Actual): March 1, 2023
• Study Completion (Actual): March 1, 2023

Study Registration Dates

• First Submitted: August 12, 2020
• First Submitted That Met QC Criteria: August 12, 2020
• First Posted (Actual): August 17, 2020

Study Record Updates

• Last Update Posted (Estimated): November 14, 2024
• Last Update Submitted That Met QC Criteria: November 12, 2024
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neurologic Manifestations; Nervous System Diseases; Pathologic Processes; Genetic Diseases, Inborn; Disease; Neurobehavioral Manifestations; Heredodegenerative Disorders, Nervous System; Mental Retardation, X-Linked; Intellectual Disability; Genetic Diseases, X-Linked; Syndrome; Rett Syndrome
• Other Study ID Numbers: SENS-101-RSRT

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No