Oropharyngeal Dysphagia
Conditions
Keywords
Tongue, Dysphagia, Autologous
Brief summary
The main purpose of this registry is to collect observational, long-term safety and effectiveness data in participants who have received iltamiocel as part of the blinded portion of the REVIVE clinical study.
Detailed description
This is an observational registry with a non-experimental cohort design that will provide long-term safety and effectiveness data from participants who participated in the REVIVE clinical study. Participants will be enrolled by either of the two participating sites. All visits will be conducted either remotely or in person. The objectives of this study are to: * Evaluate the long-term safety of iltamiocel * Evaluate the long-term effectiveness of iltamiocel treatment as demonstrated through anterior tongue strength and patient reported outcomes (PROs) related to tongue dysphagia (TD) symptoms, quality of life (QOL), and overall satisfaction. The duration of participants' participation in this registry will extend to 5 years after enrollment. This is an observational registry with a non-experimental cohort design that will provide long-term safety and effectiveness data from participants who participated in the REVIVE clinical study. Participants will be enrolled by either of the two participating sites. All visits will be conducted either remotely or in person.
Interventions
OTHEROther: No Intervention (subjects were previously treated with Iltamiocel). This is a observational study and all participants will be observed with no intervention.
Participants are followed observationally to collect long-term safety and effectiveness data after prior receipt of iltamiocel in the separate Revive clinical trial; no intervention is administered as part of this registry. Long-term safety outcomes and efficacy will be separated in analysis \& reporting
Sponsors
University of California, Davis
Cook MyoSite
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Participants who have received iltamiocel as part of participation in the REVIVE clinical study for males and females with tongue dysphagia (TD) are eligible, provided the following criteria are fulfilled: * Has completed the Month 24 visit in the REVIVE study. * Has received iltamiocel as part of the blinded portion of the REVIVE study. * Must be willing and able to comply with the study procedures, is able to understand all study requirements and must agree to read and sign the informed consent form prior to any study-related procedures.
Exclusion criteria
* The following criteria will exclude participants from participation: * Has received placebo as part of the blinded portion of the REVIVE study. * Unable or unwilling to provide informed consent. * Not available for, or willing to comply with the follow-up evaluations as required by the protocol.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Iltamiocel-Related Adverse Events and Serious Adverse Events | From baseline through 5 years of follow-up, assessed annually | Iltamiocel-related adverse events (AEs) and iltamiocel-related serious adverse events (SAEs) assessed by the investigator. Long-term safety outcomes and efficacy outcomes will be separated in analysis and reporting. |
| Change From Baseline in Anterior Tongue Pressure as Measured by Iowa Oral Performance Instrument (IOPI) | Baseline and annually up to 5 years | Anterior tongue pressure measured using the Iowa Oral Performance Instrument (IOPI). Range 0-100 kPa, where a lower number indicates less strength. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Dysphagia Symptoms as Measured by Eating Assessment Tool (EAT-10) | Baseline and annually up to 5 years | Patient-reported dysphagia symptoms assessed using the Eating Assessment Tool (EAT-10) total score. Eating Assessment Tool (EAT-10), range 0-40, where higher scores indicate worse dysphagia symptoms. Survey consisting of 10 questions (see table below), each score on a scale of 0-4, with 0= no problem, 4= severe problem. 922 Belafsky et al. Eating Assessment Tool TABLE 3. EATING ASSESSMENT TOOL (EAT-10) Circle the appropriate response. To what extent are the following scenarios problematic for you? 1. My swallowing problem has caused me to lose weight. 2. My swallowing problem interferes with my ability to go out for meals. 3. Swallowing liquids takes extra effort. 4. Swallowing solids takes extra effort. 5. Swallowing pills takes extra effort. 6. Swallowing is painful. 7. The pleasure of eating is affected by my swallowing. 8. When I swallow food sticks in my throat. 9. I cough when I eat. 10. Swallowing is stressful |
| Change From Baseline in Smell and Taste Function as Measured by ChemoSensory Smell and Taste Questionnaire (CSQ) | Baseline and annually up to 5 years | Patient-reported smell and taste function assessed using the ChemoSensory Smell and Taste Questionnaire (CSQ). Consists of 8 items divided into two subscales: 4 items for smell and 4 items for taste and each with a range of 1 to 5, where 1 is never, 2 is Rarely, 3 is Sometimes, 4 is Frequently and 5 is Always. Each scale ranges from 4 to 20, where higher scores indicate better chemosensory function. |
| Change From Baseline in Functional Oral Intake as Measured by Functional Oral Intake Scale (FOIS) | Baseline and annually up to 5 years | Functional oral intake assessed using the Functional Oral Intake Scale (FOIS). It is a 7-level ordinal scale used to document the functional level of oral food and liquid intake in patients with dysphagia. It ranges from Level 1 (no oral intake/tube-dependent) to Level 7 (full oral intake without restrictions), where the higher the score the less oral intake restrictions. |
| Change From Baseline in Dysphagia-Related Quality of Life as Measured by MD Anderson Dysphagia Inventory (MDADI) | Baseline and annually up to 5 years | Dysphagia-related quality of life assessed using the MD Anderson Dysphagia Inventory (MDADI). It is a 20-item questionnaire that measures swallowing-related quality of life, scoring from 20 to 100. Higher scores indicate better functioning, with 20 representing the lowest and 100 the highest. |
Countries
United States
Contacts
CONTACTRandev Sandhu, BS
CONTACTJohnathon Anderson, PhD
PRINCIPAL_INVESTIGATORPeter Belafsky, MD
University of California, Davis
Outcome results
None listed