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Autologous Muscle Derived Cells for Treatment of Tongue Dysphagia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05421689
Recruitment Status : Recruiting
First Posted : June 16, 2022
Last Update Posted : June 16, 2022
Sponsor:
Collaborators:
California Institute for Regenerative Medicine (CIRM)
Cook MyoSite
Information provided by (Responsible Party):
University of California, Davis

Brief Summary:
The primary objective of this double-blind, randomized, placebo-controlled, multicenter clinical trial is to evaluate the safety of AMDC-GIR during the 24 months following 2 consecutive treatments of tongue dysphagia in male and female patients who have undergone surgery and/or chemo- and/or radiotherapy for squamous cell cancer of the oropharynx.

Condition or disease Intervention/treatment Phase
Oropharyngeal Dysphagia Biological: Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR) Other: Placebo Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 62 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Double-blind, randomized, placebo-controlled, multicenter clinical trial
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Patients will receive two treatments of intramuscular injection of 1 AMDC-GIR dose of 150 x 10⁶ cells or identical placebo. Both patients and study investigators at all sites will be blinded to the treatment assignment of patients.
Primary Purpose: Other
Official Title: A Multi-Center, Double-Blind, Randomized Placebo-Controlled Investigation of Autologous Muscle Derived Cells (AMDC) for the Treatment of Tongue Dysphagia Resulting From the Treatment of Head and Neck Cancer
Actual Study Start Date : May 26, 2022
Estimated Primary Completion Date : January 31, 2026
Estimated Study Completion Date : January 31, 2026

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Experimental: 150 x 10⁶ AMDC-GIR dosage
31 subjects will be receiving two doses of 150 x 10⁶ AMDC-GIR spaced 4-6 weeks apart.
Biological: Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR)
The study will treat 62 patients at 2 clinical sites: UC Davis Center for Voice and Swallowing and UCSF Voice and Swallowing Center. Patients will be randomized 1:1 to receive either 2 AMDC-GIR doses of 150 x 10⁶ cells or 2 doses of identical placebo composed of the same cryopreservation medium used for AMDC-GIR. Enrollment is expected to be completed within 2 years of initiating the study. Patients will be followed for 24 months post-treatment.
Other Name: Autologous muscle derived stem cells

Sham Comparator: Experimental: Identical Placebo composed of the same cryopreservation medium used for AMDC-GIR
31 subjects will be receiving two doses of identical placebo composed of the same cryopreservation medium used for AMDC-GIR. Doses will be spaced 4-6 weeks apart.
Other: Placebo
two doses of placebo will be administered and spaced 4-6 weeks apart.
Other Name: identical placebo composed of the same cryopreservation medium used for AMDC-GIR




Primary Outcome Measures :
  1. Study product-related, biopsy procedure-related, and injection procedure-related adverse events. [ Time Frame: 24 months ]
    Safety will be determined by the frequency and severity of adverse events related to study procedures and study product.

  2. Anterior tongue pressure measured from Iowa Oral Performance Instrument (IOPI) [ Time Frame: 24 months ]
    Efficacy of AMDC-GIR in the improvement of objective Anterior Tongue Pressure Measurement (IOPI)


Secondary Outcome Measures :
  1. Incidence of patient aspiration pneumonia [ Time Frame: 24 months ]
    Effects of AMDC-GIR on incidence of patient reported aspiration pneumonia (yes/no scoring criteria)

  2. Incidence of patient survival [ Time Frame: 24 months ]
    Effects of AMDC-GIR on incidence of patient reported survival (yes/no scoring criteria)

  3. Penetration Aspiration scale rating following swallowing fluoroscopy [ Time Frame: 24 months ]

    The 8-point Penetration-Aspiration Scale (scores of 1-8, 1=better, 8=worse, see table below) is the standard method used measure the severity of airway invasion during swallowing.

    1. Material does not enter the airway
    2. Material enters the airway, remains above the vocal folds, and is ejected from the airway
    3. Material enters the airway, remains above the vocal folds, and is not ejected from the airway
    4. Material enters the airway, contacts the vocal folds, and is ejected from the airway
    5. Material enters the airway, contacts the vocal folds, and is not ejected from the airway
    6. Material enters the airway, passes below the vocal folds, and is ejected into the larynx or out of the airway
    7. Material enters the airway, passes below the vocal folds, and is not ejected from the trachea despite effort
    8. Material enters the airway, passes below the vocal folds, and no effort is made to eject

  4. Peak Pharyngeal pressure measurement from high-resolution manometry [ Time Frame: 24 months ]
    Efficacy of AMDC-GIR in the improvement of objective manometric swallowing parameters

  5. Patient-reported dysphagia symptoms based on Eating Assessment Tool EAT10 score [ Time Frame: 24 months ]

    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


Other Outcome Measures:
  1. Pharyngeal Constriction Ratio measurement for swallowing fluoroscopy [ Time Frame: 24 months ]
    Efficacy of AMDC-GIR in the improvement of objective fluoroscopic swallowing parameters

  2. Upper Esophageal Sphincter opening measurement for swallowing fluoroscopy [ Time Frame: 24 months ]
    Efficacy of AMDC-GIR in the improvement of objective fluoroscopic swallowing parameters

  3. Pharyngeal transit time measurement for swallowing fluoroscopy [ Time Frame: 24 months ]
    Efficacy of AMDC-GIR in the improvement of objective fluoroscopic swallowing parameters

  4. Patient-reported survey of smell and taste [ Time Frame: 24 months ]
    Effects of AMDC-GIR on patient reported smell and taste as reported by the 8-question survey ChemoSensory Questionnaire (CCQ). Minimum score for each scale, 4 Maximum score for each scale, 20. High score indicates better function

  5. Clinical assessment of vocal quality (CAPE-V) [ Time Frame: 24 months ]
    Effects of AMDC-GIR in the improvement of objective voice quality. The CAPE-V approach uses a form where Overall Severity, Roughness, Breathiness, Strain, Pitch, and Loudness are rated using a 100-mm visual analog scale. The rater places a vertical mark along each horizontal line where the far left end of the line represents the least impaired status and the far right represents the one most impaired. Scores are obtained by making measurements to the nearest millimeter from the left most (least impaired) end of the horizontal line. The higher the score the worse the impairment.

  6. Patient-reported Quality of Life based on FACT-HN score [ Time Frame: 24 months ]
    Effects of AMDC-GIR on patient reported quality of life. FACT-HN is a 39-question survey, with a score range from 0-148. The higher the score the better the quality of life for the patient.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Male or Female, at least 18 years old, with primary symptoms of TD following surgery and/or chemo- and/or radiotherapy for treatment of squamous cell carcinoma for oropharyngeal cancer. Treatment must be completed at least 24 months prior to enrollment, with TD and disease-free status confirmed by medical history, clinical symptoms, a focused head and neck examination, swallowing fluoroscopy, and high-resolution pharyngeal manometry.
  2. TD severity should be moderate as defined by a Functional Oral Intake Scale (FOIS, provided in Appendix C). Individuals must have a FOIS of 3 or better and EAT-10 score of greater than 5.
  3. Patient has failed to achieve resolution of symptoms following contemporary therapies.

Exclusion Criteria:

  1. Simultaneously participating in another investigational drug or device study or has completed the follow-up phase for the primary endpoint of any previous study less than 30 days prior to the first evaluation in this study.
  2. Previously treated with an investigational device, drug, or procedure for TD within 6 months prior to signing consent.
  3. TD of neurogenic etiology or uncorrected congenital abnormality leading to TD.
  4. Neuromuscular disorder (e.g., Parkinson's disease, muscular dystrophy, multiple sclerosis) that could lead to TD.
  5. Severe fibrosis at injection site.
  6. Uncontrolled diabetes.
  7. Compromised immune system due to disease state, chronic corticosteroid use, or other immunosuppressive therapy.
  8. Medical condition or disorder that may limit life expectancy or that may cause CIP deviations (e.g., unable to perform self-evaluations or accurately report medical history, symptoms, or data).
  9. History of bleeding diathesis or uncorrectable coagulopathy.
  10. Known allergy or hypersensitivity to bovine proteins or allergens, gentamicin sulfate, or ampicillin that medically warrants exclusion as determined by the physician.
  11. Any non-skin cancer that has necessitated treatment within the past 24 months.

Patient's Current Status-based Criteria:

  1. Evidence or known high risk of recurrent or persistent cancer as determined by the physician during screening.
  2. Tests positive for Hepatitis B (required tests: Hepatitis B Surface Antigen [HBsAg] and Anti-Hepatitis B Core Antibody [Anti-HBc]), Hepatitis C (required test: Hepatitis C Antibody [Anti-HCV]), HIV (required tests: HIV Type 1 and 2 Antibodies [Anti-HIV-1, 2]), and/or Syphilis.

    a. Tests performed by certified/authorized testing laboratory using licensed/approved tests and performed on blood samples collected within 30 days prior to muscle tissue procurement.

  3. Cannot, or is not willing to maintain the current treatment regimen for existing contemporary therapy (e.g., swallowing therapy).
  4. Requires prophylactic antibiotics for chronic infection or has required 2 or more courses of antibiotics for infections in the 2 months prior to signing consent.
  5. Any condition, including current infection or immunodeficiency, which could lead to significant postoperative complications.
  6. Refuses or cannot provide written informed consent.
  7. Not available for, or willing to comply with the baseline and follow-up evaluations as required by the CIP.
  8. Pregnant, lactating, or plans to become pregnant during the course of the study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05421689


Contacts
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Contact: Johnathon D Anderson, PhD 916-703-9300 joanderson@ucdavis.edu
Contact: Randev S Sandhu, BS 916-734-2863 rssandhu@ucdavis.edu

Locations
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United States, California
UC Davis Medical Center, Department of Otolaryngology Recruiting
Sacramento, California, United States, 95817
Contact: Johnathon D Anderson, PhD    916-703-9300    joanderson@ucdavis.edu   
Contact: Randev S Sandhu, BS    916-734-2863    rssandhu@ucdavis.edu   
Principal Investigator: Peter Belafsky, MD         
Sub-Investigator: Maggie Kuhn, MD         
Sub-Investigator: Daniel Cates, MD         
UC San Francisco Medical Center, Voice and Swallow Center Not yet recruiting
San Francisco, California, United States, 94115
Contact: Clark Rosen, MD    415-885-7700    Clark.Rosen@ucsf.edu   
Contact: Grant Gochman, BS    415-476-2753    grant.gochman@ucsf.edu   
Sponsors and Collaborators
University of California, Davis
California Institute for Regenerative Medicine (CIRM)
Cook MyoSite
Investigators
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Principal Investigator: Peter Belafsky, MD University of California Davis, Department of Otolaryngology
Principal Investigator: Maggie Kuhn, MD University of California Davis, Department of Otolaryngology
Study Director: Johnathon D Anderson, PhD University of California Davis, Department of Otolaryngology
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Responsible Party: University of California, Davis
ClinicalTrials.gov Identifier: NCT05421689    
Other Study ID Numbers: 1800521
1800521 ( Other Identifier: UC DAVIS )
First Posted: June 16, 2022    Key Record Dates
Last Update Posted: June 16, 2022
Last Verified: June 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Deglutition Disorders
Esophageal Diseases
Gastrointestinal Diseases
Digestive System Diseases
Pharyngeal Diseases
Otorhinolaryngologic Diseases