Autologous Muscle Derived Cells for Treatment of Tongue Dysphagia

NCT05421689 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: 1800521
• Study Type: interventional
• Target: 66
• Locations: 1 country
• Sites: 2

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.

Conditions

Oropharyngeal Dysphagia

Outcome Measures

Primary Outcomes (2)

• Study product-related, biopsy procedure-related, and injection procedure-related adverse events.

  Safety will be determined by the frequency and severity of adverse events related to study procedures and study product.

  24 months

• Anterior tongue pressure measured from Iowa Oral Performance Instrument (IOPI)

  Efficacy of AMDC-GIR in the improvement of objective Anterior Tongue Pressure Measurement (IOPI)

  24 months

Secondary Outcomes (5)

• Incidence of patient aspiration pneumonia

  24 months

• Incidence of patient survival

  24 months

• Penetration Aspiration scale rating following swallowing fluoroscopy

  24 months

• Peak Pharyngeal pressure measurement from high-resolution manometry

  24 months

• Patient-reported dysphagia symptoms based on Eating Assessment Tool EAT10 score

  24 months

Other Outcomes (6)

• Pharyngeal Constriction Ratio measurement for swallowing fluoroscopy

  24 months

• Upper Esophageal Sphincter opening measurement for swallowing fluoroscopy

  24 months

• Pharyngeal transit time measurement for swallowing fluoroscopy

  24 months

Study Arms (2)

Experimental: 150 x 10⁶ AMDC-GIR dosage

EXPERIMENTAL

33 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)

Experimental: Identical Placebo composed of the same cryopreservation medium used for AMDC-GIR

SHAM COMPARATOR

33 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

Interventions

Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR) BIOLOGICAL

The study will treat 66 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.

Also known as: Autologous muscle derived stem cells

Experimental: 150 x 10⁶ AMDC-GIR dosage

Placebo OTHER

two doses of placebo will be administered and spaced 4-6 weeks apart.

Also known as: identical placebo composed of the same cryopreservation medium used for AMDC-GIR

Experimental: Identical Placebo composed of the same cryopreservation medium used for AMDC-GIR

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.
• Patient has failed to achieve resolution of symptoms following contemporary therapies.

You may not qualify if:

• 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.
• Previously treated with an investigational device, drug, or procedure for TD within 6 months prior to signing consent.
• TD of neurogenic etiology or uncorrected congenital abnormality leading to TD.
• Neuromuscular disorder (e.g., Parkinson's disease, muscular dystrophy, multiple sclerosis) that could lead to TD.
• Severe fibrosis at injection site.
• Uncontrolled diabetes.
• Compromised immune system due to disease state, chronic corticosteroid use, or other immunosuppressive therapy.
• 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).
• History of bleeding diathesis or uncorrectable coagulopathy.
• Any non-skin cancer that has necessitated treatment within the past 24 months.
• Patient's Current Status-based Criteria:
• Evidence or known high risk of recurrent or persistent cancer as determined by the physician during screening.
• 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.
• Cannot, or is not willing to maintain the current treatment regimen for existing contemporary therapy (e.g., swallowing therapy).

Sponsors & Collaborators

• California Institute for Regenerative Medicine (CIRM): collaborator
• University of California, Davis: lead
• Cook MyoSite: collaborator

Study Sites (2)

UC Davis Medical Center, Department of Otolaryngology

Sacramento, California, 95817, United States

Location

UC San Francisco Medical Center, Voice and Swallow Center

San Francisco, California, 94115, United States

Location

MeSH Terms

Conditions

Deglutition Disorders

Condition Hierarchy (Ancestors)

Esophageal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Pharyngeal Diseases; Otorhinolaryngologic Diseases

Study Officials

• Peter Belafsky, MD

  University of California Davis, Department of Otolaryngology

  PRINCIPAL INVESTIGATOR

• Maggie Kuhn, MD

  University of California Davis, Department of Otolaryngology

  PRINCIPAL INVESTIGATOR

• Johnathon D Anderson, PhD

  University of California Davis, Department of Otolaryngology

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: 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.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Double-blind, randomized, placebo-controlled, multicenter clinical trial

Study Record Dates

• First Submitted: June 6, 2022
• First Posted: June 16, 2022
• Study Start: May 26, 2022
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: December 12, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)