Oropharyngeal DysphagiaPhase 1NCT02838316
Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR) for Tongue Dysphagia
A Phase 1 study testing Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR) in people with Oropharyngeal Dysphagia, run by Peter Belafsky, MD at 1 site in United States.
Trial summary
The primary objective of this study is to evaluate the safety of Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR) during the 12 months following treatment of tongue dysphagia in male and female patients who have undergone surgery and/or chemo- and/or radiotherapy for squamous cell cancer of the… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Oropharyngeal Dysphagia
- What is being tested?
- Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- 24 May 2017to Dec 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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 and clinical symptoms, including a focused head and neck examination evaluation, 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.
- Patient has failed to achieve acceptable resolution of symptoms following conservative therapies.
Who cannot join
- Patient History-based Criteria:
- 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.
- Has ever been treated with a cell therapy for TD.
- Symptoms of aspiration pneumonia prior to enrollment.
- TD of neurogenic etiology or uncorrected congenital abnormality leading to TD.
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- Neuromuscular disorder (e.g., Parkinson's disease, muscular dystrophy, multiple sclerosis) that could lead to TD.
- Moderate or severe fibrosis at likely injection site.
- Morbidly obese (BMI ≥ 35).
- 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 clinical investigation plan (CIP) deviations (e.g., unable to perform self-evaluations or accurately report medical history, symptoms, or data).
- History of bleeding diathesis or uncorrectable coagulopathy.
- Known allergy or hypersensitivity to bovine proteins or allergens, gentamicin sulfate, or ampicillin that medically warrants exclusion as determined by the physician.
- 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.
- 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 conservative therapy (e.g., swallowing therapy).
- Requires prophylactic antibiotics for chronic infections, or has required 2 or more courses of antibiotics for infections in the 2 months prior to signing consent.
- Any condition, including current infection, which could lead to significant postoperative complications.
- Refuses to provide written informed consent.
- Not available for, or willing to comply, with the baseline and follow-up evaluations as required by the CIP.
- Pregnant, lactating, or plans to become pregnant during the course of the study.
Trial locations and contacts
1 site in 1 country.
United States1 site
UC Davis Medical Center, Department of Otolaryngology, Head and Neck Surgery
Sacramento, California, 95817
About this study
Official title: Muscle Cell Mediated Therapy for Tongue Dysphagia: An Investigation of Cook MyoSite Autologous Muscle Derived Cells
The primary objective of this study is to evaluate the safety of Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR) during the 12 months following treatment 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.
Read the detailed description
This preliminary, prospective, dose escalating clinical study will evaluate the safety and potential efficacy of Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR) for the treatment of tongue dysphagia (TD) that develops following treatment for head and neck cancer.
Surgery, chemo- and radiotherapy can induce significant TD resulting in long-term TD. Therefore, augmenting tongue muscle function may be beneficial to patients. Autologous muscle cell therapy, which involves isolation of cells from skeletal muscle biopsies, ex vivo expansion, and subsequent injection into the tongue, may serve as a potential durable therapy. In animal studies, muscle derived cells have successfully integrated within tissue to improve tongue strength and function. Intramuscular injection of AMDC-GIR is expected to produce localized tissue changes near the injection site and is not expected to produce a systemic effect.
Patients will receive a single treatment intramuscular injection of 1 of 2 doses of AMDC-GIR. Patients will have quantitative and qualitative measures of dysphagia assessed before treatment and at various times after treatment.
The study will treat up to 20 patients at 1 clinical site. Enrollment is expected to be completed within 2 years of initiating the study. Patients will be followed for 24 months post-treatment. The first 3 patients at each dose must reach 1-month follow-up before subsequent patients can be treated.
Male and female patients at least 18 years of age who have undergone surgery and/or chemo- and or radiotherapy for primary treatment of oropharyngeal squamous cell cancer and who present with symptoms and findings of TD will be eligible for participation. Eligible patients will have muscle tissue harvested using a needle biopsy technique during an outpatient procedure. The harvested muscle tissue will be transported to the manufacturer for cell processing. The muscle derived cells (MDC) will be isolated and expanded in culture over several weeks.
After reaching the desired concentration, the isolated and expanded AMDC-GIR will be frozen and shipped back to the investigating physician. The physician will thaw the AMDC-GIR and dilute the sample with an approximately equal volume of physiological saline. Under direct vision, the resulting suspension will be injected into the patient's tongue in a brief outpatient procedure.
Patients will be assessed for improvement in TD symptoms at 3 months, 6 months, 12 months and 24 months following treatment. Adverse events will be assessed at those visits, as well as during follow-up calls at 1-2 days, 1 week, 15 months, 18 months and 21 months.
- Conditions
- Oropharyngeal Dysphagia
- Being tested
- Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR)
- Medical subject headings
- Deglutition Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR)Drug
Autologous muscle derived stem cells
| Study group | Role | What they receive |
|---|---|---|
| 150 x 106 dosage | Experimental | 10 subjects will be receiving a dosage of 150 x 106 AMDC-GIR Receives: Drug: Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR) |
| 300 x 106 dosage | Experimental | 10 subjects will be receiving a dosage of 300 x 106 AMDC-GIR Receives: Drug: Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Study product-related Serious Adverse Events (SAEs)
Evaluate the safety of AMDC-GIR following treatment of tongue dysphagia
Measured: 24 months
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
Measured: 24 months
Secondary outcomes (9)
Penetration-Aspiration scale rating from swallowing fluoroscopy
Efficacy of AMDC-GIR in the improvement of objective fluoroscopic swallowing parameters
Measured: 24 months
Pharyngeal Constriction Ratio measurement from swallowing fluoroscopy
Efficacy of AMDC-GIR in the improvement of objective fluoroscopic swallowing parameters
Measured: 24 months
Upper Esophageal Sphincter opening measurement from swallowing fluoroscopy
Efficacy of AMDC-GIR in the improvement of objective fluoroscopic swallowing parameters
Measured: 24 months
Pharyngeal transit time measurement from swallowing fluoroscopy
Efficacy of AMDC-GIR in the improvement of objective fluoroscopic swallowing parameters
Measured: 24 months
Peak pharyngeal pressure measurement from high-resolution manometry
Efficacy of AMDC-GIR in the improvement of objective manometric swallowing parameters
Measured: 24 months
Anterior tongue pressure measurement from Iowa Oral Performance Instrument (IOPI)
Efficacy of AMDC-GIR in the improvement of objective Anterior Tongue Pressure Measurement (IOPI)
Measured: 24 months
Patient-reported dysphagia symptoms based on Eating Assessment Tool- EAT10 score
Effect of AMDC-GIR on patient-reported dysphagia symptoms [Eating Assessment Tool- EAT10]
Measured: 24 months
Patient-reported quality of life based on SF-12 survey score
Effect of AMDC-GIR on patient-reported dysphagia symptoms quality of life (QOL) [SF-12]
Measured: 24 months
Patient-reported voice symptoms based on Voice Handicap Index - VHI10 score
Effect of AMDC-GIR on patient-reported dysphagia symptoms and voice symptoms [Voice Handicap Index - VHI10]
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2016 (estimated)
- Study start24 May 2017
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Oropharyngeal DysphagiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- Phase I open label clinical trial
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Peter Belafsky, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Cook MyoSite
- Responsible party
- Sponsor-investigator, Peter Belafsky, MD, University of California, Davis
- Investigators
- Peter Belafsky, MD, PhD, Principal investigator, University of California Davis, Department of OtolaryngologyMaggie Kuhn, MD, Principal investigator, University of California Davis, Department of OtolaryngologyNogah Nativ, PhD, Study director, University of California Davis, Department of Otolaryngology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02838316
- Sponsor's protocol ID
- 801019
- Other IDs
- 801019-1
- Status last verified
- Feb 2026
- First submitted
- 12 Jul 2016
- Last updated
- 20 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Muscle Cell Mediated Therapy for Tongue Dysphagia: An Investigation of Cook MyoSite Autologous Muscle Derived Cells
- ClinicalTrials.gov ID
- NCT02838316
- Acronym / sponsor ID
- 801019
- Conditions
- Oropharyngeal Dysphagia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Peter Belafsky, MDUniversity, hospital or other
- Collaborators
- Cook MyoSite
- Study start
- 24 May 2017 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 20 Jul 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study is to evaluate the safety of Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR) during the 12 months following treatment 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.
Detailed description
This preliminary, prospective, dose escalating clinical study will evaluate the safety and potential efficacy of Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR) for the treatment of tongue dysphagia (TD) that develops following treatment for head and neck cancer.
Surgery, chemo- and radiotherapy can induce significant TD resulting in long-term TD. Therefore, augmenting tongue muscle function may be beneficial to patients. Autologous muscle cell therapy, which involves isolation of cells from skeletal muscle biopsies, ex vivo expansion, and subsequent injection into the tongue, may serve as a potential durable therapy. In animal studies, muscle derived cells have successfully integrated within tissue to improve tongue strength and function. Intramuscular injection of AMDC-GIR is expected to produce localized tissue changes near the injection site and is not expected to produce a systemic effect.
Patients will receive a single treatment intramuscular injection of 1 of 2 doses of AMDC-GIR. Patients will have quantitative and qualitative measures of dysphagia assessed before treatment and at various times after treatment.
The study will treat up to 20 patients at 1 clinical site. Enrollment is expected to be completed within 2 years of initiating the study. Patients will be followed for 24 months post-treatment. The first 3 patients at each dose must reach 1-month follow-up before subsequent patients can be treated.
Male and female patients at least 18 years of age who have undergone surgery and/or chemo- and or radiotherapy for primary treatment of oropharyngeal squamous cell cancer and who present with symptoms and findings of TD will be eligible for participation. Eligible patients will have muscle tissue harvested using a needle biopsy technique during an outpatient procedure. The harvested muscle tissue will be transported to the manufacturer for cell processing. The muscle derived cells (MDC) will be isolated and expanded in culture over several weeks.
After reaching the desired concentration, the isolated and expanded AMDC-GIR will be frozen and shipped back to the investigating physician. The physician will thaw the AMDC-GIR and dilute the sample with an approximately equal volume of physiological saline. Under direct vision, the resulting suspension will be injected into the patient's tongue in a brief outpatient procedure.
Patients will be assessed for improvement in TD symptoms at 3 months, 6 months, 12 months and 24 months following treatment. Adverse events will be assessed at those visits, as well as during follow-up calls at 1-2 days, 1 week, 15 months, 18 months and 21 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Phase I open label clinical trial
| Arm | Type | Receives |
|---|---|---|
| 150 x 106 dosage | Experimental | Drug: Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR)10 subjects will be receiving a dosage of 150 x 106 AMDC-GIR |
| 300 x 106 dosage | Experimental | Drug: Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR)10 subjects will be receiving a dosage of 300 x 106 AMDC-GIR |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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 and clinical symptoms, including a focused head and neck examination evaluation, 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.
- Patient has failed to achieve acceptable resolution of symptoms following conservative therapies.
Key exclusion criteria (25 listed)
- 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.
- Has ever been treated with a cell therapy for TD.
- Symptoms of aspiration pneumonia prior to enrollment.
- TD of neurogenic etiology or uncorrected congenital abnormality leading to TD.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR)Autologous muscle derived stem cells | Drug | 150 x 106 dosage, 300 x 106 dosage |
Primary outcome
Study product-related Serious Adverse Events (SAEs)
Time frame: 24 months
Evaluate the safety of AMDC-GIR following treatment of tongue dysphagia
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
9 secondary outcomes
Penetration-Aspiration scale rating from swallowing fluoroscopy
Time frame: 24 months
Pharyngeal Constriction Ratio measurement from swallowing fluoroscopy
Time frame: 24 months
Upper Esophageal Sphincter opening measurement from swallowing fluoroscopy
Time frame: 24 months
Pharyngeal transit time measurement from swallowing fluoroscopy
Time frame: 24 months
Peak pharyngeal pressure measurement from high-resolution manometry
Time frame: 24 months
Anterior tongue pressure measurement from Iowa Oral Performance Instrument (IOPI)
Time frame: 24 months
Patient-reported dysphagia symptoms based on Eating Assessment Tool- EAT10 score
Time frame: 24 months
Patient-reported quality of life based on SF-12 survey score
Time frame: 24 months
Patient-reported voice symptoms based on Voice Handicap Index - VHI10 score
Time frame: 24 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Apr 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Peter Belafsky, MD, PhD principal investigator
University of California Davis, Department of Otolaryngology
Maggie Kuhn, MD principal investigator
University of California Davis, Department of Otolaryngology
Nogah Nativ, PhD study director
University of California Davis, Department of Otolaryngology
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Autologous Muscle Derived Cells for Gastro-Intestinal Repair (AMDC-GIR) in people with Oropharyngeal Dysphagia, run by Peter Belafsky, MD at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 20 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Study product-related Serious Adverse Events (SAEs) (time frame: 24 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Apr 2026; AzTrials last refreshed its copy on 8 Nov 2026.