Overview

This trial is active, not recruiting.

Conditions dysphonia resulting from vocal fold scarring, age-related dysphonia
Treatments azficel-t (autologous fibroblasts), placebo
Phase phase 2
Sponsor Fibrocell Technologies, Inc.
Start date March 2014
End date April 2016
Trial size 20 participants
Trial identifier NCT02120781, FI-V-002

Summary

The objectives of this study are to assess the safety of azficel-T treatment for dysphonia related to vocal fold function and to evaluate the efficacy of azficel-T for the treatment of dysphonia related to vocal fold function.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, investigator, outcomes assessor)
Primary purpose treatment
Arm
(Experimental)
Azficel-T will be injected into the vocal fold(s) three times at two week intervals.
azficel-t (autologous fibroblasts) LAVIV™
Autologous fibroblasts will be cultured from two 3-mm hard palate punch biopsies. Biopsies will be shipped from the clinical sites to the Fibrocell manufacturing site where the cells will be harvested, tested for sterility, endotoxin level, cell identity, viability and concentration. When the desired cell number is reached, cells will be transported to the investigative site as a suspension in shipping media. Depending upon the clinical circumstances for each subject, the vocal fold(s) will be injected transorally or percutaneously in order to deposit 1.0 mL of study drug into the lamina propria layer of each vocal fold. The injection process will be visualized via a flexible fiberoptic laryngoscope inserted through the nostril.
(Placebo Comparator)
Sterile saline will be injected into the vocal fold(s) three times at two week intervals.
placebo Sterile saline for injection
Subjects randomized to placebo will receive injections of sterile saline into the vocal fold(s).

Primary Outcomes

Measure
Videostroboscopy
time frame: Four months after final treatment
Voice Handicap Index Score
time frame: Four months after final treatment
Perceptual analysis using GRBAS scale
time frame: Four months after final treatment

Secondary Outcomes

Measure
Improvement in voice quality on visual analog scale
time frame: Four months after final treament
Improvement in voice quality using questionnaire
time frame: Four months after final treatment
Change from baseline in auditory-perceptual ratings
time frame: Four months after final treatment
Change from baseline in the noise-to-harmonic ratio
time frame: Four months from final treatment
Change from baseline in maximum phonation time
time frame: Four months after final treatment
Proportion of subjects with "Normal" fundamental frequency
time frame: Four months after final treatment
Change from baseline in percentage jitter
time frame: Four months after final treatment
Change from baseline in shimmer
time frame: Four months after final treatment

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: 1. Subject has read and signed the Institutional Review Board (IRB)-approved informed consent form (ICF) before treatment 2. Subject is at least 18 years of age 3. Subject has presence of unilateral or bilateral vocal fold scarring or age-related dysphonia, as diagnosed by medical history and physical examination 4. Subject must have Grade 1-2 mucosal waves as determined by videostroboscopy 5. Subject has failed any one or more of the following treatments including, but not limited to, anti-reflux regimen, speech therapy, or vocal fold injection augmentation prior to screening 6. Subject feels that their voice quality is a major handicap 7. Subject must have a blood sample tested and found to be non-reactive for human immunodeficiency virus-1 (HIV-1) antibody, hepatitis B surface antigen and hepatitis C virus (HCV) antibody 8. If the subject is female and of childbearing potential, she must agree to use a medically acceptable means of birth control, and test negative on a urine pregnancy test 9. Subject must be willing and able to follow study procedures and instructions Exclusion Criteria: 1. Subject is pregnant or lactating 2. Subject is a smoker 3. Subject has an upper respiratory infection at baseline (subject can be rescheduled after four weeks) 4. Subject is already participating, or has participated in another clinical trial involving therapeutic intervention within 30 days prior to enrollment 5. Subject plans to begin or continue other vocal fold therapies during the course of this study 6. Subject has other concurrent laryngeal pathology including lesions that would require removal

Additional Information

Official title A Phase II, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Azficel-T for the Treatment of Vocal Fold Scarring and Age-Related Dysphonia
Principal investigator Dinesh Chhetri, MD
Description Twenty subjects with dysphonia caused by vocal fold scarring or age-related dysphonia will be randomized for treatment with autologous cultured fibroblasts (azficel-T, n=14) or placebo (saline, n=6). Subjects will receive treatment to the vocal fold(s) in the lamina propria compartment. Subjects with both unilateral and bilateral vocal fold scarring will be treated in this study. Only one vocal fold will be treated at each treatment session alternating to the opposite vocal fold (if applicable) at the next treatment. Subjects are to receive a total of three treatments with study drug (azficel-T or placebo) if one vocal fold is to be treated and up to a total of six treatments with study drug (azficel-T or placebo) if two vocal folds are to be treated at approximately 2-week intervals. Follow-up examinations will be performed at 1, 4, 8, and 12 months after the final treatment. If there are any evident safety issues, follow-up treatments will be delayed or withheld. In the Blinded Phase of the study, subjects will be followed for safety and efficacy for 4 months after the final treatment. After all subjects have completed the 4-month follow-up visit, the study will be unblinded and subjects will continue to be followed for safety for 12 months after the final treatment. Efficacy assessments will be made through the 12-month follow-up visit in order to document any duration of effect. All AEs that have an onset date from biopsy through the 4-month follow-up visit will be recorded.
Trial information was received from ClinicalTrials.gov and was last updated in July 2016.
Information provided to ClinicalTrials.gov by Fibrocell Technologies, Inc..