Clinical Trial: Controlled Study to Evaluate the Safety and Efficacy of Azficel-T for the Treatment of Vocal Fold Scarring and Age-Related Dysphonia

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

Brief 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.

Detailed Summary:

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.


Sponsor: Fibrocell Technologies, Inc.

Current Primary Outcome:

  • Videostroboscopy [ Time Frame: Four months after final treatment ]
    Absolute change from baseline in mucosal wave grade using videostroboscopy on each treated vocal fold 4 months after the final treatment.
  • Voice Handicap Index Score [ Time Frame: Four months after final treatment ]
    Absolute and percentage change from baseline in the Voice Handicap Index (VHI) score 4 months after the final treatment.
  • Perceptual analysis using GRBAS scale [ Time Frame: Four months after final treatment ]
    Perceptual analysis using the GRBAS (Grade of dysphonia, Roughness, Breathiness, Asthenia, Strain) scale as assessed by a blinded voice clinician 4 months after the final treatment.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Improvement in voice quality on visual analog scale [ Time Frame: Four months after final treament ]
    Subject's impression of improvement in voice quality using the visual analog scale (VAS) 4 months after the final treatment.
  • Improvement in voice quality using questionnaire [ Time Frame: Four months after final treatment ]
    Subject's impression of improvement in voice quality using questionnaire 4 months after the final treatment.
  • Change from baseline in auditory-perceptual ratings [ Time Frame: Four months after final treatment ]
    Absolute change from baseline in auditory-perceptual ratings 4 months after the final treatment.
  • Change from baseline in the noise-to-harmonic ratio [ Time Frame: Four months from final treatment ]
    Absolute change from baseline in the noise-to-harmonic ratio (dB) as measured from acoustic voice analysis 4 months after the final treatment.
  • Change from baseline in maximum phonation time [ Time Frame: Four months after final treatment ]
    Absolute change from baseline in maximum phonation time (sec) 4 months after the final treatment.
  • Proportion of subjects with "Normal" fundamental frequency [ Time Frame: Four months after final treatment ]
    Proportion of subjects with "Normal" fundamental frequency as measured from acoustic voice analysis 4 months after the final treatment.
  • Change from baseline in percentage jitter [ Time Frame: Four months after final treatment ]
    Absolute change from baseline in percentage jitter as measured from acoustic voice analysis 4 months after the final treatment.
  • Change from baseline in shimmer [ Time Frame: Four months after final treatment ]
    Absolute change from baseline in shimmer (dB) as measured from acoustic voice analysis 4 months after the final treatment.


Original Secondary Outcome: Same as current

Information By: Fibrocell Technologies, Inc.

Dates:
Date Received: March 21, 2014
Date Started: March 2014
Date Completion:
Last Updated: January 30, 2017
Last Verified: January 2017