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Also known as Azficel-T (autologous fibroblasts), Autologous fibroblasts
Azficel-T · 1 trial · 2 indications
Any improvement from baseline in mucosal wave grade using videostrobscopy to assess the pliability of vocal folds which is visualized as mucosal waves on the vocal fold surface while a participant is phonating a sustained vowel sound.
Absolute change (decrease by 18 or more points) from baseline in the Voice Handicap Index (VHI) score 4 months after the final treatment. The VHI is a 30-item test with 10 items in 3 subscales: emotional, physical, and functional. Each item is scored on a 5-point scale: "0" indicating the subject never felt this about the voice problem and "4" indicating the subject always felt this to be the case. An 8-point difference on any subscale (sub-scale range 0-40, higher values represent worse outcomes) has been found to be significant, as was an 18-point difference in the total VHI score (Total scale range 0-120, higher values represent worse outcomes).
Mean percentage change from baseline in the Voice Handicap Index (VHI) score 4 months after the final treatment. The VHI is a 30-item test with 10 items in 3 subscales: emotional, physical, and functional. Each item is scored on a 5-point scale: "0" indicating the subject never felt this about the voice problem and "4" indicating the subject always felt this to be the case. An 8-point difference on any subscale (sub-scale range 0-40, higher values represent worse outcomes) has been found to be significant, as was an 18-point difference in the total VHI score (Total scale range 0-120, higher values represent worse outcomes).
Perceptual analysis of dysphonia using the Grade of Dysphonia, Roughness, Breathiness, Asthenia, Strain (GRBAS) scale is assessed by a blinded voice clinician 4 months after the final treatment. The voice clinician considers pre- and post-treatment voice recordings, and categorizes the individual parameters on the GRBAS scale (absent, mild, moderate, and severe). Scores of mild, moderate, or severe represent increasingly worse outcomes than a score of absent.
Perceptual analysis of roughness using the Grade of Dysphonia, Roughness, Breathiness, Asthenia, Strain (GRBAS) scale is assessed by a blinded voice clinician 4 months after the final treatment. The voice clinician considers pre- and post-treatment voice recordings, and categorizes the individual parameters on the GRBAS scale (absent, mild, moderate, and severe). Scores of mild, moderate, or severe represent increasingly worse outcomes than a score of absent.
Perceptual analysis of breathiness using the Grade of Dysphonia, Roughness, Breathiness, Asthenia, Strain (GRBAS) scale is assessed by a blinded voice clinician 4 months after the final treatment. The voice clinician considers pre- and post-treatment voice recordings, and categorizes the individual parameters on the GRBAS scale (absent, mild, moderate, and severe). Scores of mild, moderate, or severe represent increasingly worse outcomes than a score of absent.
Perceptual analysis of asthenia using the Grade of Dysphonia, Roughness, Breathiness, Asthenia, Strain (GRBAS) scale is assessed by a blinded voice clinician 4 months after the final treatment. The voice clinician considers pre- and post-treatment voice recordings, and categorizes the individual parameters on the GRBAS scale (absent, mild, moderate, and severe). Scores of mild, moderate, or severe represent increasingly worse outcomes than a score of absent.
Perceptual analysis of strain using the Grade of Dysphonia, Roughness, Breathiness, Asthenia, Strain (GRBAS) scale is assessed by a blinded voice clinician 4 months after the final treatment. The voice clinician considers pre- and post-treatment voice recordings, and categorizes the individual parameters on the GRBAS scale (absent, mild, moderate, and severe). Scores of mild, moderate, or severe represent increasingly worse outcomes than a score of absent.
| Arm | Type | Description |
|---|---|---|
| Azficel-T (autologous fibroblasts) | EXPERIMENTAL | Azficel-T will be injected into the vocal fold(s) three times at two week intervals. |
| Control | PLACEBO_COMPARATOR | Sterile saline will be injected into the vocal fold(s) three times at two week intervals. |
| Name | Type | Description |
|---|---|---|
| Azficel-T (autologous fibroblasts) | BIOLOGICAL | Autologous fibroblasts will be cultured from three 3-mm post auricular 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 | DRUG | Subjects randomized to placebo will receive injections of sterile saline into the vocal fold(s). |
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 medic...
Azficel-T is being studied for dysphonia resulting from vocal fold scarring, a voice disorder in which scar tissue on the vocal folds impairs voice production. It is also being evaluated in age-related dysphonia. It is an investigational cell therapy and is not approved for these uses.
Azficel-T is being developed by Castle Biosciences, Inc., which trades on the Nasdaq under the ticker CSTL. The company is the sponsor of the clinical program evaluating the therapy in voice disorders.
Azficel-T is in Phase 2 clinical development. One Phase 2 trial has been completed, and no trials are currently active. The program remains investigational, meaning the therapy has not been approved by the FDA for any indication.
Azficel-T has been studied in one completed Phase 2 trial, NCT02120781, titled a controlled study to evaluate the safety and efficacy of Azficel-T for vocal fold scarring and age-related dysphonia. The trial enrolled 21 participants in the United States and was randomized, double-blind, and placebo-controlled.
Yes. Azficel-T is also known as Azficel-T (autologous fibroblasts). The name reflects that it is a cell therapy derived from a patient's own fibroblasts, which is the basis of its development as an injectable treatment for vocal fold scarring and related dysphonia.