A Study of Local Administration of Autologous Mesenchymal Stromal Cells in Dysphonic Patients With Vocal Fold Scarring

NCT04290182 | Completed Phase 1 DMC

• 1 other identifier: 2019-06160 and 5.1-2019-92069
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

The overall aim of the project is to develop a new method for treatment of untreatable severe hoarseness due to vocal fold scarring by local injection of autologous mesenchymal stromal cells (MSC). At present there is no lasting effective treatment for this condition which results in personal suffering, and often extended sick leave, change of work or unemployement for the patients. Based on the previous results the investigators expect the autologous MSC product KI-MSC-PL-204 to be a new effective treatment without side effects for many patients with severe hoarseness or aphonia due to vocal fold scarring.

Conditions

Hoarseness; Dysphonia; Aphonia; Vocal Fold Scar

Keywords

Vocal fold scar; MSC; dysphonia

Outcome Measures

Primary Outcomes (1)

• Safety and tolerability of treatment

  Number of serious adverse events/ adverse events treatment and conseques

  3 year

Secondary Outcomes (6)

• Assessment of VF function by high speed laryngeal/stroboscopy recordings

  from baseline to 1 year after treatment.

• Assessment of subjective VHI change (points)

  changes from baseline to 1 year after treatement

• Assessment of Phonation Threshold Pressure changes (cmH2O)

  changes from baseline to 1 year after treatment

• Assessment of perceptual voice analysis changes (points)

  changes from baseline to 1 year after treatment

• Assessment of subjective VFI change (points)

  from baseline to 1 year after treatment

Study Arms (1)

Single arm: MSC administration to vocal fold scar

EXPERIMENTAL

1 single arm: Local injection of autologus MSC product (KI-MSC-PL-204) into scarred vocal fold (0,5-1 million cellls/Vocal fold, maximum 2 million cells if bilateral vocal fold scar)

Biological: MSC-KI-PL-204

Interventions

MSC-KI-PL-204 BIOLOGICAL

Autologous MSC product

Single arm: MSC administration to vocal fold scar

Eligibility Criteria

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

You may qualify if:

• Patients between 18-65 years with VF scarring and severe voice problems, such as permanent severe hoarseness, complete aphonia or severe voice strain during speech (\>1 year) where other treatments have proven ineffective and no alternative treatment is possible.
• No alternative treatment ongoing or planned (phonosurgery with augmentation implantation, voice therapy or other medical treatment).

You may not qualify if:

• Active treatment of laryngeal disorder, inflammatory condition of the larynx, or laryngeal/VF papilloma.
• Diagnosed or suspicion of local malignancy or other malignancies, Disease-free period of \>5 years after malignant disease (\>10 years for local laryngeal cancer).
• Smokers.
• Large scar defects.
• Pregnant or nursing (lactating) women.
• Serological evidence of infection with HIV, HBV, HCV, HTLV and/or syphilis.
• Active ongoing local or systemic infections.
• Ongoing immune suppressive treatment.

Sponsors & Collaborators

• Karolinska University Hospital: lead
• Swedish Foundation for Strategic Research: collaborator

Study Sites (1)

Karolinska Trial Alliance

Stockholm, Stockholm County, 11324, Sweden

Location

MeSH Terms

Conditions

Hoarseness; Dysphonia; Aphonia

Condition Hierarchy (Ancestors)

Voice Disorders; Laryngeal Diseases; Respiratory Tract Diseases; Respiration Disorders; Otorhinolaryngologic Diseases; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Signs and Symptoms, Respiratory

Study Officials

• Patric Scicluna, Clin Research Manager

  Karolinska Trial Alliance

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Adjunct Professor. MD PhD

Model Details: This is a phase I/IIa open, single-arm clinical study evaluating the safety and tolerability as well as the influence on VF function, of local injection of the autologous MSC product KI-MSC-PL-204, in patients suffering from chronic VF dysfunction.

Study Record Dates

• First Submitted: February 25, 2020
• First Posted: February 28, 2020
• Study Start: March 1, 2021
• Primary Completion: January 1, 2025
• Study Completion: January 1, 2025
• Last Updated: January 27, 2026

Record last verified: 2025-03

Locations

SE (1)