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

NCT ID: NCT04290182

Last Updated: 2026-01-27

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-03-01
• Study Completion Date: 2025-01-01

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.

Detailed Description

The general aim of the project is to develop a treatment for severe hoarseness due to vocal fold (VF) scarring. Vocal fold scarring can be caused by tumor surgery, radiotherapy, severe inflammation or is early acquired (sulcus vocalis with scar) and results in stiff vocal folds with decreased vibratory capacity and severe deterioration or total loss of voice (aphonia). There is no lasting effective treatment. Bone marrow derived mesenchymal stem cells (MSC) are immunomodulatory, decrease inflammation and improve endogenous healing. After receiving ethical permission the investigators have since 2012 treated 16 patients with manifest vocal fold scarring and severe hoarseness by scar resection and local injection of autologous bone marrow MSC to restore speech. This project was the first in the world to study the effects of MSC treatment of vocal fold scarring in humans. Analysis was made before and up to 12 months post operatively with voice recordings, examination with high speed camera and elasticity measurements of the vocal folds with novel technology. No side effects were found for any patient and for two thirds of the patients with 12 months follow-up the vocal fold function improved and no patient deteriorated.

While cell therapy with autologous MSC was classified according to the Tissue Legislation before 2015, it is now regarded as drug treatment. In accordance with this legislation, the MSC production is now full scale GMP. The investigators have recently received permissions from Swedish Medical Product Agency (DNr 5.1-2019-92069) and from the Regional ethic committee (Drn 2019-06160) for an open Phase I/Il study in patients with severe dysphonia and vocal fold scarring to evaluate safety, tolerability and vocal function after surgery with local administration of autologous mesenchymal stromal cell product KI-MSC-PL-204 as an extended study on 15 patients.

MSC may in the future be used to treat patients with severe hoarseness due to scarring, as well as other damages in the airways.

Conditions

Hoarseness; Dysphonia; Aphonia; Vocal Fold Scar

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Single arm: MSC administration to vocal fold scar

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)

Group Type: EXPERIMENTAL

MSC-KI-PL-204

Intervention Type: BIOLOGICAL

Autologous MSC product

Interventions

MSC-KI-PL-204

Autologous MSC product

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

• 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).

Exclusion Criteria

• 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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Swedish Foundation for Strategic Research

OTHER

Sponsor Role: collaborator

Karolinska University Hospital

OTHER

Sponsor Role: lead

Responsible Party

Stellan Hertegård

Adjunct Professor. MD PhD

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Patric Scicluna, Clin Research Manager

Role: STUDY_CHAIR

Karolinska Trial Alliance

Locations

Karolinska Trial Alliance

Stockholm, Stockholm County, Sweden

Countries

Sweden

Other Identifiers

2019-06160 and 5.1-2019-92069

Identifier Type: -

Identifier Source: org_study_id