HoarsenessPhase 1/2NCT04290182
A Study of Local Administration of Autologous Mesenchymal Stromal Cells in Dysphonic Patients With Vocal Fold Scarring
A Phase 1/2 study testing MSC-KI-PL-204 in people with Hoarseness and Dysphonia, run by Karolinska University Hospital at 1 site in Sweden.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Hoarseness, Dysphonia, Aphonia
- What is being tested?
- MSC-KI-PL-204
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteSweden
- Study dates
- 1 Mar 2021to 1 Jan 2025
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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).
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Open Phase I/II Study in Patients With Dysphonia and Vocal Fold Scarring to Evaluate Safety, Tolerability and Vocal Function After Surgery With Local Administration of Autologous Mesenchymal Stromal Cells
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.
Read the 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
- Being tested
- MSC-KI-PL-204
- Keywords
- Vocal fold scar, MSC, dysphonia
- Medical subject headings
- Hoarseness, Dysphonia, Aphonia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MSC-KI-PL-204Biological
Autologous MSC product
| Study group | Role | What they receive |
|---|---|---|
| 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) Receives: Biological: MSC-KI-PL-204 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of treatment
Number of serious adverse events/ adverse events treatment and conseques
Measured: 3 year
Secondary outcomes (6)
Assessment of VF function by high speed laryngeal/stroboscopy recordings
Description: Assessment of VF function change from baseline to 1 year after treatment, as evaluated by an expert panel of high speed laryngeal/stroboscopy recordings. (Expert panel catagories: (Improved, unchanged, deteriorated)
Measured: from baseline to 1 year after treatment.
Assessment of subjective VHI change (points)
Assessment of patient´s subjective VHI (voice handicap index) ratings change (points)
Measured: changes from baseline to 1 year after treatement
Assessment of Phonation Threshold Pressure changes (cmH2O)
Measurements of phonation threshold pressure (PTP) changes from baseline to 1 year after treatment
Measured: changes from baseline to 1 year after treatment
Assessment of perceptual voice analysis changes (points)
Evaluation by an expert panel of perceptual voice parameters, changes from baseline to 1 year after treatment
Measured: changes from baseline to 1 year after treatment
Assessment of subjective VFI change (points)
Assessment of Vocal fatigue index (VFI) changes from baseline to 1 year after treatment
Measured: from baseline to 1 year after treatment
Assessment of maximum phonation time change (seconds)
Measurements of maximum phonation time change from baseline to 1 year after treatment
Measured: from baseline to 1 year after treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Feb 2020
- Study start1 Mar 2021
- Primary completion1 Jan 2025
- Study completion1 Jan 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for HoarsenessStudies of the same condition, open and under way.
- More trials run by Karolinska University HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- 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.
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Karolinska University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Swedish Foundation for Strategic Research
- Responsible party
- Principal investigator, Stellan Hertegård, Karolinska University Hospital
- Investigators
- Patric Scicluna, Clin Research Manager, Study chair, Karolinska Trial Alliance
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04290182
- Sponsor's protocol ID
- 2019-06160 and 5.1-2019-92069
- Other IDs
- 2019
- Status last verified
- Mar 2025
- First submitted
- 25 Feb 2020
- Last updated
- 27 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Bergstrom Borlin E, Nygren U, Sodersten M, Granqvist S, Kadri N, Rasmusson Duprez I, Le Blanc K, Hertegard S. An open phase I/IIa study evaluating safety, patient-reported outcomes and voice function after surgery, local administration of mesenchymal stromal cells and voice training in patients with vocal fold scarring and dysphonia. Stem Cell Res Ther. 2026 Apr 19;17(1):146. doi: 10.1186/s13287-026-05022-4.
PubMed 42002761
Study at a glance
From the registry record, last updated by the sponsor on 27 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Phase I/II Study in Patients With Dysphonia and Vocal Fold Scarring to Evaluate Safety, Tolerability and Vocal Function After Surgery With Local Administration of Autologous Mesenchymal Stromal Cells
- ClinicalTrials.gov ID
- NCT04290182
- Acronym / sponsor ID
- 2019-06160 and 5.1-2019-92069
- Conditions
- Hoarseness, Dysphonia, Aphonia, Vocal Fold Scar
- Keywords
- Vocal fold scar, MSC, dysphonia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Karolinska University HospitalUniversity, hospital or other
- Collaborators
- Swedish Foundation for Strategic Research
- Study start
- 1 Mar 2021 (actual)
- Primary completion
- 1 Jan 2025 (actual)
- Study completion
- 1 Jan 2025 (actual)
- First posted
- 28 Feb 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- 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.
| Arm | Type | Receives |
|---|---|---|
| Single arm: MSC administration to vocal fold scar | Experimental | Biological: MSC-KI-PL-2041 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) |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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).
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MSC-KI-PL-204Autologous MSC product | Biological | Single arm: MSC administration to vocal fold scar |
Primary outcome
Safety and tolerability of treatment
Time frame: 3 year
Number of serious adverse events/ adverse events treatment and conseques
6 secondary outcomes
Assessment of VF function by high speed laryngeal/stroboscopy recordings
Time frame: from baseline to 1 year after treatment.
Assessment of subjective VHI change (points)
Time frame: changes from baseline to 1 year after treatement
Assessment of Phonation Threshold Pressure changes (cmH2O)
Time frame: changes from baseline to 1 year after treatment
Assessment of perceptual voice analysis changes (points)
Time frame: changes from baseline to 1 year after treatment
Assessment of subjective VFI change (points)
Time frame: from baseline to 1 year after treatment
Assessment of maximum phonation time change (seconds)
Time frame: from baseline to 1 year after treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jan 2026.
- Derived from this study: PubMed 42002761Bergstrom Borlin E, Nygren U, Sodersten M, Granqvist S, Kadri N, Rasmusson Duprez I, Le Blanc K, Hertegard S. An open phase I/IIa study evaluating safety, patient-reported outcomes and voice function after surgery, local administration of mesenchymal stromal cells and voice training in patients with vocal fold scarring and dysphonia. Stem Cell Res Ther. 2026 Apr 19;17(1):146. doi: 10.1186/s13287-026-05022-4.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Patric Scicluna, Clin Research Manager study chair
Karolinska Trial Alliance
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing MSC-KI-PL-204 in people with Hoarseness and Dysphonia, run by Karolinska University Hospital at 1 site in Sweden.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of treatment (time frame: 3 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04290182, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jan 2026.