Mesenchymal Stem Cell Exosomes for Congenital Myasthenic Syndrome

Recruiting at 2 trial locations

Overseen By Chadwick Prodromos

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: The Foundation for Orthopaedics and Regenerative Medicine

Disqualifiers: Pregnancy, Active cancer

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: Duration not specified

20 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment using Mesenchymal Stem Cell Exosomes, a type of cell-based therapy, to assist individuals with Congenital Myasthenic Syndrome, a condition that causes muscle weakness. The researchers aim to determine if these tiny cell parts can improve muscle strength and reduce symptoms. Suitable participants have received a diagnosis of Congenital Myasthenic Syndrome from a doctor and can travel to the treatment site. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative therapy.

Do I have to stop taking my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that mesenchymal stem cell exosomes have been studied for safety, particularly in treating conditions like ALS (a disease affecting nerve cells in the brain and spinal cord), Kennedy Disease, and CMS (Congenital Myasthenic Syndrome). These studies found the exosomes to be completely safe when administered nasally. No major side effects were reported, and they even helped slow disease progression and improve strength. Although specific safety data for the current trial is not yet available, these earlier findings suggest that the treatment is well-tolerated. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Congenital Myasthenic Syndrome, which typically involve medications like anticholinesterase inhibitors and immunosuppressants, Mesenchymal Stem Cell Exosome Solution offers a novel approach. This treatment is derived from exosomes, which are tiny particles released by stem cells that can promote healing and repair at a cellular level. Researchers are excited because these exosomes could potentially enhance muscle function and repair damaged neuromuscular junctions more effectively than current options. This innovative mechanism of action could lead to new therapeutic possibilities for patients who have limited options with existing treatments.

What evidence suggests that Mesenchymal Stem Cell Exosome Solution might be an effective treatment for Congenital Myasthenic Syndrome?

Studies have shown that tiny particles called exosomes, released by mesenchymal stem cells, can improve strength in people with muscle-related conditions. Research indicates that these treatments may benefit patients with Congenital Myasthenic Syndrome, a group of conditions causing muscle weakness. Exosomes facilitate cell communication and repair. Early findings suggest they can safely enhance muscle strength and slow the progression of diseases like ALS and Kennedy Disease. This offers hope for those with Congenital Myasthenic Syndrome as well. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

This trial is for individuals who have been diagnosed with Congenital Myasthenic Syndrome. Participants or their guardians must be able to give informed consent and travel to the treatment site. Pregnant individuals or those with active cancer are not eligible.

Inclusion Criteria

Patients must be able to provide informed consent, or have a guardian who does

I have been diagnosed with Congenital Myasthenic Syndrome by a doctor.

I can travel to the treatment location.

Exclusion Criteria

Patients will be excluded from the trial if they are pregnant

I do not have any active cancer at the time of screening.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Patients receive Mesenchymal Stem Cell Exosome solution

Duration not specified

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Mesenchymal Stem Cell Exosome Solution

Trial Overview The trial is testing a treatment called AlloEx exosomes, which are derived from Mesenchymal Stem Cells, on patients with Congenital Myasthenic Syndrome to assess its effectiveness and safety.

How Is the Trial Designed?

Group I: Treatment Group Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

The Foundation for Orthopaedics and Regenerative Medicine

Lead Sponsor

Trials

31

Recruited

540+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07226726

Patients With Congenital Myasthenic Syndrome Will be ... Study Overview. Patients with Congenital Myasthenic Syndrome will be treated with Mesenchymal Stem Cell Exosome solution. Single Arm non-controlled study. ...

2. e-century.us e-century.us/files/ajsc/14/4/ajsc0166665.pdf

Original Article Exosome-rich mesenchymal stem cell ... Conclusion: Intranasally-instilled AlloEx Exosomes® are completely safe, attenu- ate progression, and improve strength in ALS, Kennedy Disease, ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT07226726?rank=8

Patients With Congenital Myasthenic Syndrome Will be ... Study Overview. Patients with Congenital Myasthenic Syndrome will be treated with Mesenchymal Stem Cell Exosome solution. Single Arm non-controlled study. ...

4. researchgate.net researchgate.net/publication/392027205_Exosome-Rich_Mesenchymal_Stem_Cell_Secretome_Improves_Strength_in_Patients_with_ALS_Kennedy_Disease_Congenital_Myasthenic_Syndrome_and_Lewy_Body_Dementia

(PDF) Exosome-Rich Mesenchymal Stem Cell Secretome ... Exosome-Rich Mesenchymal Stem Cell Secretome Improves Strength in Patients with ALS, Kennedy Disease, Congenital Myasthenic Syndrome and Lewy Body Dementia.

5. htsyndication.com htsyndication.com/us-fed-news/article/clinical-trial%3A-patients-with-congenital-myasthenic-syndrome-will-be-treated-with-mesenchymal-stem-cell-exosome-solution/555770893

Patients With Congenital Myasthenic Syndrome Will be ... Brief Summary: Patients with Congenital Myasthenic Syndrome will be treated with Mesenchymal Stem Cell Exosome solution. Study Start Date: Jan.

6. meddatax.com meddatax.com/clinical-trials/NCT07226726

Patients With Congenital Myasthenic Syndrome Will be ... Patients with Congenital Myasthenic Syndrome will be treated with Mesenchymal Stem Cell Exosome solution ... safety data for this trial yet.