The Evaluation of the Effect of Mesenchymal Stem Cells on the Immune System of Patients With ALS

NCT04651855 | Unknown Phase 1 DMC

• 1 other identifier: ALSTEM
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

The objective of this study is to evaluate the safety of intrathecal administration of Wharton's Jelly Mesenchymal Stem Cells (WJMSC) and the impact on the immune system of patients with Amyotrophic Lateral Sclerosis.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

Amyotrophic Lateral Sclerosis; Stem Cells

Outcome Measures

Primary Outcomes (1)

• The number of (S)AESI [(Serious) Adverse Event of Special Interest]

  (S)AESI are defined as: 1. Meningitis and encephalitis. 2. Toxic encephalopathy. 3. High fever \>39⁰C. 4. Epileptic seizures that are not connected to conditions above (meningitis, encephalitis, toxic encephalopathy, high fever).

  3 month FU (follow-up)

Secondary Outcomes (14)

• Disease progression

  screening, run-in period (-60 day and -30 day), at baseline and at 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 and 18 month FU

• Pulmonary function decline

  screening, run-in period (-60 day and -30 day), at baseline and at 1, 2, 3, 6, 9 and 12 month FU.

• Muscle strength decline

  screening, run-in period (-60 day and -30 day), at baseline and at 1, 2, 3, 6, 9 and 12 month FU

• Upper motor neuron function

  screening, run-in period (-60 day and -30 day), at baseline and at 1, 2, 3, 6, 9 and 12 month FU

• Cognitive function

  screening and 12 month FU

Study Arms (1)

Treatment arm

EXPERIMENTAL

It is planned that IMP administration will be performed three times for each enrolled patient. IMP administration could be performed only if the patients does not have any contraindications for lumbar puncture.

Drug: Mesenchymal stem cells isolated from Wharton's jelly

Interventions

Mesenchymal stem cells isolated from Wharton's jelly DRUG

Intrathecal administration of mesenchymal stem cells

Treatment arm

Eligibility Criteria

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

You may qualify if:

• Adult patients (at least 18 years old)
• The minimum patient's weight is not less than 40 kg
• Diagnosis of sporadic ALS, definite or probable, as defined by El Escorial World Federation of Neurology criteria
• History of ALS symptoms less than 2 years duration from the first symptoms of the disease
• More than 6 months from diagnosis of the disease
• Disease progression at 6 past months at least 3 points during this period of time assessed in ALSFRS-R scale
• ALSFRS-R scale of at least 30 at screening appointment
• Forced vital capacity \>70% of predicted value for age, gender and height
• Treatment with stable dose of riluzole(2x 50mg per 24h) before baseline visit (for at least 1 month)
• Capable of providing written informed consent
• Able to comply with study requirements and willing to follow all study procedures and follow-up visits
• Women of child-bearing age and men with partners of child-bearing potential must agree to use two forms of contraceptive therapy throughout the course of the trial
• Women of child-bearing age must undergo pregnancy test
• Polish-language native speakers or patients who are proficient in the Polish language

You may not qualify if:

• Pregnancy or breastfeeding
• Tracheostomy
• Ventilator dependence
• Renal disease with creatinine \>2mg/dl
• Liver disease with ALT, AST or GGTP 2-fold higher than upper normal limit
• Positive test for HBV, HCV, HIV with NAT method
• Positive tests for syphilis
• Any other clinically significant abnormalities on laboratory evaluation
• Any condition that would compromise ability of undergoing lumbar puncture
• Active systemic disease
• Autoimmune disease (Hashimoto disease under control is allowed)
• Uncontrolled diabetes (HbA1c \> 8%)
• Pulmonary disease that could affect interpretation of spirometry
• Neurological concomitant disease
• Unstable psychiatric concomitant disease

Sponsors & Collaborators

• Polski Bank Komorek Macierzystych JSC (PBKM): lead
• National Center for Research and Development, Poland: collaborator

Study Sites (1)

JST sp. z o.o.

Częstochowa, 42-202, Poland

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 30, 2020
• First Posted: December 3, 2020
• Study Start: December 2, 2020
• Primary Completion: March 8, 2022
• Study Completion: April 1, 2023
• Last Updated: May 3, 2022

Record last verified: 2022-04

Locations

PL (1)