The Evaluation of the Effect of Mesenchymal Stem Cells on the Immune System of Patients With ALS
The Evaluation of the Effect of Wharton's Jelly Mesenchymal Stem Cells (WJMSCs) on the Immune System of Patients With Amyotrophic Lateral Sclerosis (ALS)
  • Phase

    Phase 1/Phase 2
  • Study Type

  • Status

    Active, not recruiting
  • Study Participants

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.
Clinical Phase: I/II

Population: Patients with Amyotrophic Lateral Sclerosis.

Project Design: One arm, non-blinded, open label study

Planned Sample Size: 20 patients

Investigational Medicinal Product: active IMP - mesenchymal stem cells isolated from Wharton's jelly


Three visits on site to check the eligibility criteria (around 90, 60 and 30 days before first IMP administration)

Treatment (IMP administration):

Each patient will receive IMP three times: on baseline (day 0), 30 and 60 days after baseline (+/- 7 days).

Administration route: intrathecal

Follow up:

Duration: 18 months after first IMP administration Four on-site visits (3, 6, 9, 12 months after first IMP administration) and seven phone visits (4, 5, 7, 8, 10, 11 and 18 months after first IMP administration)
Study Started
Dec 02
Primary Completion
Mar 08
Study Completion
Apr 30
Last Update
May 03

Drug Mesenchymal stem cells isolated from Wharton's jelly

Intrathecal administration of mesenchymal stem cells

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.


Inclusion Criteria:

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

Exclusion Criteria:

Pregnancy or breastfeeding
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
High risk of suicide
History of substance abuse within past year
History of malignancy, within the previous 5 years, including melanoma with exception of localized skin cancers
Any other clinically significant medical condition that can compromise patient's safety in the opinion of the investigator
Treatment with immunomodulatory drugs (for example immunoglobulins, corticosteroids or other immunosuppressant) in last 6 months
Participation in another clinical trial in last 6 months
Previous cellular therapy of any kind
Hypersensitivity to any component used in the cell culture
Nuchal rigidity and other signs of meningitis
Patients on chronic anticoagulation treatment (heparin/ warfarin/acenocoumarol/(N)OAC)
No Results Posted