AzTrials
AzTrials

Amyotrophic Lateral SclerosisPhase 1/2ALSTEMNCT04651855

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

A Phase 1/2 study testing Mesenchymal stem cells isolated from Wharton's jelly in people with Amyotrophic Lateral Sclerosis, run by Polski Bank Komorek Macierzystych JSC (PBKM) at 1 site in Poland.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Amyotrophic Lateral Sclerosis
What is being tested?
Mesenchymal stem cells isolated from Wharton's jelly
Could I receive a placebo?
No, everyone receives an active treatment
How many people
20 participants
Where
1 sitePoland
Study dates
2 Dec 2020to Apr 2023
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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
Show all 14Show fewer
  • 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

Who cannot join

  • 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
Show all 25Show fewer
  • 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)

Trial locations and contacts

1 site in 1 country.

Poland1 site

  • JST sp. z o.o.

    Częstochowa, 42-202

Other trials in Poland

About this study

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

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.

Read the detailed description

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

Screening:

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)

Being tested
Mesenchymal stem cells isolated from Wharton's jelly
Keywords
Amyotrophic Lateral Sclerosis, Stem Cells
Medical subject headings
Amyotrophic Lateral Sclerosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Mesenchymal stem cells isolated from Wharton's jellyDrug

    Intrathecal administration of mesenchymal stem cells

Study groups in NCT04651855
Study groupRoleWhat they receive
Treatment armExperimentalIt 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.
Receives: Drug: Mesenchymal stem cells isolated from Wharton's jelly

What is being measured

The primary outcome is the main question the study is designed to answer.

  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).

    Measured: 3 month FU (follow-up)

Secondary outcomes (14)

  1. Disease progression

    Disease progression assessed in ALSFRS-R scale (Revised Amyotrophic Lateral Sclerosis Functional Rating Scale). Higher scores mean a better outcome.

    Minimum: 0 points Maximum: 48 points

    Measured: 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

  2. Pulmonary function decline

    Pulmonary function decline assessed in spirometry (forced vital capacity)

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

  3. Muscle strength decline

    Muscle strength decline

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

  4. Upper motor neuron function

    Upper motor neuron function assessed in UMNS scale (Upper Motor Neuron Scale). Best outcome 16 points, worst outcomes: 0 points and 48 points Minimum: 0 points Maximum: 48 points

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

  5. Cognitive function

    Cognitive function assessed in ECAS (The Edinburgh Cognitive and Behavioural ALS Screen). Higher scores mean a better outcome.

    Minimum: 0 points Maximum: 136 points

    Measured: screening and 12 month FU

  6. Quality of life changes

    Quality of life changes, assessed by EQ-5D questionnaire - standardized instrument for measuring generic health status. Higher scores mean a better outcome.

    Measured: 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

  7. The change of defined cytokines, chemokines, growth factors and pNFH (phosphorylated neurofilament heavy chain) level in CSF (Cerebrospinal fluid)

    The change of defined cytokines, chemokines, growth factors and pNFH level assessed in the samples of CSF

    Measured: run-in visit (-60 day), at baseline and at 1, 2 and 6 month FU (12 month FU optional)

  8. The change of defined cytokines, chemokines level in blood

    The change of defined cytokines, chemokines level assessed in the samples of blood serum

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

  9. The change of creatinine and p75ECD level in urine

    The change of creatinine and p75ECD level

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

  10. Muscle function changes

    Muscle function changes, assessed based on EMG examination (Electrophysiological examination of the muscle - MUNIX - motor unit number estimation)

    Measured: baseline and at 1, 2, 6 and 12 month FU

  11. The change of the brain visualization

    The change of the brain visualization in MRI (T1, T2 and DTI)

    Measured: run-in visit (-60 day), 6 and 12 month FU

  12. SAE (Serious Adverse Event)/AE (Adverse Event) and (S)AESI

    The number of SAE/AE and (S)AESI - defined as in Outcome 1

    Measured: 18 month FU

  13. Survival period to disease progression

    The number of days from patients randomization to the end of the patients participation in the trial or to the one of the following:

    • PAV (permanent assisted ventilation)
    • Tracheostomy
    • Death

    Measured: 18 month FU

  14. Mortality rate

    Percentage of deaths in the entire study population.

    Measured: 18 month FU

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Dec 2020
  2. Study start2 Dec 2020
  3. Primary completion8 Mar 2022
  4. Study completionApr 2023 (estimated)

Resources

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
Masking
None (open label)
Enrolment
20 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04651855
Sponsor's protocol ID
ALSTEM
Acronym
ALSTEM
Status last verified
Apr 2022
First submitted
30 Sep 2020
Last updated
3 May 2022
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Mesenchymal stem cells isolated from Wharton's jelly in people with Amyotrophic Lateral Sclerosis, run by Polski Bank Komorek Macierzystych JSC (PBKM) at 1 site in Poland.

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 14 inclusion and 25 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: status not confirmed, as of the sponsor's last update on 3 May 2022. 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 Poland. 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?

Results are not available in the source record.

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?

The number of (S)AESI [(Serious) Adverse Event of Special Interest] (time frame: 3 month FU (follow-up)). 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 NCT04651855.

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 3 May 2022.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in