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ALS (Amyotrophic Lateral Sclerosis)Phase 1/2NCT03482050

A Study to Evaluate Transplantation of Astrocytes Derived From Human Embryonic Stem Cells, in Patients With Amyotrophic Lateral Sclerosis (ALS)

A Phase 1/2 study testing AstroRx in people with ALS (Amyotrophic Lateral Sclerosis), run by Kadimastem at 1 site in Israel.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This is a study of transplantation of Astrocytes derived from human embryonic stem cells, in patients with Amyotrophic Lateral Sclerosis (ALS). There will be no change in the routine ALS treatment of the patients enrolled into the study. Read more about this study

Who is it for?
Ages 18 to 70 years
Conditions the study is for
ALS (Amyotrophic Lateral Sclerosis)
What is being tested?
AstroRx
Could I receive a placebo?
No, everyone receives an active treatment
How many people
16 participants
Where
1 siteIsrael
Study dates
12 Apr 2018to 22 Jun 2020
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 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Main Inclusion Criteria:

  1. El Escorial criteria for probable or definite ALS
  2. Males and and non pregnant females between 18 and 70 years of age
  3. Patients with an ALS-FRS-R score of at least 30 with an ALS diagnosis of two years or less
  4. No history of active psychiatric disorder. Patients receiving antidepressants as a preventive treatment, with no history of active psychiatric disorder may be included.
  5. Patient has a good understanding of the study and nature of the procedure
  6. Patient provides written informed consent prior to any study procedure
  7. Patients should either be on a stable dose of Riluzole and/or Radicava® (if applicable) for at least 30 days, or not be treated with Riluzole and/or Radicava®
  8. Patient is medically able to tolerate immunosuppression regimen
  9. Presence of a willing and able caregiver who understands the need to attend all follow-up visits, even if mobility declines

Main Exclusion Criteria:

  1. Patient has a past infection or a positive test for HBV,HCV or HIV
  2. Patient is in need of respiratory support
  3. Patient has a lower than 10/12 in ALS-FRS-R respiratory parameters or below 70% of predicted slow vital capacity (SVC)
  4. Patient has renal failure
  5. Patient has impaired hepatic function
  6. Patient has a Body Mass Index (BMI) of <18.5 or > 30
  7. Patient suffers from significant cardiac disease, diabetes, autoimmune diseases, chronic severe infection, malignant disease or any other disease or condition that may risk the patient or interfere with the ability to interpret the study results
  8. Patient has systemic inflammation or active infections
  9. Patient has been treated previously with any stem cell therapy
  10. Current use of immunosuppressant medication or use of such medication within 6 weeks of Screening visit (Visit 0)
  11. Patient has participated in another clinical treatment trial or received other experimental medications outside of a clinical trial within 1 month prior to start of this study
  12. Any known immunodeficiency syndrome
  13. Any concomitant disease or condition limiting patient safety to participate

Trial locations and contacts

1 site in 1 country.

Israel1 site

  • Hadassah Ein Kerem Medical Center

    Jerusalem

Other trials in Israel

About this study

Official title: A Phase I/IIa, Open Label, Dose-escalating Clinical Study to Evaluate the Safety, Tolerability and Theraputic Effects of Transplantation of Astrocytes Derived From Human Embryonic Stem Cells (hESC), in Patients With Amyotrophic Lateral Sclerosis (ALS)

This is a study of transplantation of Astrocytes derived from human embryonic stem cells, in patients with Amyotrophic Lateral Sclerosis (ALS).

There will be no change in the routine ALS treatment of the patients enrolled into the study. Treatment will be administered in addition to the appropriate standard of care treatment.

The study hypothesis is that transplantation of Astrocyte(AstroRx) cells can compensate for the malfunctioning of patients' own astrocytes by restoring physiological capabilities like the reuptake of excessive glutamate, reducing oxidative stress, reducing other toxic compounds, as well as by secreting different neuroprotective factors

Being tested
AstroRx
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.

  • AstroRxBiological

    Astrocytes derived from human embryonic stem cells

Study groups in NCT03482050
Study groupRoleWhat they receive
AstroRxExperimentalNot described
Receives: Biological: AstroRx

What is being measured

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

  1. Number of participants with treatment emergent adverse events as assessed by CTCAE Version 4.03

    Safety and tolerability assessment will be based on treatment emerged adverse events

    Measured: 11 Months

Secondary outcomes (5)

  1. Change in the ALS functional rating scale

    Preliminary efficacy assessment to measure response to treatment or progression of disease. Scale includes 12 questions related to tasks. Each task rated on a five-point scale from 0 = unable to do the task, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0 = worst and 48 = best.

    Measured: 11 Months

  2. Change in predicted slow vital capacity (%SVC)

    Preliminary efficacy assessment to measure respiratory muscle strength

    Measured: 11 Months

  3. Change in muscle strength grading by JAMAR grip strength

    Preliminary efficacy assessment to measure hand grip strength

    Measured: 11 Months

  4. Change in muscle strength grading by hand held dynamometer (HHD)

    Preliminary efficacy assessment to measure muscle strength in limb muscles

    Measured: 11 Months

  5. Change in Quality of Life questionnaire (ALSAQ-40)

    Preliminary efficacy assessment to measure the subjective well-being of patients

    Measured: 11 Months

Timeline

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

  1. First posted29 Mar 2018
  2. Study start12 Apr 2018
  3. Primary completion22 Jun 2020
  4. Study completion22 Jun 2020

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
Sequential
Model description
Dose-escalating, four subject-groups clinical study; a single treatment administration of AstroRx, administered in an escalating low, medium and high dose or two consecutive administrations of the medium dose separated by an interval. AstroRx will be administered by intrathecal (spinal) injection to subjects with ALS at the early disease stage.
Masking
None (open label)
Enrolment
16 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03482050
Sponsor's protocol ID
ASTRO-001-IL
Status last verified
Jan 2021
First submitted
14 Mar 2018
Last updated
15 Jan 2021
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Publications

  • Gotkine M, Caraco Y, Lerner Y, Blotnick S, Wanounou M, Slutsky SG, Chebath J, Kuperstein G, Estrin E, Ben-Hur T, Hasson A, Molakandov K, Sonnenfeld T, Stark Y, Revel A, Revel M, Izrael M. Safety and efficacy of first-in-man intrathecal injection of human astrocytes (AstroRx(R)) in ALS patients: phase I/IIa clinical trial results. J Transl Med. 2023 Feb 14;21(1):122. doi: 10.1186/s12967-023-03903-3.

    PubMed 36788520

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing AstroRx in people with ALS (Amyotrophic Lateral Sclerosis), run by Kadimastem at 1 site in Israel.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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 15 Jan 2021. 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 Israel. 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?

Number of participants with treatment emergent adverse events as assessed by CTCAE Version 4.03 (time frame: 11 Months). 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 NCT03482050, 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 15 Jan 2021.

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