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Amyotrophic Lateral SclerosisPhase 1NCT05306457

CNS10-NPC-GDNF Delivered to the Motor Cortex for ALS

A Phase 1 study testing CNS10-NPC-GDNF in people with Amyotrophic Lateral Sclerosis, run by Cedars-Sinai Medical Center at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

The investigator is examining the safety of transplanting cells, that have been engineered to produce a growth factor, into the motor cortex (brain) of patients with Amyotrophic Lateral Sclerosis (ALS). 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?
CNS10-NPC-GDNF
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
16 participants
Where
1 siteUnited States
Study dates
8 May 2022to Oct 2026
Phase
Phase 1
Are there results?
Not yet: the study is under way

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

  • Confirmed diagnosis of ALS (Possible, Lab-supported Probable, Probable or Definite El Escorial Criteria)
  • Duration of ALS symptoms ≤ 36 months
  • Progressive weakness in upper extremities, with EMG supported evidence of denervation in both upper extremities
  • Forced Vital Capacity ≥50% of predicted normal in supine
  • Age: 18 years or older
  • Able to provide Informed Consent
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  • Be geographically accessible to the study site and able to travel to study site for required visits
  • Have caregiver to assist in the transportation and care required by participation in the study
  • Not taking riluzole and/or edaravone or on a stable dose for ≥ 30 day
  • For women of child bearing capacity, negative pregnancy test prior to surgery and willingness to use birth control for the duration of the trial.
  • Medically able to undergo craniotomy as determined by the site PI and/or investigators
  • Medically able to tolerate the immunosuppression regimen as determined by the site PI

Who cannot join

  • Using invasive ventilatory assistance
  • Diagnosis of another active or unstable medical illness that may interfere with study participation at discretion of PI
  • Presence of any of the following conditions:
  • Current drug or alcohol abuse
  • Any known immunodeficiency syndrome
  • Unstable medical condition
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  • Unstable psychiatric illness including psychosis and untreated major depression within 90 days of screening
  • Persons of child bearing capacity not willing to practice birth control
  • Receiving any investigational device/biologic/drug in the past 30 days or any previous exposure to stem cell therapy
  • Any condition in the upper extremities that precludes serial strength or coordination testing
  • Any condition that the investigators feel may pose complications for the surgery
  • Any condition or ALS disease phenotype that the site PI feels may interfere with participation in the study or in the interpretation of study endpoints
  • Allergy to Beta-Lactam antibiotics
  • Donor Specific Antibodies (DSA) ≥ 2500MFI or CPRA ≥ 20%
  • Contraindications to MRI

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Cedars-Sinai Medical Center

    Los Angeles, California, 90048

Other trials in United States

About this study

Official title: Human Neural Progenitor Cells Secreting Glial Cell Line-Derived Neurotrophic Factor (CNS10-NPC-GDNF) Delivered to the Motor Cortex for the Treatment of Amyotrophic Lateral Sclerosis

The investigator is examining the safety of transplanting cells, that have been engineered to produce a growth factor, into the motor cortex (brain) of patients with Amyotrophic Lateral Sclerosis (ALS). The cells are called neural progenitor cells, which are a type of stem cell that can become several different types of cells in the nervous system. These cells have been derived to specifically become astrocytes, which is a type of neural cell. The growth factor is called glial cell line-derived neurotrophic factor, or GDNF. GDNF is a protein that promotes the survival of many types of neural cells. Therefore, the cells are called "CNS10-NPC-GDNF." The investigational treatment has been tested in people by delivering it to the spinal cord. However, it has only been delivered to the motor cortex of animals. In this study, we want to learn if CNS10-NPC-GDNF cells are safe to transplant into the motor cortex (brain) of people.

Read the detailed description

This study will be the first to use a genetically modified progenitor cell line delivered to the motor cortex to treat a neurodegenerative disease. This is a Phase 1/2a, single-center, safety study of two escalating doses of human neural progenitor cells expressing GDNF (CNS10-NPC-GDNF) delivered unilaterally to the "hand-knob" area of the motor cortex of patients with ALS.

Subjects meeting all Eligibility Criteria and providing Informed Consent will be enrolled in one of three sequential dosing groups. Subjects will be treated sequentially with a minimum of one month interval between surgeries for the first three subjects in each dosing cohort. The remaining subjects in the cohort will be treated with a minimum interval of at least one week between surgeries.

Primary Outcome:

Safety, as evaluated by:

  • Adverse Events and Serious Adverse Events
  • Post-op MRI and/or CT (with contrast) and as clinically indicated
Being tested
CNS10-NPC-GDNF
Keywords
ALS, Stem cells, Growth Factor, Regenerative, Neural Progenitor Cells, NPC, Transplantation
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.

  • CNS10-NPC-GDNFBiological

    Unilateral injections of CNS10-NPC-GDNF into the motor cortex

Study groups in NCT05306457
Study groupRoleWhat they receive
CNS10-NPC-GDNF - Group AExperimentalUnilateral, Motor Cortex, 0.25x10^6 cells in 10 µL/site, 21 sites (5.25x10^6 total cells) - Motor cortex corresponding to the non-dominant hand
Receives: Biological: CNS10-NPC-GDNF
CNS10-NPC-GDNF - Group BExperimentalUnilateral, Motor Cortex, 0.5x10^6 cells in 10 µL/site, 21 sites (10.5x10^6 total cells) - Motor cortex corresponding to the non-dominant hand
Receives: Biological: CNS10-NPC-GDNF
CNS10-NPC-GDNF - Group CExperimentalUnilateral Motor Cortex, 0.5x10^6 cells in 10 µL/site, 21 sites (10.5x10^6 total cells) - Motor cortex corresponding to the dominant hand
Receives: Biological: CNS10-NPC-GDNF

What is being measured

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

  1. Safety, as evaluated by the incidence of Adverse Events and Serious Adverse Events and their relationship to the treatment

    Measured: 12 months post-operatively

  2. Safety, as evaluated by changes from baseline in the brain MRI

    Measured: 12 months post-operatively

Secondary outcomes (7)

  1. Force Generation via Accurate Test of Limb Isometric Strength (ATLIS) testing

    Change from baseline for force generation by ATLIS

    Measured: ATLIS testing will be performed 7 times over 15 months

  2. Pinch Strength

    Change from baseline for pinch strength

    Measured: Pinch Strength testing will be performed 7 times over 15 months

  3. Hand/Wrist Strength

    Change from baseline for Hand/Wrist strength using Hand-held dynamometer

    Measured: Hand/Wrist strength testing will be performed 7 times over 15 months

  4. Compound Motor Action Potential (CMAP)

    Change from baseline for CMAP

    Measured: CMAP will be performed 7 times over 15 months

  5. Functional Hand assessments using 9-hole peg test

    Change from baseline for 9-hole peg test

    Measured: 9-hole peg testing will be performed 7 times over 15 months

  6. Penn Upper Motor Neuron Score (PUMNS)

    Change from baseline for Penn Upper Motor Neuron Score. (Scale of 0-32, where 0 is normal)

    Measured: PUMNS will be performed 7 times over 15 months

  7. Hand Knob - Functional MRI (fMRI)

    Changes from baseline in brain activity in the hand knob area evaluated by fMRI

    Measured: fMRI will be performed up to 4 times over 15 months

Timeline

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

  1. First posted1 Apr 2022
  2. Study start8 May 2022
  3. Primary completionOct 2026 (estimated)
  4. Study completionOct 2026 (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
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
16 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05306457
Sponsor's protocol ID
STUDY00000278
Status last verified
Feb 2025
First submitted
21 Dec 2021
Last updated
19 Aug 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing CNS10-NPC-GDNF in people with Amyotrophic Lateral Sclerosis, run by Cedars-Sinai Medical Center at 1 site in United States.

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 12 inclusion and 15 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: under way, not looking for participants, as of the sponsor's last update on 19 Aug 2025. 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 United States. 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 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.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

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?

Safety, as evaluated by the incidence of Adverse Events and Serious Adverse Events and their relationship to the treatment (time frame: 12 months post-operatively). The study lists 2 primary outcomes in all. 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 NCT05306457.

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 19 Aug 2025.

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