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Amyotrophic Lateral SclerosisPhase 1/2NCT02478450

Study to Investigate the Safety of the Transplantation (by Injection) of Human Glial Restricted Progenitor Cells (hGRPs; Q-Cells®) Into Subjects With Amyotrophic Lateral Sclerosis (ALS)

A Phase 1/2 study testing Q-Cells in people with Amyotrophic Lateral Sclerosis, run by Q Therapeutics, Inc..

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Official record
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Trial summary

This study is a non-randomized, open-label, partially blinded, sequential cohort, dose-escalation study designed to obtain preliminary data on the safety, tolerability, and early efficacy of Q-Cells® transplantation in subjects with 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?
Q-Cells
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
No sites listed yet
Study dates
Dec 2026to Dec 2029
Phase
Phase 1/2
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

  • Subject has the ability to understand the purpose and risks of the study and provide a signed and dated informed consent and authorization to collect and use protected health information (PHI) in accordance with national and local subject privacy regulations.
  • Subject lives within reasonable driving distance of study center (approximately 3 hours).
  • Subject has a caregiver willing/able to assist in the transportation and care required by study participation.
  • Subject is 18 - 80 years of age (inclusive) on the first day of the Screening Period.
  • Subject is diagnosed with sporadic or familial ALS within the past 48 months.
  • Subject meets the laboratory-supported probable, clinically probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology El Escorial criteria.
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  • Subject has an upright FVC ≥65% of predicted value for age, height, and gender at Screening.
  • Subject has not taken riluzole for at least 30 days prior to the first day of the Screening Period, or has been on a stable dose of riluzole for at least 30 days prior to the first day of the Screening Period (Riluzole-naïve subjects are permitted in the study).
  • Subject is medically able to undergo the study procedures and physically able to adhere to the visit schedule at the time of study entry.
  • Women of childbearing capacity must have a negative pregnancy test during the Screening Period and at the Pre-Operative Visit.
  • Subject must agree to practice effective birth control during study participation.

Who cannot join

  • Subject in whom causes of neuromuscular weakness other than ALS have not been practically excluded.
  • Subject with a diagnosis of significant cognitive impairment, clinical dementia, or major psychiatric illness including psychosis, bipolar disease, major depression, as determined by the DSM-V.
  • Subject with a diagnosis of other neurodegenerative disease (e.g., Parkinson's disease, Alzheimer's disease).
  • Subject with a diagnosis of any medical condition that impairs nerve or muscle function (e.g., notable peripheral neuropathy, metabolic muscle disease).
  • Subject with a clinically significant history of unstable cardiac, pulmonary, renal, hepatic, endocrine, hematologic, or active malignancy or infectious disease or other medically significant illness, which, in the opinion of the Investigator, would preclude study participation.
  • Subject with a history of spine surgery or anatomic variation incompatible with route of administration (as determined by neurosurgeon).
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  • Subject with severe cervical or lumbar stenosis, cord compression, or cervical or lumbar myelopathy.
  • Subject with abnormal flow voids on the surface of the spinal cord suggestive of arteriovenous malformation (AVM).
  • Subject demonstrating any evidence of CNS malignancy or CNS lesions as defined by imaging studies of the CNS (MRI of brain and spinal cord).
  • Subject having uncontrolled hypertension (Systolic BP>180mmHg and/or Diastolic BP >110mmHg) or having a history of thrombotic events or poorly controlled medical conditions that, in the opinion of the Investigator and/or surgeon, increase risk of surgery.
  • Subject who cannot undergo MRI examination because of the presence of a pacemaker, an implanted defibrillator or certain other implanted electronic or metallic devices, or who have been or might have been exposed to metal fragments, or any reason the subject cannot undergo an MRI routinely for the duration of the trial.
  • Subject with clinically significant abnormal clinical laboratory values, as determined by the Investigator during the Screening Period.
  • Subject who is immune compromised or who has a condition contraindicated to treatment with immunosuppression agents (e.g., tuberculosis, latent infection).
  • Subject with an aspartate aminotransferase (AST) or alanine aminotransferase (ALT) value >3.0 times the upper limit of normal or creatinine >1.5 times the upper limit of normal and/or eGFR <50cc/min during the Screening Period.
  • Subject with a history of alcohol or drug abuse or dependence within 1 year of the first day of the Screening Period, per DSM-V criteria.
  • Subject unlikely to comply with study requirements, as determined by Investigator.
  • Subject who has been exposed to any other experimental agent (off-label use or investigational) within 30 days of the first day of the Screening Period. Biologic agents may need additional time for washout and will be evaluated by the Sponsor on a case-by-case basis.
  • Subject who has previously been administered stem cells.
  • Subject with pre-existing anti-human leukocyte antigen (HLA) class I or class II antibodies directed against the Q-Cells®, as determined by panel reactive antibody (PRA) assay during the Screening Period.
  • Subject with an allergy to Q-Cells® or any of its constituents (e.g., chicken eggs), or an allergy to any of the co-administered immunosuppressants or any of their excipients.
  • Subject with any medical condition or using concomitant medication that would contraindicate the use of tacrolimus, mycophenolate mofetil, or prednisone as determined by Investigator.
  • Subject with evidence of deep vein thrombosis (DVT) by venous ultrasound or any previous evidence of DVT.
  • Subject who, in the opinion of the Investigator, has taken or is taking concomitant medications, supplements, or other agents that may interfere with the safety evaluation of Q-Cells® or may affect the course of the subject's ALS progression.

About this study

Official title: A Phase 1/2a Open-Label Study to Investigate the Safety of the Transplantation (by Injection) of Human Glial Restricted Progenitor Cells (hGRPs; Q-Cells®) Into Subjects With Amyotrophic Lateral Sclerosis (ALS): Assessment of Localized Therapeutic Activity by Blinded Observation and Lateral Transplantation (ALTA-BOLT)

This study is a non-randomized, open-label, partially blinded, sequential cohort, dose-escalation study designed to obtain preliminary data on the safety, tolerability, and early efficacy of Q-Cells® transplantation in subjects with ALS. Following an initial cohort receiving cell transplants unilaterally in the lumbar spinal cord, subsequent cohorts will receive escalating doses transplanted unilaterally in cervical spinal cord. Subjects and outcome measure assessors will be blinded to side of treatment. The study will be conducted at sites with extensive clinical experience with the care of patients with ALS.

Read the detailed description

This study is a non-randomized, open-label, partially blinded, sequential cohort, dose-escalation study designed to obtain preliminary data on the safety, tolerability, and early efficacy of Q-Cells® transplantation in subjects with ALS. Following an initial cohort receiving cell transplants unilaterally in the lumbar spinal cord, subsequent cohorts will receive escalating doses transplanted unilaterally in cervical spinal cord. Subjects and outcome measure assessors will be blinded to side of treatment. The study will be conducted at sites with extensive clinical experience with the care of patients with ALS.

The study is planned to enroll up to 30 subjects over 24 months. Each subject will receive a single time point administration of Q-Cells®: with 5 or 10 (dependent upon dose level) transplantation foci targeted to the anterior horn in either the lumbar or cervical spinal cord.

The study consists of Screening, Pre-operative/Treatment, and Post-treatment study periods. The study data will be assessed for safety and efficacy after the last subject has completed the 9-month study visit. Following the 9-month study period, subjects who consent will continue to be followed for safety and efficacy long-term in a separate protocol.

Being tested
Q-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.

  • Q-CellsBiological

    Also called glial restricted progenitor cells

    cellular therapeutic comprised of human cells of the glial lineage

Study groups in NCT02478450
Study groupRoleWhat they receive
Cohort 1ExperimentalUnilateral lumbar surgical transplantation of Q-Cells dose level 1
Receives: Biological: Q-Cells
Cohort 2ExperimentalUnilateral cervical surgical transplantation of Q-Cells dose level 1
Receives: Biological: Q-Cells
Cohort 3ExperimentalUnilateral cervical surgical transplantation of Q-Cells dose level 2
Receives: Biological: Q-Cells
Cohort 4ExperimentalUnilateral cervical surgical transplantation of Q-Cells dose level 3
Receives: Biological: Q-Cells
Cohort 5ExperimentalUnilateral cervical surgical transplantation of Q-Cells dose level 4
Receives: Biological: Q-Cells
Cohort 6ExperimentalUnilateral cervical surgical transplantation of Q-Cells dose level 5
Receives: Biological: Q-Cells

What is being measured

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

  1. Safety of human glial restricted progenitor cell transplantation in patients with Amyotrophic Lateral Sclerosis. (adverse events)

    Safety will be measured by the number of therapy related adverse events.

    Measured: 9-month

Secondary outcomes (9)

  1. Score on the amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)

    Measured: 9-month

  2. Quantitative muscle strength values from a hand held dynamometer (HHD) will be tested bilaterally

    Measured: 9-month

  3. Hand grip (bilateral) values from a grip dynamometer

    Measured: 9-month

  4. Forced vital capacity (FVC)

    Measured: 9-month

  5. Maximum sniff nasal inspiratory pressure (SNIP)

    Measured: 9-month

  6. Electrical Impedance Myography (EIM) values on bilateral upper and lower limbs

    Measured: 9-month

  7. Pain scores on the Visual Analog Scale (VAS)

    Measured: 9-month

  8. Score on the ALS Specific Quality of Life Questionnaire - Revised

    Measured: 9-month

  9. Score on the Ashworth Spasticity Scale

    Measured: 9-month

Timeline

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

  1. First posted23 Jun 2015 (estimated)
  2. Study startDec 2026 (estimated)
  3. Primary completionDec 2028 (estimated)
  4. Study completionDec 2029 (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
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
30 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02478450
Sponsor's protocol ID
QALS-101
Status last verified
Jul 2026
First submitted
11 Jun 2015
Last updated
8 Jul 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Q-Cells in people with Amyotrophic Lateral Sclerosis, run by Q Therapeutics, Inc..

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 11 inclusion and 23 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: opening soon, as of the sponsor's last update on 8 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

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?

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 of human glial restricted progenitor cell transplantation in patients with Amyotrophic Lateral Sclerosis. (adverse events) (time frame: 9-month). 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 NCT02478450.

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 8 Jul 2026.

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