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)

NCT02478450 | Unknown Phase 1 DMC FDA Drug

• 1 other identifier: QALS-101
• Study Type: interventional
• Target: 30
• Locations: 0 countries
• Sites: N/A

Brief 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. 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.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (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.

  9-month

Secondary Outcomes (9)

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

  9-month

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

  9-month

• Hand grip (bilateral) values from a grip dynamometer

  9-month

• Forced vital capacity (FVC)

  9-month

• Maximum sniff nasal inspiratory pressure (SNIP)

  9-month

Study Arms (6)

Cohort 1

EXPERIMENTAL

Unilateral lumbar surgical transplantation of Q-Cells dose level 1

Biological: Q-Cells

Cohort 2

EXPERIMENTAL

Unilateral cervical surgical transplantation of Q-Cells dose level 1

Biological: Q-Cells

Cohort 3

EXPERIMENTAL

Unilateral cervical surgical transplantation of Q-Cells dose level 2

Biological: Q-Cells

Cohort 4

EXPERIMENTAL

Unilateral cervical surgical transplantation of Q-Cells dose level 3

Biological: Q-Cells

Cohort 5

EXPERIMENTAL

Unilateral cervical surgical transplantation of Q-Cells dose level 4

Biological: Q-Cells

Cohort 6

EXPERIMENTAL

Unilateral cervical surgical transplantation of Q-Cells dose level 5

Biological: Q-Cells

Interventions

Q-Cells BIOLOGICAL

cellular therapeutic comprised of human cells of the glial lineage

Also known as: glial restricted progenitor cells

Cohort 1; Cohort 2; Cohort 3; Cohort 4; Cohort 5; Cohort 6

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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).
• 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.

Sponsors & Collaborators

• Q Therapeutics, Inc.: lead

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 11, 2015
• First Posted: June 23, 2015
• Study Start: March 1, 2024
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: August 21, 2023

Record last verified: 2023-08