Repeated Mesenchymal Stem Cell Injections in ALS

NCT04821479 | Completed Phase 1 DMC

• 1 other identifier: 0208-16-HMO
• Study Type: interventional
• Target: 20
• Locations: 0 countries
• Sites: N/A

Brief Summary

An open-label, single-center clinical trial to evaluate the safety and efficacy of repeated intrathecal administrations of autologous bone marrow derived mesenchyme stem cells in ALS patients. The study includes 20 subjects (age: 20-70) with definite diagnosis of ALS and ALS-FRS-R score of at least 20 and disease-duration of less than 3 years. The treatment protocol includes four intrathecal injections of MSC, at intervals of 3 months between the injections. The primary endpoints are safety and tolerability. Several efficacy measures are assessed as secondary endpoints.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS; Mesenchymal stem cells; ALSFRSr

Outcome Measures

Primary Outcomes (2)

• Safety of multiple intrathecal MSC administration. Incidence of adverse events following multiple intrathecal MSC injections during 2 years follow-up period

  To evaluate the safety of 4 successive intrathecal administrations of autologous MSC cells administered every three months in ALS patient. Patients will be follow-up every 3 months for change in their neurological status and the incidence of adverse events. Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

  Up to 18 months: starting from inclusion until 3 months after the last MSC-injection

• Effect of repeated intrathecal MSC injections on the progression rate (monthly changes) of ALSFRSr

  Degree of change in the rate of progression (calculation of monthly change in ALSFRSr score: 0-48, higher scores are better) between the run-in period to 3 months post last MSC-injection

  Up to 18 months: starting from inclusion until 3 months after the last MSC-injection.

Secondary Outcomes (1)

• Effect of repeated intrathecal MSC injections on the progression rate (monthly changes) of respiratory forced vital capacity FVC

  Up to 18 months: starting from inclusion until 3 months after the last MSC-injection.

Study Arms (1)

Repeated MSCs treatment in ALS patients

EXPERIMENTAL

Four intrathecal (IT) administrations of autologous MSC cells administered every 3 months in ALS patients. the IT treatment will be administered through a regular lumbar puncture at a dose of 1x10\^6 MSCs per kg body weight in 3 ml saline.

Biological: Mesenchymal stem cells (MSC)

Interventions

Mesenchymal stem cells (MSC) BIOLOGICAL

Intrathecal injections of autologous bone marrow derived mesenchymal stem cells

Also known as: stem cells, Stromal cells

Repeated MSCs treatment in ALS patients

Eligibility Criteria

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

You may qualify if:

• Males and females ages 18 to 70 years old, at the Screening Visit.
• Sporadic or familial ALS diagnosed patients fulfilling the El Escorial clinical criteria for definite.
• Capable of providing informed consent and willing and able to follow study procedures, including willingness to undergo multiple/repeated lumbar punctures.
• ALSFRS-R ≥15 at the Screening Visit.

You may not qualify if:

• Patients with severe cognitive decline or inability to understand and sign the informed consent.
• Participation in another clinical trial within 1 year prior to start of the study
• Patients with active infections.
• Positive test for Hepatitis B, Hepatitis C, and/or HIV per laboratory evaluations at the screening visit.
• Any history of solid malignancy including any malignancy affecting the central nervous system within 5 years of the Screening Visit

Sponsors & Collaborators

• Hadassah Medical Organization: lead

Related Publications (1)

• Petrou P, Kassis I, Yaghmour NE, Ginzberg A, Karussis D. A phase II clinical trial with repeated intrathecal injections of autologous mesenchymal stem cells in patients with amyotrophic lateral sclerosis. Front Biosci (Landmark Ed). 2021 Oct 30;26(10):693-706. doi: 10.52586/4980.

  PMID: 34719198 DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

SLC25A33 protein, human

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 Officials

• Dimitrios Karussis, Professor

  Hadassah HMO

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Single center open label study

Study Record Dates

• First Submitted: October 19, 2020
• First Posted: March 29, 2021
• Study Start: January 1, 2016
• Primary Completion: June 30, 2020
• Study Completion: December 31, 2020
• Last Updated: March 29, 2021

Record last verified: 2020-05