Biological Treatment of Amyotrophic Lateral Sclerosis (NeuStem-ALS)

Pomeranian Medical University Szczecin

Status and phase: Unknown; Phase 1

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Other: Biological: Cell-based therapeutics; Other: Symptomatic treatment of ALS

Study type: Interventional

Funder types: Other

Identifiers

NCT02193893

ZPO 02

ZPO 02, ALS-BMSC #01 (Other Identifier)

Details and patient eligibility

About

The purpose of this study is to test the safety and effectiveness of an autologous bone marrow-derived stem/progenitor cells infusion in the subjects with diagnosed amyotrophic lateral sclerosis.

Full description

Amyotrophic lateral sclerosis (ALS) is a universally fatal neurodegenerative condition that causes weakness leading to paralysis and death. Life expectancy is less that 5 years. The cause is generally unknown and there is no effective treatment. Patients with ALS typically exhibit a progressive paralysis due to the continued loss of motoneurons. Intraspinal injections of bone marrow mononuclear cells have been able to ameliorate the course of ALS in murine models. The purpose of this prospective, nonrandomized, open label, pilot study is to conduct the investigation of the safety and efficacy of infusion of autologous bone marrow-isolated stem/progenitor cells with different selected phenotypes into the subjects with diagnosed ALS. Especially, this clinical trial is designated to test the therapeutic (pro-regenerative and neuro-protective) functions of different stem/progenitor cell populations able to secrete bioactive neurotrophic factors. All patients enrolled will have a documented history of ALS disease prior to study enrollment. Patients diagnosed as early stage of ALS with the duration of disease less than 6 months and patients diagnosed with advanced stage of ALS disease with duration of 6-12 months will be recruited and allocated based on their disease severity to two treatment groups: Group I - patients of early ALS disease stage and Group II - patients of advanced ALS disease. Next, autologous bone marrow-isolated stem/progenitor cells administration to the cerebrospinal fluid at the site of the spinal cord will be performed. Finally, treatment safety, adverse events and exploratory parameters, including electromyographic (EMG) studies, forced vital capacity (FVC), functional rating scale (FRS) and maximum voluntary isometric contraction-arm (MVIC-arm) evaluation, to establish ALS progression rate will be recorded throughout the duration of the post-treatment follow up period.

Enrollment: 50 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• diagnosis of the ALS disease before the cell transplantation (diagnose established following the El Escorial criteria for definite ALS)
• good understanding of the protocol and willingness to consent
• patient is mentally intact and psychologically stable
• signed informed consent

Exclusion criteria

Concomitant of other systemic disease or diseases:

• inflammation (high protein or lymphocytosis in the CSF), active infections.
• diabetes,
• cardio-vascular disorders,
• cancer,
• autoimmune diseases
• renal failure,
• impaired hepatic function.
• subject is a respiratory dependent.
• subject unwilling or unable to comply with the requirements of the protocol.
• patient has been treated previously with any cellular therapy.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

50 participants in 2 patient groups

Stem/progenitor cells transplantation.

Active Comparator group

Description:

Intervention: Biological: Cell-based therapeutics Autologous bone marrow-derived stem/progenitor cells will be transplanted intrathecally (via a standard lumbar puncture) into early vs. progressive ALS subjects.

Treatment:

Other: Biological: Cell-based therapeutics

Standard treatment of ALS

Sham Comparator group

Description:

Symptomatic treatment of ALS without biologic cell-based treatment

Treatment:

Other: Symptomatic treatment of ALS