Clinical Trial on The Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis (Extension CMN/ELA)

Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia

Status and phase: Completed; Phase 2; Phase 1

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Procedure: Laminectomy and bone marrow stem cells transplantation; Procedure: Intrathecal infusion of placebo (saline solution).; Procedure: Intrathecal infusion of autologous bone marrow stem cells

Study type: Interventional

Funder types: Other

Identifiers

NCT01254539

Extension CMN/ELA

2006-003096-12 (EudraCT Number)

EC07/90762 (Other Identifier)

Details and patient eligibility

About

The purpose of this clinical trial is to assess the feasibility and the security of the intraspinal and intrathecal infusion of autologous bone marrow stem cells for the treatment of Amyotrophic Lateral Sclerosis patients.

Full description

Patients with Amyotrophic Lateral Sclerosis (ALS) typically endure a progressive paralysis due to the continued loss of motoneurons that leads them to death in less than 5 years. No treatment has changed its natural history. Intraspinal injections of bone marrow mononuclear cells (MNC) have been able to ameliorate the course of ALS in murine models, acting as pumps of trophic factors that keep the motoneurons functional. Moreover, the clinical trial (Study NCT00855400 on www.ClinicalTrials.gov) conducted by our research group to determine the safety and efficacy of Autologous Stem Cell transplantation in Amyotrophic Lateral Sclerosis in humans, found that this procedure is feasible and safe. Continuing with that study, we have designed a phase I/II clinical trial to check the feasibility of the intraspinal and intrathecal infusion of autologous bone marrow stem cells.

Enrollment: 63 patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Diagnose established following the World Federation of Neurology criteria
• More than 6 and less than 36 months of evolution of the disease
• Medullar onset of the disease
• More than 18 and less than 70 years old
• Forced Vital Capacity ≥ 50%
• Total time of oxygen saturation <90% inferior to 5% of the sleeping time
• Signed informed consent

Exclusion criteria

• Neurological or psychiatric concomitant disease
• Need of parenteral or enteral nutrition through percutaneous endoscopic gastrostomy or nasogastric tube
• Concomitant systemic disease
• Treatment with corticosteroids, immunoglobulins or immunosuppressors during the last 12 months
• Inclusion in other clinical trials
• Unability to understand the informed consent

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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63 participants in 3 patient groups, including a placebo group

Autologous bone marrow stem cells intraspinal transplantation

Experimental group

Description:

T3-T4 laminectomy and bone marrow ficoll separated mononuclear autologous cells intraspinal transplantation

Treatment:

Procedure: Laminectomy and bone marrow stem cells transplantation

Intrathecal infusion of autologous bone marrow stem cells

Experimental group

Description:

Patients were drawn 2 ml of cerebrospinal fluid and infused 2 ml (two 1 ml syringes) of Autologous Stem Cells.

Treatment:

Procedure: Intrathecal infusion of autologous bone marrow stem cells

Intrathecal infusion of placebo (saline solution).

Placebo Comparator group

Description:

Patients were infused 2 ml of saline solution

Treatment:

Procedure: Intrathecal infusion of placebo (saline solution).