Clinical trial NCT00855400: Clinical Trial on the Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis
Official title: Phase I/II Clinical Trial on the Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Keywords: Amyotrophic Lateral Sclerosis
Bone marrow
Stem cells
Autologous
Safety
Lead sponsor: Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
Sponsor class: OTHER
Collaborators: name: Carlos III Health Institute
name: Hospital Universitario Virgen de la Arrixaca
name: Hospital General Universitario Morales Meseguer
name: Diógenes Foundation
First posted: 2009-03-04
Enrollment: 11
Sex: ALL
Minimum age (years): 20
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

Detailed 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. We have designed a phase I/II clinical trial to check the feasibility of this approach in humans.

Eligibility criteria: 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 20 and less than 65 years old
* Forced Vital Capacity equal or superior to 50%
* Total time of oxygen saturation \<90% inferior to 2% 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

Primary outcomes:
measure: Forced vital capacity
timeFrame: Every 3 months
Secondary outcomes:
measure: ALS-FRS, MRC and Norris scales
timeFrame: Every 3 months
measure: Absence of adverse events
timeFrame: Every week / month depending on the study phase
Other outcomes:
Locations:
city: El Palmar
country: Spain
facility: Hospital Universitario Virgen de la Arrixaca
state: Murcia
Study officials:
affiliation: Hospital Universitario Virgen de la Arrixaca
name: Jose Maria Moraleda Jiménez, M.D. Ph.D.
role: STUDY_DIRECTOR
References:
citation: Blanquer M, Moraleda JM, Iniesta F, Gomez-Espuch J, Meca-Lallana J, Villaverde R, Perez-Espejo MA, Ruiz-Lopez FJ, Garcia Santos JM, Bleda P, Izura V, Saez M, De Mingo P, Vivancos L, Carles R, Jimenez J, Hernandez J, Guardiola J, Del Rio ST, Antunez C, De la Rosa P, Majado MJ, Sanchez-Salinas A, Lopez J, Martinez-Lage JF, Martinez S. Neurotrophic bone marrow cellular nests prevent spinal motoneuron degeneration in amyotrophic lateral sclerosis patients: a pilot safety study. Stem Cells. 2012 Jun;30(6):1277-85. doi: 10.1002/stem.1080.