A Multicenter Phase I/II Clinical Trial to Evaluate Safety of Mesenchymal Stem Cell in Patients With Amyotrophic Sclerosis Lateral

Andalusian Initiative for Advanced Therapies - Fundación Pública Andaluza Progreso y Salud

Status and phase: Completed; Phase 2; Phase 1

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: Intravenous administration of 2 million of MSC; Drug: Intravenous administration of 4 million of MSC; Drug: Intravenous administration of 1 million of MSC; Other: Intravenous administration of placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT02290886

CeTMAd/ELA/2011

Details and patient eligibility

About

A multicenter phase I/II Clinical trial,randomized, controlled with placebo, triple blind to evaluate the safety of the intravenous administration of 3 doses of autologous mesenchymal stem cells cells from adipose tissue in patients with Amyotrophic Lateral Sclerosis (ALS).

Full description

A multicenter phase I/II Clinical trial,randomized, controlled with placebo, triple blind to evaluate the safety of the intravenous administration of 3 doses of autologous mesenchymal stem cells cells from adipose tissue in patients with Amyotrophic Lateral Sclerosis (ALS).

40 patients will be enrolled and will be randomized into one of the following 4 arms:

• 10 patients in the control group (placebo)
• 10 patients received a dose of 1 million MSC / kg
• 10 patients received a dose of 2 million MSC / kg.
• 10 patients received a dose of 4 million MSC / kg

The follow-up phase of each patient from the cell infusion/placebo will be 6 months.

At the time that each patient completed the follow-up period (i.e., 6 months after the infusion of the cellular product or placebo), the blind will be open, and patients who have been assigned to the control group, will receive the cell product as secondary treatment. These patients will be randomized to receive each of the doses used in the first phase. From this point, they begin a second period of follow up of 6 months.

In addition, after 6 months of MSC infusion, every patient will continue in an open extension study for 36 months to assess the safety of MSC.

Enrollment: 52 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Women and males over 18-year-old.
2. Good understanding of the protocol and aptitude to grant the informed assent.
3. Diagnosis of sporadic ALS, with diagnosis of certainty, that is to say, definite or probable, in agreement with the criteria of "El Escorial", of the World Federation of Neurology.
4. Forced vital capacity of at least 50 % of the one that would correspond to them for sex, height and age.
5. More than 6 and less than 36 months of evolution of the disease (from the beginning of the symptoms).
6. Possibility of obtaining, at least, 50gr of adipose tissue.
7. Treatment with riluzole, for at least, a month before the inclusion.

Exclusion criteria

1. Any concomitant disease that under investigator's criteria could concern the measures of the clinical variables of the trial (hepatic, renal or cardiac insufficiency, diabetes mellitus, etc).
2. Previous therapy with stem cells.
3. Participation in another clinical trial during 3 months previous to the entry in this trial.
4. Any disease lymphoproliferative
5. Tracheostomy and /or gastrostomy.
6. Haemophilia, diathesis hemorrhagic or anticoagulative current therapy.
7. Hypersensitivity known to the bovine foetal whey or the gentamicin.
8. Medical precedents of infection of the HIV or any serious condition of immunocompromised.
9. Positive HBV or HCV serology
10. Levels of creatinine in whey > 3.0 in subjects not submitted to haemodialysis.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Single Group Assignment

Masking: Triple Blind

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52 participants in 4 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

Intravenous administration of placebo

Treatment:

Other: Intravenous administration of placebo

1 million of MSC

Experimental group

Description:

Intravenous administration of 1 million of MSC/ kg

Treatment:

Drug: Intravenous administration of 1 million of MSC

2 million of MSC

Experimental group

Description:

Intravenous administration of 2 million of MSC/ kg

Treatment:

Drug: Intravenous administration of 2 million of MSC

4 million of MSC

Experimental group

Description:

Intravenous administration of 4 million of MSC/ kg

Treatment:

Drug: Intravenous administration of 4 million of MSC