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

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

A Multicenter Phase I/II Clinical Trial, Randomized, Controlled With Placebo, Triple Blind to Evaluate Safety, and Indications of Efficiency of the Intravenous Administration of the Therapy With 3 Doses of MSC in Patients With ASL Moderated to Severe

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02290886
• Other study ID #: CeTMAd/ELA/2011
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: July 2014
• Est. completion date: March 2, 2022

Study information

• Verified date: April 2022
• Source: Andalusian Network for Design and Translation of Advanced Therapies
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 52
• Est. completion date: March 2, 2022
• Est. primary completion date: March 2, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Other:
• Intravenous administration of placebo

Drug:
• Intravenous administration of 1 million of MSC

• Intravenous administration of 2 million of MSC

• Intravenous administration of 4 million of MSC

Locations

Country	Name	City	State
Spain	Hospital Regional Universitario Reina Sofía	Cordoba
Spain	Hospital Regional Universitario de Málaga	Málaga
Spain	Hospital Universitario Virgen del Rocío	Seville
Spain	Hospital Universitario Virgen Macarena, Servicio de Neurología	Seville

Sponsors (2)

Lead Sponsor	Collaborator
Andalusian Initiative for Advanced Therapies - Fundación Pública Andaluza Progreso y Salud	Iniciativa Andaluza en Terapias Avanzadas

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of adverse serious unexpected reactions or not, attributable to the treatment (SUSSARs or SAE)	To evaluate the safety of the intravenous administration of 3 doses of autologous mesenchymal stem cells (MSC) from adipose tissue in patients with Amyotrophic lateral Sclerosis (ALS) ABSENCE of: complications in the place of the infusion, appearance of a new neurological effect not attributable to the natural progression of this pathology and adverse serious unexpected reactions or not, attributable to the treatment (SUSSARs or SAE)	6 months
Primary	Complications in the place of the infusion	To evaluate the safety of the intravenous administration of 3 doses of autologous	6 months
Primary	Appearance of a new neurological effect not attributable to the natural progression of this pathology	To evaluate the safety of the intravenous administration of 3 doses of autologous	6 months
Secondary	Changes in the progression of the disease (modifications in the scale of functionality of the ALS)		6 months
Secondary	Changes in the degree of muscular force		6 months
Secondary	Changes in the vital forced capacity		6 months
Secondary	Changes of the muscular mass estimated by Nuclear Magnetic Resonance (NMR) of the upper and low extremities		6 months
Secondary	Changes in neurophysiological parameters and of quality of life		6 months
Secondary	Need and time to tracheotomy or permanent assisted ventilation		6 months