Back to results

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

Primary Purpose

Amyotrophic Lateral Sclerosis

Status

Completed

Phase

Phase 1

Locations

Spain

Study Type

Interventional

Intervention

Intravenous administration of placebo

Intravenous administration of 1 million of MSC

Intravenous administration of 2 million of MSC

Intravenous administration of 4 million of MSC

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring safety evaluation, intravenous administration, 3 doses of autologous MSC, ALS

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Women and males over 18-year-old.
Good understanding of the protocol and aptitude to grant the informed assent.
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.
Forced vital capacity of at least 50 % of the one that would correspond to them for sex, height and age.
More than 6 and less than 36 months of evolution of the disease (from the beginning of the symptoms).
Possibility of obtaining, at least, 50gr of adipose tissue.
Treatment with riluzole, for at least, a month before the inclusion.

Exclusion Criteria:

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).
Previous therapy with stem cells.
Participation in another clinical trial during 3 months previous to the entry in this trial.
Any disease lymphoproliferative
Tracheostomy and /or gastrostomy.
Haemophilia, diathesis hemorrhagic or anticoagulative current therapy.
Hypersensitivity known to the bovine foetal whey or the gentamicin.
Medical precedents of infection of the HIV or any serious condition of immunocompromised.
Positive HBV or HCV serology
Levels of creatinine in whey > 3.0 in subjects not submitted to haemodialysis.

Sites / Locations

Hospital Regional Universitario Reina Sofía
Hospital Regional Universitario de Málaga
Hospital Universitario Virgen Macarena, Servicio de Neurología
Hospital Universitario Virgen del Rocío

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Placebo Comparator

Experimental

Arm Label

Placebo

1 million of MSC

2 million of MSC

4 million of MSC

Arm Description

Intravenous administration of placebo

Intravenous administration of 1 million of MSC/ kg

Intravenous administration of 2 million of MSC/ kg

Intravenous administration of 4 million of MSC/ kg

Outcomes

Primary Outcome Measures

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)

Complications in the place of the infusion

To evaluate the safety of the intravenous administration of 3 doses of autologous

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

Secondary Outcome Measures

Changes in the progression of the disease (modifications in the scale of functionality of the ALS)

Changes in the degree of muscular force

Changes in the vital forced capacity

Changes of the muscular mass estimated by Nuclear Magnetic Resonance (NMR) of the upper and low extremities

Changes in neurophysiological parameters and of quality of life

Need and time to tracheotomy or permanent assisted ventilation

Full Information

NCT ID

NCT02290886

First Posted

October 9, 2014

Last Updated

April 5, 2022

Sponsor

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

Collaborators

Iniciativa Andaluza en Terapias Avanzadas

1. Study Identification

Unique Protocol Identification Number

NCT02290886

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2022

Overall Recruitment Status

Completed

Study Start Date

July 2014 (Actual)

Primary Completion Date

March 2, 2022 (Actual)

Study Completion Date

March 2, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

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

Collaborators

Iniciativa Andaluza en Terapias Avanzadas

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

Detailed 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Amyotrophic Lateral Sclerosis

Keywords

safety evaluation, intravenous administration, 3 doses of autologous MSC, ALS

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

Masking

ParticipantCare ProviderInvestigator

Allocation

Randomized

Enrollment

52 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Intravenous administration of placebo

Arm Title

1 million of MSC

Arm Type

Experimental

Arm Description

Intravenous administration of 1 million of MSC/ kg

Arm Title

2 million of MSC

Arm Type

Experimental

Arm Description

Intravenous administration of 2 million of MSC/ kg

Arm Title

4 million of MSC

Arm Type

Experimental

Arm Description

Intravenous administration of 4 million of MSC/ kg

Intervention Type

Other

Intervention Name(s)

Intravenous administration of placebo

Intervention Type

Drug

Intervention Name(s)

Intravenous administration of 1 million of MSC

Intervention Type

Drug

Intervention Name(s)

Intravenous administration of 2 million of MSC

Intervention Type

Drug

Intervention Name(s)

Intravenous administration of 4 million of MSC

Primary Outcome Measure Information:

Title

Number of adverse serious unexpected reactions or not, attributable to the treatment (SUSSARs or SAE)

Description

Time Frame

6 months

Title

Complications in the place of the infusion

Description

To evaluate the safety of the intravenous administration of 3 doses of autologous

Time Frame

6 months

Title

Appearance of a new neurological effect not attributable to the natural progression of this pathology

Description

To evaluate the safety of the intravenous administration of 3 doses of autologous

Time Frame

6 months

Secondary Outcome Measure Information:

Title

Changes in the progression of the disease (modifications in the scale of functionality of the ALS)

Time Frame

6 months

Title

Changes in the degree of muscular force

Time Frame

6 months

Title

Changes in the vital forced capacity

Time Frame

6 months

Title

Changes of the muscular mass estimated by Nuclear Magnetic Resonance (NMR) of the upper and low extremities

Time Frame

6 months

Title

Changes in neurophysiological parameters and of quality of life

Time Frame

6 months

Title

Need and time to tracheotomy or permanent assisted ventilation

Time Frame

6 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Women and males over 18-year-old. Good understanding of the protocol and aptitude to grant the informed assent. 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. Forced vital capacity of at least 50 % of the one that would correspond to them for sex, height and age. More than 6 and less than 36 months of evolution of the disease (from the beginning of the symptoms). Possibility of obtaining, at least, 50gr of adipose tissue. Treatment with riluzole, for at least, a month before the inclusion. Exclusion Criteria: 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). Previous therapy with stem cells. Participation in another clinical trial during 3 months previous to the entry in this trial. Any disease lymphoproliferative Tracheostomy and /or gastrostomy. Haemophilia, diathesis hemorrhagic or anticoagulative current therapy. Hypersensitivity known to the bovine foetal whey or the gentamicin. Medical precedents of infection of the HIV or any serious condition of immunocompromised. Positive HBV or HCV serology Levels of creatinine in whey > 3.0 in subjects not submitted to haemodialysis.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Óscar Fernández, MD

Organizational Affiliation

Hospital Regional U. de Málaga

Official's Role

Study Chair

Facility Information:

Facility Name

Hospital Regional Universitario Reina Sofía

City

Cordoba

ZIP/Postal Code

14004

Country

Spain

Facility Name

Hospital Regional Universitario de Málaga

City

Málaga

ZIP/Postal Code

29010

Country

Spain

Facility Name

Hospital Universitario Virgen Macarena, Servicio de Neurología

City

Seville

ZIP/Postal Code

41009

Country

Spain

Facility Name

Hospital Universitario Virgen del Rocío

City

Seville

ZIP/Postal Code

41013

Country

Spain

12. IPD Sharing Statement

Citations:

PubMed Identifier

28674982

Citation

Ilieva H, Maragakis NJ. Motoneuron Disease: Clinical. Adv Neurobiol. 2017;15:191-210. doi: 10.1007/978-3-319-57193-5_7.

Results Reference

derived

Learn more about this trial

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

We'll reach out to this number within 24 hrs