Safety and Efficacy of Stem Cell Therapy of Motor Neuron Disease.

• Register: EUCTR
• Last refreshed on: 4 September 2017
• Main ID: EUCTR2011-000362-35-CZ
• Date of registration: 05/04/2011
• Prospective Registration: Yes
• Primary sponsor: Bioinova, s.r.o.
• Public title: Safety and Efficacy of Stem Cell Therapy of Motor Neuron Disease.
• Scientific title: A Prospective, Non-randomized, Open Label Study to Assess the Safety and the Efficacy of Autologous Multipotent Mesenchymal Stem Cells in the Treatment of Amyotrophic Lateral Sclerosis. - AMSC in ALS
• Date of first enrolment: 29/03/2012
• Target sample size: 30
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-000362-35
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: Open: Single blind: Double blind: Parallel group: Cross over: Other: If controlled, specify comparator, Other Medicinial Product: Placebo: Other:
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Czech Republic

Contacts

• Name: Headquarters
• Affiliation: Bioinova, s.r.o.

• Name: Headquarters
• Affiliation: Bioinova, s.r.o.

Key inclusion & exclusion criteria

Inclusion criteria:
To participate in this trial, patients will have to meet following criteria:
1. established diagnosis of definite ALS according to El Escorial kriteria and data available from detailed neurologic observation (including ALSFRS, Norris scale, spirometry, brain and spinal cord MRI) for at least 6 months prior to the study entrance,
2. riluzole naive or at stable dose for at least 2 months,
3. patients between 18–65 years, both sexes, life expectancy more than 2 years,
4. patients able to provide written informed consent.


Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
Patients meeting any of the following criteria will be excluded from the participation in this study:
1. FVC less than 70% ,
2. in case of primary bulbar paralysis less than 15 points on Norris bulbar scale,
3. less than 15 points on Norris spinal scale,
4. pregnancy, breastfeeding
5. coagulopathy,
6. skin infection at the site of bone marrow aspiration or application of the cell product,
7. gastrostomy,
8. any significant medical condition that would compromise the safety of the patient (e.g. recent myocardial infarction, congestive heart failure, renal failure, liver failure, cancer, systemic infection, reccurent thromboembolic disease .....),
9. alcohol or drug abuse,
10. cancer,
11. women of childbearing potential not using effective contraception (established oral contraception, intrauterine device, ligation of the uterine tube) including proven contraceptive measures taken by their sexual partners,
12. fertile men not using proven contraceptive measures including effective contraception of their partner (established oral contraception, intrauterine device, ligation of the uterine tube).


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Amyotrophic lateral sclerosis
MedDRA version: 17.0 Level: PT Classification code 10002026 Term: Amyotrophic lateral sclerosis System Organ Class: 10029205 - Nervous system disorders

Intervention(s)

Product Name: Suspension of human autologous MSC 3P in 1,5 ml
Product Code: AMSC
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: Human autologous mesenchymal stem cells
Current Sponsor code: AMSC
Concentration unit: million organisms/ml million organisms/millilitre
Concentration type: range
Concentration number: 7-13

Primary Outcome(s)

Primary end point(s): 1) Complication at the site of intrathecal infusion
2) New neurologic deficit (i. e. meningism, paraplegia, urinary incontinence) not attributable to the natural progression of the disease.

Main Objective: Safety: To assess an absence of complications at the site of intrathecal infusion and no new neurologic deficit (i. e. meningism, paraplegia, urinary incontinence) not attributed to the natural progression of the disease.

Secondary Objective: Efficacy: To assess progress of the disease measured according to ALS functional rating scale (ALSFRS) and Norris scale and spirometry (FVC) after the AMSC treatment.

Timepoint(s) of evaluation of this end point: ad 1: at Visits IV, V, and VI (1 day to 3 months after treatment administration)
ad 2: at Visits IV, V, VI and IX (1 day to 12 months after treatment administration)

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: At 3, 6, 9, 12 and 18 months following treatment administration.

Secondary end point(s): ALS disease progression (assessment of functional deterioration milestones)

Secondary ID(s)

AMSC-ALS-001

Source(s) of Monetary Support

Bioinova, s.r.o.