Study of Two Intrathecal Doses of Autologous Mesenchymal Stem Cells for Amyotrophic Lateral Sclerosis

AMYOTROPHIC LATERAL SCLEROSIS Clinical Trial

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02917681
• Other study ID #: 401922/2014-6
• Status: Recruiting
• Phase: Phase 1/Phase 2
• First received: September 26, 2016
• Last updated: September 27, 2016
• Start date: September 2016
• Est. completion date: August 2019

Study information

• Verified date: September 2016
• Source: University of Sao Paulo General Hospital
• Contact: Fabrício Castro Borba, MD
• Phone: 551130617460
• Email: contato[@]projetoelabrasil.com.br
• Is FDA regulated: No
• Health authority: Brazil: Ministry of Health
• Study type: Interventional

Clinical Trial Summary

The study aims to evaluate primarily safety of two injections of autologous mesenchymal stem cells in Amyotrophic Lateral Sclerosis patients. Secondary outcomes of efficacy will also be evaluated

Description:

The study is an open-label, unmasked, uncontrolled phase I and II trial to evaluate safety and efficacy of two injections of autologous mesenchymal stem cells (MSC) in Amyotrophic Lateral Sclerosis (ALS) patients. Patients will be recruited trough a web-based registration system, open for all ALS Brazilian patients. The patients will be enrolled after in-person screening at the University of Sao Paulo Medical School Clinics Hospital and inclusion criteria fulfilled. There will be 9 national calls for registration, two months apart each. The enrolled patients will be followed for 3 months before bone marrow aspiration (BMA). Patients will receive 2 intrathecal MSC injections, 1 and 2 months after BMA. The patients will be followed for 6 months after the interventions.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 28
• Est. completion date: August 2019
• Est. primary completion date: February 2019
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 70 Years

Eligibility

Inclusion Criteria:

1. Age between 18 and 70

2. Possible, probable or definite ALS following El Escorial Revised Criteria (Brooks, 2000)

3. ALSFRS-r =30 at enrollment

4. Forced Vital Capacity =65% of the height and weight standard

5. No-pregnancy agreement

6. Regional accessibility to the study site

7. Capability to give away agreed consensus

8. Patients will be followed at Academic Institutions at their hometown

Exclusion Criteria:

1. Previous cellular therapy

2. Incapacity to lay still during bone marrow aspirate or intrathecal MSC injections

3. Personal history of auto-Immune, myeloproliferative or myelodysplastic diseases, leukemia, lymphoma, whole-body irradiation, hip fracture, severe scoliosis or incapacity to undergo any of the study's proposed procedures

4. Any other disease that may interfere with the study

5. Any other neurological diseases

6. Aspartate or alanine aminotransferases elevated >3x normality upper limit

7. Serum creatinine >2x normality upper limit

8. Hepatitis B and C, HIV, HTLV I and II and syphilis

9. Immunosuppressant drug use within 6 weeks from the study's screening

10. Pregnancy or breast-feeding

11. Acquired or inherited Immunodeficiency

12. Participation in other clinical trials

13. Non-invasive ventilation, tracheostomy or diaphragm pacing

14. Substance abuse within one year and other unstable mental health diseases according to researcher's judgement

15. Gastrostomy or any alternative feeding means

16. Inappropriate in-vitro MSC expansion

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• AMYOTROPHIC LATERAL SCLEROSIS
• Motor Neuron Disease
• Sclerosis

Intervention

Other:
• Two intrathecal MSC injections

Locations

Country	Name	City	State
Brazil	University of Sao Paulo School of Medicine Clinics Hospital	Sao Paulo	SP

Sponsors (2)

Lead Sponsor	Collaborator
University of Sao Paulo General Hospital	Pontifícia Universidade Católica do Paraná

Country where clinical trial is conducted

Brazil, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety	Clinical and laboratory monitoring of possible reactions to intrathecal MSC delivery	10 months	Yes
Secondary	Change in Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R)	Specific scale for assessing ALS disease progression	10 months	No
Secondary	Change in Handheld dynamometry		10 months	No
Secondary	Change in Electrical Impedance Myography		10 months	No
Secondary	Change in Forced Vital Capacity (FVC)		10 months	No
Secondary	Change in Isometric Strength		10 months	No
Secondary	Changes in morphometric parameters of MRI of encephalon and spinal cord		10 months	No