Study of Two Intrathecal Doses of Autologous Mesenchymal... | NCT02917681

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

NCT02917681

Sponsor

University of Sao Paulo General Hospital

Status

Unknown status

Last Update Posted

Sep 28, 2016Estimated

Enrollment

28Estimated

Phase

Phase 1Phase 2

Conditions

AMYOTROPHIC LATERAL SCLEROSIS

Interventions

Two intrathecal MSC injections

Countries

Brazil

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Sep 2016

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Sep 2016

Primary Completion Date

Feb 2019Estimated

Completion Date

Aug 2019Estimated

First Submitted Date

Sep 26, 2016

First Submission Date that Met QC Criteria

Sep 27, 2016

First Posted Date

Sep 28, 2016Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Sep 27, 2016

Last Update Posted Date

Sep 28, 2016Estimated

Name	Class
Pontifícia Universidade Católica do Paraná	OTHER

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief 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

Detailed 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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

28Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
MSC injection	Experimental	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.	Other: Two intrathecal MSC injections

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Two intrathecal MSC injections	Other		MSC injection

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Safety	Clinical and laboratory monitoring of possible reactions to intrathecal MSC delivery	10 months

Secondary Outcomes

Measure	Description	Time Frame
Change in Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R)	Specific scale for assessing ALS disease progression	10 months
Change in Handheld dynamometry		10 months

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Age between 18 and 70
Possible, probable or definite ALS following El Escorial Revised Criteria (Brooks, 2000)
ALSFRS-r ≥30 at enrollment
Forced Vital Capacity ≥65% of the height and weight standard
No-pregnancy agreement
Regional accessibility to the study site
Capability to give away agreed consensus
Patients will be followed at Academic Institutions at their hometown

Exclusion Criteria:

Previous cellular therapy
Incapacity to lay still during bone marrow aspirate or intrathecal MSC injections
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
Any other disease that may interfere with the study
Any other neurological diseases
Aspartate or alanine aminotransferases elevated >3x normality upper limit
Serum creatinine >2x normality upper limit
Hepatitis B and C, HIV, HTLV I and II and syphilis
Immunosuppressant drug use within 6 weeks from the study's screening
Pregnancy or breast-feeding
Acquired or inherited Immunodeficiency
Participation in other clinical trials
Non-invasive ventilation, tracheostomy or diaphragm pacing
Substance abuse within one year and other unstable mental health diseases according to researcher's judgement
Gastrostomy or any alternative feeding means
Inappropriate in-vitro MSC expansion

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

70 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Fabrício Castro Borba, MD	Contact	551130617460		contato@projetoelabrasil.com.br
Jéssica Ruivo Maximino, PhD	Contact	551130617460		contato@projetoelabrasil.com.br

Overall Officials

Name	Affiliation	Role
Gerson Chadi, MD, PhD	Full Professor	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
University of Sao Paulo School of Medicine Clinics Hospital	Recruiting	São Paulo	São Paulo	05403000	Brazil

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

ID	Term
D013118	Spinal Cord Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases
D016472	Motor Neuron Disease