NCT02193893

Brief Summary

The purpose of this study is to test the safety and effectiveness of an autologous bone marrow-derived stem/progenitor cells infusion in the subjects with diagnosed amyotrophic lateral sclerosis.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
50

participants targeted

Target at P50-P75 for phase_1

Timeline
Completed

Started Jan 2010

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2010

Completed
4.5 years until next milestone

First Submitted

Initial submission to the registry

July 16, 2014

Completed
2 days until next milestone

First Posted

Study publicly available on registry

July 18, 2014

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2017

Completed
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2017

Completed
Last Updated

July 18, 2014

Status Verified

July 1, 2014

Enrollment Period

7.5 years

First QC Date

July 16, 2014

Last Update Submit

July 17, 2014

Conditions

Amyotrophic Lateral Sclerosis

Keywords

cell-based therapyautologous bone marrow-derived stem/progenitor cellsstem cell infusion, stem cell transplantationbiological therapy with neurotrophic factorsbioactive neurotrophic factors

Outcome Measures

Primary Outcomes (1)

  • Safety of autologous bone marrow stem/progenitor cell infusion in enrolled patients

    Confirm the safety of autologous bone marrow stem/progenitor cell infusion in enrolled patients by repeated follow-up over one year with clinical and laboratory evaluations.

    1 year

Secondary Outcomes (1)

  • Efficacy of autologous bone marrow stem/progenitor cell infusion in enrolled patients.

    1 year

Study Arms (2)

Stem/progenitor cells transplantation.

ACTIVE COMPARATOR

Intervention: Biological: Cell-based therapeutics Autologous bone marrow-derived stem/progenitor cells will be transplanted intrathecally (via a standard lumbar puncture) into early vs. progressive ALS subjects.

Other: Biological: Cell-based therapeutics

Standard treatment of ALS

SHAM COMPARATOR

Symptomatic treatment of ALS without biologic cell-based treatment

Other: Symptomatic treatment of ALS

Interventions

Biological: Cell-based therapeuticsOTHER

Human autologous bone marrow-derived stem/progenitor cell transplantation in ALS patients.

Stem/progenitor cells transplantation.
Symptomatic treatment of ALSOTHER

Symptomatic neurological treatment of ALS

Standard treatment of ALS

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • diagnosis of the ALS disease before the cell transplantation (diagnose established following the El Escorial criteria for definite ALS)
  • good understanding of the protocol and willingness to consent
  • patient is mentally intact and psychologically stable
  • signed informed consent

You may not qualify if:

  • Concomitant of other systemic disease or diseases:
  • inflammation (high protein or lymphocytosis in the CSF), active infections.
  • diabetes,
  • cardio-vascular disorders,
  • cancer,
  • autoimmune diseases
  • renal failure,
  • impaired hepatic function.
  • subject is a respiratory dependent.
  • subject unwilling or unable to comply with the requirements of the protocol.
  • patient has been treated previously with any cellular therapy.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Department of Neurology of Pomeranian Medical University in Szczecin

Szczecin, Poland, 71-252, Poland

Location

Related Publications (1)

  • Pawlukowska W, Baumert B, Golab-Janowska M, Meller A, Machowska-Sempruch K, Welnicka A, Paczkowska E, Rotter I, Machalinski B, Nowacki P. Comparative assessment and monitoring of deterioration of articulatory organs using subjective and objective tools among patients with amyotrophic lateral sclerosis. BMC Neurol. 2019 Oct 19;19(1):241. doi: 10.1186/s12883-019-1484-2.

    PMID: 31629403DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesTDP-43 ProteinopathiesNeuromuscular DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Boguslaw Machalinski, MD, PhD

    Pomeranian Medical University Szczecin

    STUDY DIRECTOR

  • Przemyslaw Nowacki, MD, PhD

    Pomeranian Medical University Szczecin

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator: Boguslaw Machalinski, Professor and Chief, Department of General Pathology, Pomeranian Medical University, Pomeranian Medical University Szczecin

Study Record Dates

First Submitted

July 16, 2014

First Posted

July 18, 2014

Study Start

January 1, 2010

Primary Completion

July 1, 2017

Study Completion

December 1, 2017

Last Updated

July 18, 2014

Record last verified: 2014-07

Locations

PL(1)