NCT03067857

Brief Summary

Herein, the investigators study the safety and efficacy of transplanting purified autologous bone marrow-derived stem cells transplanted via the intrathecal route by interventional radiology and the intravenous route.

Trial Health

35
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
40

participants targeted

Target at P50-P75 for phase_1

Timeline
Completed

Started Sep 2016

Typical duration for phase_1

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

September 1, 2016

Completed
6 months until next milestone

First Submitted

Initial submission to the registry

February 25, 2017

Completed
4 days until next milestone

First Posted

Study publicly available on registry

March 1, 2017

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2019

Completed
7 months until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2019

Completed
Last Updated

June 6, 2018

Status Verified

June 1, 2018

Enrollment Period

2.3 years

First QC Date

February 25, 2017

Last Update Submit

June 4, 2018

Conditions

Motor Neuron DiseaseAmyotrophic Lateral SclerosisPrimary Lateral SclerosisProgressive Muscular AtrophyProgressive Bulbar Palsies

Outcome Measures

Primary Outcomes (1)

  • Changes in Functional Rating Scale - via questionnaire

    6 months

Secondary Outcomes (3)

  • Change in Appel Scale

    4 months

  • Change in SF-36 (The Short Form (36) Health Survey is a 36 Item)

    4 months

  • Change in Forced Vital Capacity (FVC)

    4 months

Study Arms (1)

Stem Cells

EXPERIMENTAL

Intravenous and Intrathecal thecal transplantation via interventional radiology of purified autologous bone-marrow derived specific stem cell populations.

Biological: Stem Cells

Interventions

Stem CellsBIOLOGICAL

Autologous bone marrow-derived stem cell populations injected via the intravenous and intrathecal routes.

Stem Cells

Eligibility Criteria

Age24 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Patients age 24-20 years old
  • Patients with the diagnosis of definite Motor Neuron Disease

You may not qualify if:

  • HIV/HBV/HCV
  • Malignancies
  • Bleeding
  • Pneumonia
  • Renal failure
  • liver dysfunction
  • Severe anemia (hb \< 8)
  • Bone Marrow diseases
  • Pregnancy and lactation
  • Acute medical conditions/infections such as respiratory infections
  • ventilatory support

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Motor Neuron DiseaseAmyotrophic Lateral SclerosisMuscular Atrophy, SpinalBulbar Palsy, Progressive

Condition Hierarchy (Ancestors)

Neurodegenerative DiseasesNervous System DiseasesNeuromuscular DiseasesSpinal Cord DiseasesCentral Nervous System DiseasesTDP-43 ProteinopathiesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 25, 2017

First Posted

March 1, 2017

Study Start

September 1, 2016

Primary Completion

January 1, 2019

Study Completion

August 1, 2019

Last Updated

June 6, 2018

Record last verified: 2018-06