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A Dose-escalation Safety Trial for Intrathecal Autologous Mesenchymal Stem Cell Therapy in Amyotrophic Lateral Sclerosis

Phase 1
18 Years
Open (Enrolling)
Amyotrophic Lateral Sclerosis

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Trial Information

A Dose-escalation Safety Trial for Intrathecal Autologous Mesenchymal Stem Cell Therapy in Amyotrophic Lateral Sclerosis

The primary objective of this study is to determine the safety of intrathecal delivery of
autologous mesenchymal stem cells (MSCs) to the cerebrospinal fluid (CSF) of patients with
ALS using a dose-escalation study. The trial will include 25 adult, non-ventilator-dependent
patients with clinically definite amyotrophic lateral sclerosis (ALS). Cells will be
isolated from adipose tissue, expanded ex vivo and then, after ~8 weeks, intrathecal (IT)
autologous delivery of MSCs will be performed. There will be 5 treatment groups of up to 5
patients each. Groups 1, 2, and 4 will receive a single dose of cells. Groups 3 and 5 will
receive 2 doses of cells separated by 1 month. Groups will be completed sequentially so that
patients will not be enrolled into the next treatment group until at least 3 patients in the
preceding group have completed the treatment and 1 month of additional observation without
significant toxicity. All patients will be followed on a regular basis until death or for a
minimum of 2 years after completion of the final infusion. Initial clinical follow-up will
be weekly with scheduled blood, CSF and magnetic resonance imaging (MRI) evaluations. After
1 month, patients will have clinical evaluations at 3 month intervals, or earlier if
indicated by clinical status.

Inclusion Criteria:

- All patients must have clinically-defined ALS as defined by the World Federation of
Neurology criteria

- Age greater than 18 years

- If female, must be post-menopausal or had a hysterectomy

- Permanent resident or citizen of the United States

- History of a chronic onset of a progressive motor weakness of less than two years

- Able to comply with protocol requirements, including MRI testing

- Can provide written informed consent

Exclusion Criteria:

- Any clinically significant medical condition (e.g., within six months of baseline,
had myocardial infarction, angina pectoris, and/or congestive heart failure) that, in
the opinion of the investigator, would compromise the safety of patient.

- Autoimmunity, including Crohn's disease, rheumatoid arthritis, psoriasis

- Malignancy including melanoma with the exception of localized skin cancers (with no
evidence of metastasis, significant invasion, or re-occurrence within three years of
baseline). Any other malignancy will not be allowed.

- Active systemic or local infection near the lumbar puncture site

- Other active systemic disease as defined by laboratory abnormalities

- Use of herbal medications or other unapproved drugs

- Enrolled in an investigational drug trial within 30 days of baseline visit

- Kokmen Short Test of Mental Status score <32

- Beck's Depression Inventory score >18

- Presence of a tracheostomy

- Ventilator dependent

Type of Study:


Study Design:

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

Outcome Measure:

Number of patients with dose-limiting toxicities

Outcome Time Frame:

2 years after completion of the final infusion

Safety Issue:



United States: Food and Drug Administration

Study ID:




Start Date:

May 2012

Completion Date:

May 2014

Related Keywords:

  • Amyotrophic Lateral Sclerosis
  • Amyotrophic Lateral Sclerosis
  • Sclerosis
  • Motor Neuron Disease



Mayo ClinicRochester, Minnesota  55905