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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01609283
• Date of registration: 18/05/2012
• Prospective Registration: No
• Primary sponsor: Mayo Clinic
• Public title: A Dose-escalation Safety Trial for Intrathecal Autologous Mesenchymal Stem Cell Therapy in Amyotrophic Lateral Sclerosis
• Scientific title: A Dose-escalation Safety Trial for Intrathecal Autologous Mesenchymal Stem Cell Therapy in Amyotrophic Lateral Sclerosis
• Date of first enrolment: May 2012
• Target sample size: 27
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01609283
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Anthony Windebank, MD
• Affiliation: Mayo Clinic

Key inclusion & exclusion criteria

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 greater than one year,
but less than two years duration

- Must have vital capacity greater than 65% of predicated for age, gender, and body type

- 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Biological: autologous mesenchymal stem cells

Primary Outcome(s)

Number of patients with dose-limiting toxicities [Time Frame: baseline -2 years after completion of the final infusion]

Secondary Outcome(s)

Change in total nucleated cell count in cerebrospinal fluid (CSF) [Time Frame: baseline, 2 years after completion of the final infusion]

Number of patients with adverse events [Time Frame: baseline -2 years after completion of the final infusion]

Change in serum sedimentation rate [Time Frame: baseline, 2 years after completion of the final infusion]

Number of patients with presence of cancer cells in their cerebrospinal fluid (CSF) [Time Frame: baseline -2 years after completion of the final infusion]

Change in complete blood counts [Time Frame: baseline, 2 years after completion of the final infusion]

Change in C-reactive protein levels [Time Frame: baseline, 2 years after completion of the final infusion]

Change in protein level in cerebrospinal fluid (CSF) [Time Frame: baseline, 2 years after completion of the final infusion]

Secondary ID(s)

11-008415

Source(s) of Monetary Support

Please refer to primary and secondary sponsors