Clinical trial NCT05003921: Safety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cell Intrathecal Injection for ALS
Official title: Safety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cell Intrathecal Injection for the Treatment of Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: SUSPENDED
Phase: PHASE1
Conditions: Amyotrophic Lateral Sclerosis
Keywords: Amyotrophic Lateral Sclerosis
ALS
Stem cells
Lead sponsor: The Foundation for Orthopaedics and Regenerative Medicine
Sponsor class: OTHER
First posted: 2021-08-13
Enrollment: 20
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This trial will study the safety and efficacy of intrathecal injection of cultured allogeneic adult umbilical cord derived mesenchymal stem cells for the treatment of amyotrophic lateral sclerosis

Detailed description: Amyotrophic lateral sclerosis is a devastating disease. There is evidence that mesenchymal stem cell treatment is safe and can improve the prognosis of afflicted patients. Patients with ALS will receive three intrathecal injections of cultured allogeneic adult umbilical cord derived mesenchymal stem cells. The dose for each injection will be 50 million cells and the injections will be at two-month intervals. Patients will be evaluated within one month pre treatment and at 1, 6, 12, 24, 36, and 48 months post treatment for safety and efficacy.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of Amyotrophic Lateral Sclerosis.
* Understanding and willingness to sign a written informed consent document

Exclusion Criteria:

* Active infection
* Active cancer
* Chronic multisystem organ failure
* Pregnancy
* Anticoagulation medicine use
* Clinically significant Abnormalities on pre-treatment laboratory evaluation
* Medical condition that would (based on the opinion of the investigator) compromise patient's safety.
* Previous organ transplant
* Hypersensitivity to sulfur
* Continued drug abuse
* Pre-menopausal women not using contraception

Primary outcomes:
measure: Safety (adverse events)
timeFrame: Four year follow-up
Secondary outcomes:
measure: Efficacy: revised ALS functional rating scale (ALSFRS-R)
timeFrame: Four year follow-up
Other outcomes:
Locations:
city: St. John's
country: Antigua and Barbuda
facility: Medical Surgical Associates Center
Study officials:
affiliation: The Foundation for Orthopaedics and Regenerative Medicine
name: Chadwick Prodromos, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Cedarbaum JM, Stambler N, Malta E, Fuller C, Hilt D, Thurmond B, Nakanishi A. The ALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function. BDNF ALS Study Group (Phase III). J Neurol Sci. 1999 Oct 31;169(1-2):13-21. doi: 10.1016/s0022-510x(99)00210-5.
citation: Petrou P, Gothelf Y, Argov Z, Gotkine M, Levy YS, Kassis I, Vaknin-Dembinsky A, Ben-Hur T, Offen D, Abramsky O, Melamed E, Karussis D. Safety and Clinical Effects of Mesenchymal Stem Cells Secreting Neurotrophic Factor Transplantation in Patients With Amyotrophic Lateral Sclerosis: Results of Phase 1/2 and 2a Clinical Trials. JAMA Neurol. 2016 Mar;73(3):337-44. doi: 10.1001/jamaneurol.2015.4321.
citation: Barczewska M, Maksymowicz S, Zdolinska-Malinowska I, Siwek T, Grudniak M. Umbilical Cord Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis: an Original Study. Stem Cell Rev Rep. 2020 Oct;16(5):922-932. doi: 10.1007/s12015-020-10016-7.
citation: Oh KW, Noh MY, Kwon MS, Kim HY, Oh SI, Park J, Kim HJ, Ki CS, Kim SH. Repeated Intrathecal Mesenchymal Stem Cells for Amyotrophic Lateral Sclerosis. Ann Neurol. 2018 Sep;84(3):361-373. doi: 10.1002/ana.25302. Epub 2018 Aug 31.