NCT04821479

Repeated Mesenchymal Stem Cell Injections in ALS

Completed Phase 1, PHASE2 Last updated 29 March 2021

What this trial tests

Phase 1, PHASE2 trial testing Mesenchymal stem cells (MSC) in Amyotrophic Lateral Sclerosis in 20 participants. Completed in 31 December 2020.

Timeline

1 January 2016

Primary endpoint
30 June 2020

31 December 2020

Quick facts

Lead sponsor	Hadassah Medical Organization
Phase	Phase 1, PHASE2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	20
Start date	1 January 2016
Primary completion	30 June 2020
Estimated completion	31 December 2020

Drugs / interventions tested

• Mesenchymal stem cells (MSC) — full drug profile →

Conditions studied

• Amyotrophic Lateral Sclerosis — all drugs for Amyotrophic Lateral Sclerosis →

Sponsor

Hadassah Medical Organization — full company profile →

Who can join

Adults 18 to 65, any sex, with Amyotrophic Lateral Sclerosis. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

An open-label, single-center clinical trial to evaluate the safety and efficacy of repeated intrathecal administrations of autologous bone marrow derived mesenchyme stem cells in ALS patients. The study includes 20 subjects (age: 20-70) with definite diagnosis of ALS and ALS-FRS-R score of at least 20 and disease-duration of less than 3 years. The treatment protocol includes four intrathecal injections of MSC, at intervals of 3 months between the injections.

The primary endpoints are safety and tolerability. Several efficacy measures are assessed as secondary endpoints.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Mesenchymal stem cell secretome and extracellular vesicles for neurodegenerative diseases: Risk-benefit profile and next steps for the market access.
   Giovannelli L, Bari E, Jommi C, Tartara F, et al · · 2023 · cited 81× · PMID 37456581 · DOI 10.1016/j.bioactmat.2023.06.013
2. The Use of Stem Cells as a Potential Treatment Method for Selected Neurodegenerative Diseases: Review.
   Cecerska-Heryć E, Pękała M, Serwin N, Gliźniewicz M, et al · · 2023 · cited 39× · PMID 37027074 · DOI 10.1007/s10571-023-01344-6
3. Therapeutic utility of mesenchymal stromal cell (MSC)-based approaches in chronic neurodegeneration: a glimpse into underlying mechanisms, current status, and prospects.
   Rahbaran M, Zekiy AO, Bahramali M, Jahangir M, et al · · 2022 · cited 38× · PMID 35842587 · DOI 10.1186/s11658-022-00359-z
4. Translational Approaches for Brain Delivery of Biologics via Cerebrospinal Fluid.
   Sadekar SS, Bowen M, Cai H, Jamalian S, et al · · 2022 · cited 27× · PMID 35064573 · DOI 10.1002/cpt.2531
5. Pathogenic Role of Adipose Tissue-Derived Mesenchymal Stem Cells in Obesity and Obesity-Related Inflammatory Diseases.
   Pestel J, Blangero F, Eljaafari A. · · 2023 · cited 19× · PMID 36766689 · DOI 10.3390/cells12030348
6. Neural Cells for Neurodegenerative Diseases in Clinical Trials.
   Fan Y, Goh ELK, Chan JKY. · · 2023 · cited 17× · PMID 37487111 · DOI 10.1093/stcltm/szad041
7. Current Status of Mesenchymal Stem/Stromal Cells for Treatment of Neurological Diseases.
   Soares MBP, Gonçalves RGJ, Vasques JF, da Silva-Junior AJ, et al · · 2022 · cited 16× · PMID 35782379 · DOI 10.3389/fnmol.2022.883378
8. Recent advances in stem cell therapy: efficacy, ethics, safety concerns, and future directions focusing on neurodegenerative disorders - a review.
   Khandia R, Gurjar P, Priyanka, Romashchenko V, et al · · 2024 · cited 13× · PMID 39705668 · DOI 10.1097/js9.0000000000001609