NCT01758510

An Open-label, Phase 1 Trial for Safety Study of HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell("HYNR-CS-Allo Inj") Treatment in Amyotrophic Lateral Sclerosis(ALS)

Completed Phase 1 Last updated 15 May 2019

What this trial tests

Phase 1 trial testing HYNR-CS-Allo in Amyotrophic Lateral Sclerosis in 6 participants. Completed in 10 April 2017.

Timeline

1 December 2012

Primary endpoint
10 April 2017

10 April 2017

Quick facts

Lead sponsor	Hanyang University Seoul Hospital
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	6
Start date	1 December 2012
Primary completion	10 April 2017
Estimated completion	10 April 2017
Sites	1 location across South Korea

Drugs / interventions tested

• HYNR-CS-Allo

Conditions studied

• Amyotrophic Lateral Sclerosis — all drugs for Amyotrophic Lateral Sclerosis →
• Motor Neuron Disease — all drugs for Motor Neuron Disease →
• Neuromuscular Disease — all drugs for Neuromuscular Disease →
• Neurodegenerative Disease — all drugs for Neurodegenerative Disease →

Sponsor

Hanyang University Seoul Hospital

Who can join

Adults 25 to 80, any sex, with Amyotrophic Lateral Sclerosis or Motor Neuron Disease. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Serious Adverse Events(SAE)
  Time frame: 6 months
  The Incidence of any treatment related serious adverse events(SAE)

Sponsor's own description

The purpose of this study is to evaluate the safety of HLA-haplo matched Allogenic Bone Marrow Derived stem cells("HYNR-CS-Allo inj"), through intrathecal delivery for the treatment in patients with amyotrophic lateral sclerosis(ALS).

This study is an open label, dose up and down study using the 3+3 design to assess the safety of HLA-haplo matched Allogenic Bone Marrow Derived stem cells("HYNR-CS-Allo inj")

Publications & conference data

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

1. Stem cell transplantation in neurological diseases: improving effectiveness in animal models.
   Adami R, Scesa G, Bottai D. · · 2014 · cited 27× · PMID 25364724 · DOI 10.3389/fcell.2014.00017
2. Stem cell transplantation for amyotrophic lateral sclerosis: therapeutic potential and perspectives on clinical translation.
   Faravelli I, Riboldi G, Nizzardo M, Simone C, et al · · 2014 · cited 23× · PMID 24699704 · DOI 10.1007/s00018-014-1613-4
3. Neural Cells for Neurodegenerative Diseases in Clinical Trials.
   Fan Y, Goh ELK, Chan JKY. · · 2023 · cited 17× · PMID 37487111 · DOI 10.1093/stcltm/szad041
4. Neuroprotective Potential of Cell-Based Therapies in ALS: From Bench to Bedside.
   Forostyak S, Sykova E. · · 2017 · cited 17× · PMID 29114200 · DOI 10.3389/fnins.2017.00591
5. 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
6. Patching Up the Permeability: The Role of Stem Cells in Lessening Neurovascular Damage in Amyotrophic Lateral Sclerosis.
   Monsour M, Garbuzova-Davis S, Borlongan CV. · · 2022 · cited 5× · PMID 36181767 · DOI 10.1093/stcltm/szac072
7. Underregistration and Underreporting of Stem Cell Clinical Trials in Neurological Disorders.
   Lee TE, Kim A, Jang M, Jeon B. · · 2018 · cited 4× · PMID 29629526 · DOI 10.3988/jcn.2018.14.2.215
8. Harnessing nanotechnology for stem-cell therapies: revolutionizing neurodegenerative disorder treatments - a state-of-the-art update.
   Chengebroyen N, Seelan A, Yoonus Thajudeen K, Alshehri SA, et al · · 2025 · cited 1× · PMID 40771915 · DOI 10.3389/fphar.2025.1630475