Evaluation the Efficacy and Safety of Mutiple Lenzumestrocel (Neuronata-R® Inj.) Treatment in Patients with ALS
- Conditions
- Amyotrophic Lateral Sclerosis
- Interventions
- Registration Number
- NCT04745299
- Lead Sponsor
- Corestemchemon, Inc.
- Brief Summary
ALSUMMIT is a double-blind, randomized, placebo-controlled, multi-center, parallel, phase III clinical trial to evaluate and confirm the efficacy and long-term safety of repeated Lenzumestrocel (Neuronata-R® inj.) treatment.
- Detailed Description
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder characterized by selective and progressive loss of motor neurons. Disease progression leads to death within 2-4 years, but there exists no definite treatment so far.
Based on phase I/II clinical trial(NCT01363401), twice intrathecal autologous bone marrow-derived mesenchymal stem cells (Lenzumestrocel) injections showed significant therapeutic benefit lasting at least six months with safety in patients with ALS.
Additionally, the switch from pro- to anti-inflammatory conditions, which was indicated from the inverse correlation between TGF-β1 and MCP-1 levels after Lenzumestrocel injections in the good responder, has been considered a plausible beneficial action mechanism.
This study is designed to investigate the following. First, to reconfirm and evaluate the long-term efficacy of twice injections (single cycle) of Lenzumestrocel, group 1 will receive a single cycle injection with a 26-day interval.
Second, to evaluate the long-term safety and efficacy of Lenzumestrocel repeated injections, group 2 will receive a single cycle injection a 26-day apart followed by three times injections every three-month interval.
Group 3 will receive comparator injections.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 115
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Single cycle administration group Placebo Comparator Study drug injections twice in a 26-day interval followed by three times comparator injections every three months. Multiple administation group Riluzole Study drug injections twice in a 26-day interval followed by repeated three times study drug injections every three months. Control group Placebo Comparator Comparator injections twice in a 26-day interval followed by three times comparator injections every three months. Single cycle administration group Riluzole Study drug injections twice in a 26-day interval followed by three times comparator injections every three months. Control group Riluzole Comparator injections twice in a 26-day interval followed by three times comparator injections every three months. Single cycle administration group Lenzumestrocel Study drug injections twice in a 26-day interval followed by three times comparator injections every three months. Multiple administation group Lenzumestrocel Study drug injections twice in a 26-day interval followed by repeated three times study drug injections every three months.
- Primary Outcome Measures
Name Time Method Joint rank scores (CAFS, Combined Assessment of Functional and Survival) at 12 months, and 6 months Joint rank score is derived from Combined Assessment of Function and Survival. Functional assessment is based on ALSFRS-R scores and survival assessment is based on the period from randomization to physical death. Joint rank score will be calculated with ALSFRS-R score data and survival. For each pairwise comparison, a study participant is assigned a score and then the summed scores are ranked for all participants.
The higher ranking, the higher score, and the lower ranking, the lower score. And the average rank score is then calculated for each treatment group. A higher mean rank score indicates that participants in that treatment group, on average, fared better.
1. The difference in joint rank scores between multiple administration group and control group at 12 months.
2. The difference in joint rank scores between single cycle administration group and control group at 6 months
- Secondary Outcome Measures
Name Time Method Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score at 12 months, 6 months ALSFRS-R is an instrument designed to evaluate functional status of subjects with amyotrophic lateral sclerosis (Lou Gehrig's disease), such as gross motor activity, fine motor activity, bulbar function and respiration function. It consists of 12 items; 3 items for mouth functions, 4 items for upper limb and overall fine motor functions, 2 items for lower limb functions and 3 items for respiration functions. Each item is evaluated in 5-point scale (0\~4). A higher score means a better functional status.
1. Change of ALSFRS-R score measured at baseline and 12 months (multiple administration group and control group)
2. Change of ALSFRS-R score measured at baseline and 6 months (single cycle administration and control group)Time to event at 12 months, 6 months Time to Event is defined as physical death, tracheostomy recognized as the point where disease progression functionally stops or chronic use of ventilator, whichever comes earlier. Chronic use of ventilator means that ventilator is used for more than 20 hours in a day and such use is continued for more than 30 days and that a subject in vegetative state requires full supports from other persons to maintain living.
1. Time to event for 12 months (multiple administration group and control group)
2. Time to event for 6 months (single cycle administration group and control group)Joint rank scores (CAFS, Combined Assessment of Functional and Survival) at 6 months Joint rank score is derived from Combined Assessment of Function and Survival. Functional assessment is based on ALSFRS-R scores and survival assessment is based on the period from randomization to physical death. Joint rank score will be calculated with ALSFRS-R score data and survival. For each pairwise comparison, a study participant is assigned a score and then the summed scores are ranked for all participants.
The higher ranking, the higher score, and the lower ranking, the lower score. And the average rank score is then calculated for each treatment group. A higher mean rank score indicates that participants in that treatment group, on average, fared better.
The difference in joint rank scores between multiple administration group and control group at 6 months.
Trial Locations
- Locations (5)
Seoul National University Hospital
🇰🇷Seoul, Korea, Republic of
Samsung Medical Center
🇰🇷Seoul, Korea, Republic of
Pusan National University Yangsan Hospital
🇰🇷Yangsan, Kyungsangnam-do, Korea, Republic of
Hanyang university hospital
🇰🇷Seoul, Korea, Republic of
Korea University Anam Hospital
🇰🇷Seoul, Korea, Republic of