Amyotrophic Lateral Sclerosis (ALS), Autoantibody, Plasmapheresis
Conditions
Keywords
amyotrophic lateral sclerosis, anti-NRIP autoantibody, plasmapheresis
Brief summary
10-20% of patients with ALS have anti-NRIP autoantibody and the titer of anti-NRIP autoantibody is correlated with motor functional decline and mortality in ALS. The PALADIN2 clinical trial is a single arm study, which intends to enroll 20 ALS patients having anti-NRIP autoantibody in plasma. Patients will receive 3 courses of plasmapheresis per 3 months in order to maintain low concentration of anti-NRIP autoantibody in plasma. The study will follow up these patients for another 6 months after plasmapheresis. This project will potentially confirm the efficacy and safety of plasmapheresis for ALS patients having anti-NRIP autoantibody.
Interventions
DEVICEplasmapheresis
Using plasmapheresis to remove plasma anti-NRIP autoantibody; each patient receive 3 courses of plasmapheresis with 3 months apart
Sponsors
National Taiwan University Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
20 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* ALS patients above 20-year-old who have anti-NRIP autoantibody in plasma * Agree to receive plasmapheresis treatment * Agree to participate in the study and receive serial examinations
Exclusion criteria
* Under permanent ventilator support * Cannot receive plasmapheresis treatment or serial examinations * Under pregnancy * Blood fibrinogen level below 50 mg/dl * Belong to special subtype of ALS, such as primary lateral sclerosis, progressive muscular atrophy, flail arm syndrome, flail leg syndrome.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in disease progression rates measuring using ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised) | 12 months | Difference between \[(ALSFRS-R at month -3 - ALSFRS-R at month 0)/3\] and \[(ALSFRS-R at month 0 - ALSFRS-R at month 9)/9\]; Months -3 to 0 vs. Months 0 to 9; the higher above ratio means higher disease progression rate |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in disease progression rates measuring using ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised) | 15 months | Difference between \[(ALSFRS-R at month -3 - ALSFRS-R at month 0)/3\] and \[(ALSFRS-R at month 0 - ALSFRS-R at month 12)/12\]; Months -3 to 0 vs. Months 0 to 12; the higher above ratio means higher disease progression rate |
| Change in disease severity measuring using ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised) | 12 months | The ALSFRS-R at months 3, 6, 9,12 as compared to that at month 0; the lower the AFSFRS-R, the worse disease severity |
| Change in disease progression rates measuring using MRC (Medical Research Council score) mega-score | 15 months | Difference between \[(MRC mega-score at month -3 - MRC mega-score at month 0)/3\] and \[(MRC mega-score at month 0 - MRC mega-score at month 12)/12\]; Months -3 to 0 vs. Months 0 to 12; the higher above ratio means higher disease progression rate |
| Change in disease progression rates measuring using Grips force (lb) | 15 months | Description: Difference between \[(Grips force at month -3 - Grips force at month 0)/3\] and \[(Grips force at month 0 - Grips force at month 12)/12\]; Months -3 to 0 vs. Months 0 to 12; the higher above ratio means higher disease progression rate |
| Change in disease progression rates measuring using 6MWT (6 minute walking test); distance in meters | 15 months | Difference between \[(distance at month -3 - distance at month 0)/3\] and \[(distance at month 0 - distance at month 12)/12\]; Months -3 to 0 vs. Months 0 to 12; the higher above ratio, the higher disease progression rate |
| Change in disease progression rates measuring using EQ-5D (EuroQol Group Life Quality Score) | 15 months | Difference between \[(Score at month -3 - Score at month 0)/3\] and \[(Score at month 0 - Score at month 12)/12\]; Months -3 to 0 vs. Months 0 to 12; the higher above ratio means higher disease progression rate |
| Change in disease progression rates measuring using respiratory vital capacity (standardized vital capacity, %) | 15 months | Difference between \[(vital capacity -3 - vital capacity at month 0)/3\] and \[(vital capacity at month 0 - vital capacity at month 12)/12\]; Months -3 to 0 vs. Months 0 to 12; the higher above ratio means higher disease progression rate |
| Change in disease progression rates measuring using CMAP (compound muscle action potential, mV) | 15 months | Difference between \[(CMAP -3 - CMAP at month 0)/3\] and \[(CMAP at month 0 - CMAP at month 12)/12\]; Months -3 to 0 vs. Months 0 to 12; the higher above ratio means higher disease progression rate |
| Change in disease progression rates measuring using MUNE (motor unit number estimation; number) | 15 months | Difference between \[(MUNE -3 - MUNE at month 0)/3\] and \[(MUNE at month 0 - MUNE at month 12)/12\]; Months -3 to 0 vs. Months 0 to 12; the higher above ratio means higher disease progression rate |
| Change in disease severity measuring using plasma NF-L (Neurofilament-light chain; pg/mL) | 12 months | The NF-L at months 3, 6, 9,12 as compared to that at month 0; the lower the NF-L, the milder disease activity |
| Any side effect under plasmapheresis | 12 months | — |
Countries
Taiwan
Contacts
CONTACTLi-Kai Tsai, MD, PhD
Outcome results
None listed