Plasmapheresis in Amyotrophic Lateral Sclerosis With Autoantibody Against NRIP (PALATIN)

Patient with amyotrophic lateral sclerosis (ALS) having anti-NRIP autoantibody showed titer-dependent detrimental Effects. Plasmapheresis might benefit this subgroup of patients via removal of anti-NRIP autoantibody

Study Overview

• Status: Recruiting
• Conditions: Amyotrophic Lateral Sclerosis; Plasmapheresis
• Intervention / Treatment: Procedure: Plasmapheresis

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Li-Kai Tsai, MD, PhD; Phone Number: 63476 886-2-23123456; Email: milikai@ntuh.gov.tw

Study Locations

• Taiwan
  • Taipei, Taiwan, 100
    • Recruiting
    • National Taiwan University Hospital
    • Contact: Li-Kai Tsai, MD, PhD; Email: milikai@ntuh.gov.tw

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Patients with amyotrophic lateral sclerosis (ALS) at the age more than 20 years and having plasma anti-NRIP autoantibody.
2. Agree to receive plasmapheresis intervention.
3. Agree to participate in the trial and receive serial examinations and follow up.

Exclusion Criteria:

1. Patients without plasma anti-NRIP autoantibody.
2. Patients requiring permanent ventilator support for ALS progression.
3. Not able to receive plasmapheresis or trial-related examinations.
4. Under pregnancy.
5. Blood fibrinogen level less than 50 mg/dl.
6. Specific ALS subtypes, including primary lateral sclerosis, progressive muscular atrophy, flail arm syndrome, or flail leg syndrome.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: ALS patients receiving plasmapheresis; Plasmapheresis in ALS patients with different titers of autoantibody against NRIP	Procedure: Plasmapheresis; Regular plasmapheresis to remove anti-NRIP autoantibody

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in ALSFRS-R decline	Change in ALSFRS-R decline before (3-month) and after (3-month) intervention. <ALSFRS-R indicates revised ALS functional rating scale, ranged from 0 to 48 with the higher, the better motor function>	Before (3-month) and after (3-month) intervention

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in ALSFRS-R decline	Change in ALSFRS-R decline before (3-month) and after (6-month) intervention	Before (3-month) and after (6-month) intervention
Changes in ALSFRS-R score	Changes in ALSFRS-R score from day 0 to days 30, 90, and 180	Day 0 to days 30, 90, and 180
Change in force vital capacity	Change in force vital capacity before intervention and on day 90	Before intervention and on day 90
Change in compound motor action potentials	Change in compound motor action potentials before intervention and on day 90	Before intervention and on day 90
Changes in anti-NRIP titer	Changes in anti-NRIP titer from day 0 to days 30, 90, and 180	Day 0 to days 30, 90, and 180
Any adverse effect under plasmapheresis	Any adverse effect during and within 6 months after plasmapheresis	Within 6 months during and after plasmapheresis

Collaborators and Investigators

• Sponsor: National Taiwan University Hospital
• Investigators: Principal Investigator: Li-Kai Tsai, MD, PhD, National Taiwan University Hospital

Study record dates

Study Major Dates

• Study Start (Actual): May 1, 2023
• Primary Completion (Estimated): March 31, 2025
• Study Completion (Estimated): March 31, 2025

Study Registration Dates

• First Submitted: September 28, 2022
• First Submitted That Met QC Criteria: September 28, 2022
• First Posted (Actual): October 3, 2022

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: December 15, 2024
• Last Verified: December 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Pathologic Processes; Neuromuscular Diseases; Metabolic Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: 202202042DINC

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No