Treatment of Intravenous Infusion Plasma in Amyotrophic Lateral Sclerosis

A Clinical Study on Safety and Effectiveness of Intravenous Infusion Plasma From Healthy Young People to Treat Amyotrophic Lateral Sclerosis

To evaluate the safety and effectiveness of intravenous infusion of plasma from healthy young people for the treatment of amyotrophic lateral sclerosis.

Study Overview

• Status: Completed
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Biological: Plasma from healthy young people treatment + Riluzole; Drug: Riluzole

Detailed Description

This is a single-center, open-label clinical study to evaluate the safety and effectiveness of intravenous infusion of plasma from healthy young people for the treatment of amyotrophic lateral sclerosis. The main outcome indicators are the record of adverse reactions and the rate of change of amyotrophic lateral sclerosisFunctional Rating Scale score. The secondary outcome indicators include survival time-time to the end event (death, tracheotomy, continuous ventilator dependence), forced vital capacity (FVC), recognition Knowledge function evaluation (ECAS score).

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Early Phase 1

Contacts and Locations

Study Locations

• China
  • Beijing, China
    • Peking University Third Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Meet the following diagnosis standard: confirmed, proposed and laboratory supported diagnosis;
• Age 50-70 years old ;
• 3-18 months course of disease;
• Forced vital capacity (FVC) ≥70% predicted value;
• Total amyotrophic lateral sclerosis Functional Rating Scale score ≥36, scores of respiratory related items ≥10;
• Take Riluzole regularly before participate in this trial (25~50mg twice a day for at least 30 days continuously) without obvious side effects and can continue to take for 22 months;
• Participants of childbearing age take reasonable and effective contraceptive measures from the time of enrollment to the end of follow-up;
• Signed informed consent.

Exclusion Criteria:

• Familial amyotrophic lateral sclerosis;
• Female during pregnancy and lactation;
• Positive hepatitis B, hepatitis C or HIV in screening
• History of cytomegalovirus and malaria infection;
• After tracheotomy and ventilator-dependent state (daily use of non-invasive ventilator ≥ 22 hours for 7 consecutive days);
• After percutaneous gastrostomy (PEG) operation;
• Has had allergic reactions and other adverse reactions during blood transfusion;
• Have diseases of the blood system (including Immunoglobulin A deficiency);
• alanine transaminase, Aspartate transaminase≥ 3 times the upper limit of normal;
• Abnormal renal function (Cr, BUN);
• History of malignant tumors;
• Combining severe cardiopulmonary diseases, autoimmune diseases, mental diseases, substance abuse history, etc;
• Currently participating in other clinical studies or using other drugs in researching.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Biological+Riluzole; Plasma from healthy young people treatment + Riluzole	Biological: Plasma from healthy young people treatment + Riluzole; Intravenous injection: the subjects received 400ml of intravenous plasma infusion every 2 weeks, with a continuous course of 2 treatments and a course of treatment every 3 months. The course of treatment lasted 10 months, with a total of 3200ml of plasma infusion.
Active Comparator: Riluzole	Drug: Riluzole; The basic treatment is Riluzole 25~50mg twice daily

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Amyotrophic lateral sclerosis Functional Rating Scale scores	Rate of amyotrophic lateral sclerosis Functional Rating Scale scores changes	22 months after intervention

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Survival time	The time of the end event (death, tracheotomy, continuous ventilator dependence);	22 months after intervention
Forced vital capacity (FVC)	The change from baseline to the end of follow-up;	22 months after intervention
Cognitive function evaluation (ECAS score)	The change from baseline to the end of follow-up;	22 months after intervention

Collaborators and Investigators

• Sponsor: Peking University Third Hospital

Study record dates

Study Major Dates

• Study Start (Actual): May 1, 2016
• Primary Completion (Actual): May 1, 2019
• Study Completion (Actual): May 1, 2019

Study Registration Dates

• First Submitted: June 29, 2020
• First Submitted That Met QC Criteria: June 29, 2020
• First Posted (Actual): July 2, 2020

Study Record Updates

• Last Update Posted (Actual): July 7, 2020
• Last Update Submitted That Met QC Criteria: July 1, 2020
• Last Verified: July 1, 2020

More Information

Terms related to this study

• Keywords: Amyotrophic lateral sclerosis; Plasma infusion
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Excitatory Amino Acid Antagonists; Excitatory Amino Acid Agents; Neuroprotective Agents; Protective Agents; Anticonvulsants; Riluzole
• Other Study ID Numbers: PUTH2017118

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