Treatment of Intravenous Infusion Plasma in Amyotrophic Lateral Sclerosis

Early Phase 1

Completed

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Drug: Riluzole; Biological: Plasma from healthy young people treatment + Riluzole

• Registration Number: NCT04454840
• Lead Sponsor: Peking University Third Hospital

Brief Summary

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

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 Timeline

• Study Start: 2016-05-01
• Primary Completion: 2019-05-01
• Study Completion: 2019-05-01
• Study First Submitted: 2020-06-29
• Study First Submitted QC: 2020-06-29
• Status Verified: 2020-07
• Last Update Submitted: 2020-07-01
• Study First Posted: 2020-07-02
• Last Update Posted: 2020-07-07

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Riluzole	Riluzole	Riluzole
Biological+Riluzole	Plasma from healthy young people treatment + Riluzole	Plasma from healthy young people treatment + Riluzole

Primary Outcome Measures

Name	Time	Method
Amyotrophic lateral sclerosis Functional Rating Scale scores	22 months after intervention	Rate of amyotrophic lateral sclerosis Functional Rating Scale scores changes

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Peking University Third Hospital; 🇨🇳 Beijing, China