Clinical trial NCT04454840: Treatment of Intravenous Infusion Plasma in Amyotrophic Lateral Sclerosis
Official title: A Clinical Study on Safety and Effectiveness of Intravenous Infusion Plasma From Healthy Young People to Treat Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: EARLY_PHASE1
Conditions: Amyotrophic Lateral Sclerosis
Keywords: Amyotrophic lateral sclerosis; Plasma infusion
Lead sponsor: Peking University Third Hospital
Sponsor class: OTHER
First posted: 2020-07-02
Enrollment: 20
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).

Eligibility criteria: 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.

Primary outcomes:
measure: Amyotrophic lateral sclerosis Functional Rating Scale scores
timeFrame: 22 months after intervention
Secondary outcomes:
measure: Survival time
timeFrame: 22 months after intervention
measure: Forced vital capacity (FVC)
timeFrame: 22 months after intervention
measure: Cognitive function evaluation (ECAS score)
timeFrame: 22 months after intervention
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking University Third Hospital
Study officials:
affiliation: Peking University Third Hospital
name: Dongsheng Fan, MD.PHD
role: PRINCIPAL_INVESTIGATOR
References: