Amyotrophic Lateral Sclerosis
Conditions
Keywords
Amyotrophic lateral sclerosis; Plasma infusion
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).
Interventions
BIOLOGICALPlasma 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.
DRUGRiluzole
The basic treatment is Riluzole 25\ 50mg twice daily
Sponsors
Peking University Third Hospital
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Masking description
Open Lable
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Amyotrophic lateral sclerosis Functional Rating Scale scores | 22 months after intervention | Rate of amyotrophic lateral sclerosis Functional Rating Scale scores changes |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Survival time | 22 months after intervention | The time of the end event (death, tracheotomy, continuous ventilator dependence); |
| Forced vital capacity (FVC) | 22 months after intervention | The change from baseline to the end of follow-up; |
| Cognitive function evaluation (ECAS score) | 22 months after intervention | The change from baseline to the end of follow-up; |
Countries
China
Outcome results
None listed