Treatment of Intravenous Infusion Plasma in Amyotrophic Lateral Sclerosis

NCT04454840 | Completed Early Phase 1

• 1 other identifier: PUTH2017118
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Amyotrophic Lateral Sclerosis

Keywords

Amyotrophic lateral sclerosis; Plasma infusion

Outcome Measures

Primary Outcomes (1)

• Amyotrophic lateral sclerosis Functional Rating Scale scores

  Rate of amyotrophic lateral sclerosis Functional Rating Scale scores changes

  22 months after intervention

Secondary Outcomes (3)

• Survival time

  22 months after intervention

• Forced vital capacity (FVC)

  22 months after intervention

• Cognitive function evaluation (ECAS score)

  22 months after intervention

Study Arms (2)

Biological+Riluzole

EXPERIMENTAL

Plasma from healthy young people treatment + Riluzole

Biological: Plasma from healthy young people treatment + Riluzole

Riluzole

ACTIVE COMPARATOR

Riluzole

Drug: Riluzole

Interventions

Plasma from healthy young people treatment + Riluzole BIOLOGICAL

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.

Biological+Riluzole

Riluzole DRUG

The basic treatment is Riluzole 25\~50mg twice daily

Riluzole

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Meet the following diagnosis standard: confirmed, proposed and laboratory supported diagnosis;
• Age 50-70 years old ;
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Peking University Third Hospital: lead

Study Sites (1)

Peking University Third Hospital

Beijing, China

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Riluzole

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Thiazoles; Sulfur Compounds; Organic Chemicals; Benzothiazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Dongsheng Fan, MD.PHD

  Peking University Third Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Masking Details: Open Lable
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 29, 2020
• First Posted: July 2, 2020
• Study Start: May 1, 2016
• Primary Completion: May 1, 2019
• Study Completion: May 1, 2019
• Last Updated: July 7, 2020

Record last verified: 2020-07

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)