Study Purpose:
The purpose of this study was to evaluate the efficacy and safety of low-dose IL-2 in the treatment of immunorelated ALS syndrome.Study Status:
Recruiting
Disease:
Amyotrophic Lateral Sclerosis
Study Type:
Interventional
Type of Intervention:
Drug
Intervention Name:
IL-2
Placebo:
No
Phase:
Phase 2
Study Chair(s)/Principal Investigator(s):
Dongsheng Fan, Peking University Third Hospital
Clinicaltrials.gov ID:
Neals Affiliated?
No
Coordinating Center Contact Information
Dongsheng Fan / email hidden; JavaScript is required / +86 13701023871
Full Study Summary:
This is a single-center, open-label, self-controlled clinical study with a sample size of 10 patients for 48 weeks, including 24 weeks of administration, and 24 weeks of follow-up. During the administration period, medication was given for 2 weeks and rest for 2 weeks, as a course of treatment, with a total of 6 courses for 24 weeks. During the administration period, the drug was given on alternate days, and IL-2 1mIU was subcutaneously injected the next day for 7 times, and then rested for 2 weeks, and the cycle was repeated for 6 times. The primary outcome index was the change in the rate of ALSFRS-R score between administration period and follow-up period. Secondary outcome measures included changes in the rate of ALSAQ-40 score, ROADS score, MRC score, survival time, FVC%, Treg and CD4+ T cell subsets, inflammatory factors, serum and cerebrospinal fluid NFL during follow-up versus administration , changes in the inhibition function of Treg; Exploratory outcome indicators included the change degree of compound muscle action potential (CMAP) amplitude, quantitative analysis of corneal nerve morphologic changes by corneal confocal microscopy (CCM), Treg single-cell sequencing transcriptome analysis. The related safety indexes were also evaluated.
Study Sponsor:
Peking University Third Hospital
Estimated Enrollment:
13
Estimated Study Start Date:
01 / 01 / 2020
Estimated Study Completion Date:
12 / 31 / 2022
Posting Last Modified Date:
08 / 18 / 2021
Date Study Added to neals.org:
07 / 07 / 2021
Minimum Age:
18 Years
Maximum Age:
70 Years
Inclusion Criteria:- 18-70 years old;
- Clinically diagnosed with ALS syndrome, i.e., with ALS -like manifestations, consisting of a combination of upper and/or lower motor neuron damage;
- significant abnormalities with rheumatoid immune-related indicators, or diagnoses of immune-mediated ALS syndrome, including but not limited to multifocal motor neuropathy (MMN), Lewis-Sumner syndrome, and other ALS-like syndromes with an immune background that cannot be clearly classified;
- Poor treatment with conventional hormones or gamma globulin;
- Permitted concomitant treatment: oral prednisone or equivalent doses of other glucocorticoids (≤1.0mg/kg/d); Oral routine dose of immunosuppressants or immunomodulators, such as cyclophosphamide, tacrolimus, etc.; Routine oral doses such as too much force; Doses and types of accompanying therapeutic drugs should not be changed from the trial enrollment to the end of follow-up.
- For women of reproductive age, contraception for at least 2 weeks at the time of enrolment and negative urine HCG;
- Reasonable and effective contraceptive measures should be taken by subjects of childbearing age from the time of trial enrollment to the end of follow-up;
- Signed informed consent.
Exclusion Criteria:
- Allergic or intolerance to IL2;
- Receive non-standard treatment or use of excessive dose of glucocorticoids or gamma globulin intravenously within 2 months before enrollment;
- Vaccination within 6 months before enrolment or between enrolment and the end of follow-up;
- Peripheral venous white blood cells < 2000/mm3, lymphocytes < 600/mm3, platelets < 80,000 /mm3;
- Complicated with severe infection or inflammation, such as bacteremia, sepsis, etc.;
- Complicated blood system diseases, infectious diseases (hepatitis, HIV, tuberculosis, etc.), mental diseases, dementia, severe hypotension, substance abuse history, malignant tumor history, organ transplantation history, etc.;
- Severe liver, kidney, lung or heart dysfunction: heart failure (≥NYHA grade III), renal insufficiency (creatinine clearance ≤30ml/min), abnormal liver function (3 times the upper limit of normal >);
- Pregnant and lactating women;
- Currently participating in other clinical studies or using other investigational drugs.
Peking University Third Hospital | Recruiting
Dongsheng Fan
Beijing, Beijing
100098
China