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Low Dose IL-2 in the Treatment of Immune-associated ALS Syndrome

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ClinicalTrials.gov Identifier: NCT04952155
Recruitment Status : Recruiting
First Posted : July 7, 2021
Last Update Posted : August 18, 2021
Sponsor:
Information provided by (Responsible Party):
Peking University Third Hospital

Brief Summary:
The purpose of this study was to evaluate the efficacy and safety of low-dose IL-2 in the treatment of immunorelated ALS syndrome.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: IL-2 Phase 2

Detailed Description:
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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 13 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Single-center, Open-label Clinical Study to Evaluate the Efficacy and Safety of Low-dose IL-2 in the Treatment of Immune-associated ALS Syndrome
Actual Study Start Date : January 1, 2020
Estimated Primary Completion Date : December 31, 2021
Estimated Study Completion Date : December 31, 2022


Arm Intervention/treatment
Experimental: IL-2
The administration period was divided into 6 courses. 6 cycles of IL-2 were administered subcutaneously at a dose of 1 million IU every other day for 2 weeks, followed by a 2-week break in treatment.
Drug: IL-2
The administration period was divided into 6 courses. 6 cycles of IL-2 were administered subcutaneously at a dose of 1 million IU every other day for 2 weeks, followed by a 2-week break in treatment. The adminstration course was 24 weeks.. The 24-week follow-up period was followed after the treatment.




Primary Outcome Measures :
  1. ALSFRS-R score [ Time Frame: week 0,week 24 and week 48 ]
    Changes in the rate of ALSFRS-R score during administration period compared with follow-up period


Secondary Outcome Measures :
  1. ALSFRS-R score [ Time Frame: week 0,week 24 and week 48 ]
    Changes in the slope of ALSFRS-R score during adminstration period compared with the follow-up period

  2. ROADS score [ Time Frame: week 0,week 24 and week 48 ]
    Changes in the rate of ROADS score during adminstration period compared with the follow-up period

  3. ALSAQ-40 score [ Time Frame: week 0,week 24 and week 48 ]
    Changes in the rate of ALSAQ-40 score during adminstration period compared with the follow-up period

  4. MRC score [ Time Frame: week 0,week 24 and week 48 ]
    Changes in the rate of ALSAQ-40 score during adminstration period compared with the follow-up period

  5. Immunological Responses [ Time Frame: week 0 and week 24 ]
    Analysis regulatory CD4+ T (Treg) cells , interleukin 17 (IL-17)-producing helper T (Th17) cells,Teff cells,follicular helper T (Tfh) cells and related cytokines before and during IL-2 treatment.

  6. NFL in the serum and cerebrospinal fluid [ Time Frame: week 0,week 24 and week 48 ]


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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04952155


Contacts
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Contact: Dongsheng Fan +86 13701023871 dsfan@sina.com

Locations
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China, Beijing
Peking University Third Hospital Recruiting
Beijing, Beijing, China, 100098
Contact: Dongsheng Fan         
Sponsors and Collaborators
Peking University Third Hospital
Investigators
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Principal Investigator: Dongsheng Fan Peking University Third Hospital
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Responsible Party: Peking University Third Hospital
ClinicalTrials.gov Identifier: NCT04952155    
Other Study ID Numbers: M2020420
First Posted: July 7, 2021    Key Record Dates
Last Update Posted: August 18, 2021
Last Verified: June 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases