MIROCALS: Modifying Immune Response and OutComes in ALS

• Status: Active, not recruiting
• Trial end date: 2021-09-01
• Target enrollment: 0
• Participant gender: All

Summary

MIROCALS is a phase II study of ld-IL-2 as a therapeutic agent for ALS. A randomized (1:1), placebo-controlled, double-blind, parallel group trial will be carried out to assess ld-IL-2 safety and clinical efficacy on survival and functional decline in newly diagnosed ALS patients treated for 18 months. Randomization will be stratified according to (i) country (n = 2 levels: UK, France) and (ii) site of onset (n= 2 levels: bulbar vs limb onset).

The primary objective to evaluate the clinical efficacy and safety of the experimental drug (ld IL-2) over an 18 months period in order to establish the proof of concept (PoC) that modifying immune responses through the enhancement of regulatory T cells modifies the rate of ALS disease progression.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Centre Hospitalier Universitaire de Nīmes
• Treatments: Interleukin-2; Pharmaceutical Solutions; Riluzole

Criteria

Main Inclusion criteria

• Patient is 18 years old and less than 76 years old

• Possible, Probable, Probable laboratory-supported or Definite ALS as defined by El Escorial Revised ALS diagnostic criteria

• Disease duration <= 24 months

• Slow Vital capacity >= 70% of normal

• No prior or present riluzole treatment

• Lumbar punctures accepted by patient and done

Main Exclusion criteria

• Other neurodegenerative disease that could explain signs or symptoms

• Contra indication for lumbar puncture (history of allergy to xylocaine, presence of contra-indicated treatment, or coagulation test abnormality, clinically significant coagulopathy or thrombocytopenia)

• Non authorized treatment

• Other disease or disorders that could preclude functional assessment, or life-threatening disorders

• Any documented, active, past or present, auto-immune disorders except asymptomatic Hashimoto thyroiditis

• Using assisted ventilation

• Feeding through gastrostomy or nasogastric tube

• Women of child-bearing potential or sexually active man without contraception

• Pregnant or breast feeding woman

• Any clinically significant laboratory abnormality (excepting cholesterol, triglyceride, glucose, CK, ferritin)

• History of documented symptomatic and treated asthma within the past 5 years