Immuno-modulation in Amyotrophic Lateral Sclerosis- a Phase II Study of Safety and Activity of Low Dose Interleukin-2

Study Purpose:

The primary objective is to evaluate in ALS patients the regulatory T cell early response to two low-doses of IL-2 at 1 and 2 MIU per day after one course of 5 consecutive days comparatively to placebo.

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:


Intervention Name:

Placebo, 1.0 MIU IL-2 per day, 2.0 MIU IL-2 per day



Phase 2

Study Chair(s)/Principal Investigator(s):

Raul Juntas-Morales, MD, CHRU de Montpellier ID:


Neals Affiliated?


Coordinating Center Contact Information

CHRU de Montpellier - Hôpital Gui de Chauliac

Montpellier, United States

Full Study Summary:

This is a phase II study on ld-IL-2 as a therapeutic agent for ALS which aims at defining the activity and safety of a range a doses for subsequent use of the best dose in a phase II/III trial. For ethical reasons, ld-IL-2 must be tested as an add-on therapy to riluzole hence all patients will need to be treated with riluzole for at least three months prior to entry. A randomized (1:1:1), placebo-controlled, double-blind, parallel group trial will be carried out to assess ld-IL-2 activity on regulatory T cells and immuno-inflammatory markers in ALS patients treated for 3 months (5 days every four weeks repeated three times).

The secondary objectives of this study are:

A. To evaluate maintenance of Tcell response after three repeated 5-day courses at one course every four weeks for 12 weeks.

B. To evaluate the safety of ld-IL-2 therapy in an ALS population, with an overall follow-up of 6 months (up to 15 weeks after last administration); C. To evaluate functional changes throughout the study; D. To evaluate changes in other pre-defined blood cytology parameters, and a blood biomarker for axonal damage.

Study Sponsor:

Centre Hospitalier Universitaire de Nīmes

Estimated Enrollment:


Estimated Study Start Date:

08 / 31 / 2015

Estimated Study Completion Date:

05 / 01 / 2016

Posting Last Modified Date:

06 / 01 / 2016

Date Study Added to

02 / 11 / 2014

For additional information, please visit https://clinicalt...+Sclerosis&rank=1

Minimum Age:

18 Years

Maximum Age:

75 Years

Inclusion Criteria:

- The patient has been correctly informed

- The patient must have given his/her informed and signed consent.

- The patient must be insured or beneficiary of a health insurance plan.

- The patient is at least 18 years old and less than 75 years old

- Probable, or laboratory-supported probable or definite ALS as defined by El Escorial Revised ALS diagnostic criteria (according to Airlie House Conference 1988)

- Stable on riluzole treatment for more than 3 months with liver function test results < 2ULN

- Disease duration ≤ 5 years

- Vital capacity ≥ 70% of normal

- Ability to swallow without the requirement for nasogastric or PEG feeding

- Agreement for patient to use an adequate method of contraception throughout the study and for 2 weeks after post study visit

- The patient is available and willing to participate in seven study visits occurring at the CHU within the next six months

Exclusion Criteria:

- The patient is participating in another interventional study

- Within the past three months, the patient has participated in another interventional

- The patient is in an exclusion period determined by a previous study

- The patient is under judicial protection

- The patient is an adult under guardianship

- The patient refuses to sign the consent

- It is impossible to correctly inform the patient

- Other life threatening disease

- Presence of contra-indicated concomitant treatments or with potential neuroprotective benefit (see section 11.2 of the protocol)

- Presence of tracheostomy or non-invasive ventilation

- Use of Percutaneous endoscopic gastrostomy (PEG) or nasogastric tube

- Presence of clinical infection (treated or untreated)

- Positive serology for CMV, EBV (confirmed by viral load), or HIV

- Vaccination within 8 weeks prior to first experimental dosing

- Other disease precluding functional assessments

- Cancer within the past 5 years (except stable non-metastatic basal cell skin carcinoma or in situ carcinoma of the cervix)

- Severe cardiac or pulmonary disease

- Documented auto-immune disorders except asymptomatic Hashimoto thyroiditis

- Women of child bearing age without contraception or pregnant or breast feeding

- Any clinically significant laboratory abnormality (excepting cholesterol, triglyceride and glucose)

CHRU de Montpellier - Hôpital Gui de Chauliac

Montpellier 34295