MedPath

Ciclosporin Followed by Low-dose IL-2 in Patients with Recently Diagnosed Type 1 Diabetes

Phase 2
Recruiting
Conditions
Type 1 Diabetes
Interventions
Registration Number
NCT05153070
Lead Sponsor
Assistance Publique - Hôpitaux de Paris
Brief Summary

Type 1 diabetes (T1D) is caused by the destruction of insulin-producing cells by effector T cells (Teffs), due to a deficiency of regulatory T cells (Tregs).

Ciclosporin effectively blocks the Teffs and controls diabetes, but cannot be considered as a long-term treatment. Low-dose interleukin-2 (ld IL-2) activates and expands Tregs in humans.

Hence, Ld IL-2 in patients in whom the autoimmune process was blocked early by a short treatment (2 months) of cyclosporine should restore immune homeostasis and maintain some insulin production over the long term.

Detailed Description

Primary Objective :

Tregs' response profile, after 4 administrations of 1MIU/day of IL-2 (Day 63-66) in patients with recently diagnosed type 1 diabetes who have been treated with ciclosporin for 2 months.

Primary assessment criterion:

Change in Tregs values at D67 compared to D63 (post-ciclosporin values)

Secondary objectives and secondary assessment criteria:

* Change in residual insulin secretion

* AUC plasma C-peptide concentration after a mixed meal tolerance test at Month 6 (Day 179), Month 12 (Day 361) and after treatment discontinuation at Month 18 (Day 536) and Month 24 (Day 719) compared to baseline;

* Insulin requirement, HbA1c dosage and IDAA1c score at Month 3 (Day 88), Month 6 (Day 179), Month 9 (Day 270), Month 12 (Day 361) and after treatment discontinuation at Month 18 (Day 536) and Month 24 (Day 719) compared to baseline

* Change in Tregs values at Month 3 (Day 88), Month 6 (Day 179), Month 9 (Day 270), Month 12 (Day 361) and after treatment discontinuation at Month 18 (Day 536) and Month 24 (Day 719) compared to baseline and post-ciclosporin values (Day 63)

* Ciclosporin and ILT-101/placebo compliance

* Tolerance

Experimental design:

This is a monocentric, randomized, placebo controlled, double-blind trial in parallel-groups, evaluating a treatment by cyclosporine 7mg/kg/day during 2 months followed by ILT-101/placebo, 1 MIU daily for 5 days and 1 MIU every week, during 10 months.

Population involved:

Male or female, aged between 16 and 35 years, with recent diagnosis of type 1 diabetes (\< 3 months).

Number of subjects: 24 Inclusion period: 12 months Duration of patient participation: 24 months (treatment period: 12 months, follow-up period: 12months) Total duration of the study: 37 months

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
24
Inclusion Criteria
  • • Age at inclusion between ≥ 16 years old (Tanner 5 pubertal stage) and ≤ 45 years old

    • Type 1 diabetes according to ADA criteria, with at least 1 positive autoantibody among the following: anti-islet, anti-GAD, anti-IA2, anti-ZnT8 and anti-insulin.
    • Diagnosis ≤ 3 months
    • No acid ketosis
    • No weight loss > 10% OR with fasting C-peptide ≥ 0.1 nmol/L (after a period of ≥ 15 days following the initiation of insulin therapy
    • Absence of clinically significant biological abnormalities on hematological, biochemical, hepatic, renal and thyroid tests.
    • No documented history of heart disease, no family history of sudden death, AND normal ECG.
    • Effective contraception in men and women of childbearing potential > 2 weeks prior to first administration of the investigational drug and throughout the treatment period (if sexually active). Specifically for women of childbearing age and sexually active, they must use an effective contraceptive method (Pearl Index < 1). The following methods are acceptable: oral hormonal contraceptives, injectable, or implanted (with the exception of oral minipills: i.e. low doses of gestagens which are not acceptable (lynestrenol and norestisteron), intrauterine contraceptives (e.g. progestin-release systems)),
    • Free, informed and written consent, signed by the patient and the investigator, prior to any examination required by the trial.

If the patient is a minor, the signatures of both parents and of the child will be collected (or the legal representative if only one parent is alive).

Exclusion Criteria
  • Known contraindications to IL2 treatment:

    • Hypersensitivity to the active substance or to one of the excipients.
    • Signs of active infection requiring antibiotics
    • Documented history of clinical autoimmune disease
    • Oxygen saturation ≤ 90%
    • Existence of a serious dysfunction in a vital organ
    • History of organ allograft,
  • Known contraindications to treatment with cyclosporine

  • Presence of unauthorized treatment, i.e. cytotoxic drugs, products known for their impact on blood glucose levels or for their interactions with the treatments under trial

  • Patients who have received anti-diabetic treatment other than insulin for more than 3 consecutive months.

  • Anti-thyroperoxidase positive and abnormal TSH and T4 at inclusion

  • Anti-transglutaminase positive at inclusion

  • EBV viral load > 2000 IU/ml

  • CMV viral load > 400 IU/ml

  • HBV, HCV or HIV infection

  • Lymphopenia ≤ 1000/ mm3

  • Presence or history of cancer that has been cured for less than five years, except in situ cervical or basal cell carcinoma in early stage management,

  • Participation in other intervention research involving humans < 3 months,

  • Pregnant or breastfeeding women

  • Lack of social security affiliation (as a beneficiary or assignee)

  • Vaccination with live attenuated virus during the last 4 weeks before the start of the experimental treatment and during the entire treatment phase.

  • Patient with active SARS-CoV-2 infection

  • Patient with chronic respiratory disease

  • Subject under legal protection (such as tutorship, curatorship, or judicial safeguard)

  • Subject hospitalized without consent, unable to express consent or deprived of liberty

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Ciclosporin/ILT-101CyclosporinCiclosporin during 2 months (for all patients) followed by ILT-101 during 10 months
Ciclosporin/ILT-101ILT101Ciclosporin during 2 months (for all patients) followed by ILT-101 during 10 months
Ciclosporin/placeboCyclosporinCiclosporin during 2 months (for all patients) followed by placebo during 10 months
Ciclosporin/placeboPlaceboCiclosporin during 2 months (for all patients) followed by placebo during 10 months
Primary Outcome Measures
NameTimeMethod
Treg variationFrom Day 63 to Day 67

Change in Tregs values at Day 67 compared to Day 63 (post-ciclosporin value)

Secondary Outcome Measures
NameTimeMethod
Variation in HbA1c value (in %) during the treatment period and during the 1-year follow-up period compared to Baseline at month 6up to month 6

Variation in HbA1c value (in %) during the treatment period and during the 1-year follow-up period compared to baseline

Change in Area under curve (AUC (T0-T120) of serum C-peptide at month 12up to month 12

Change in Area under curve (AUC (T0-T120) of serum C-peptide after mixed-meal tolerance test compared to baseline

2. Variation in HbA1c value (in %) 3. Variation in IDAA1c score (IDAA1c score = HbA1c (in %) + \[4 × insulin dose (in international units per kilogram per 24 h)\].

4. Change in Insulin requirement (insulin dose in international units per kilogram per 24 h 5. Change in Tregs values (in %/CD4+) at month 3, month 6, month 9, month 12 and after treatment interruption at month 18 and month 24 compared to baseline and post-cyclosporin values (Day 63)

Variation in HbA1c value (in %) during the treatment period and during the 1-year follow-up period compared to Baseline at month 3up to month 3

Variation in HbA1c value (in %) during the treatment period and during the 1-year follow-up period compared to baseline

Variation in HbA1c value (in %) during the treatment period and during the 1-year follow-up period compared to Baseline at month 9up to month 9

Variation in HbA1c value (in %) during the treatment period and during the 1-year follow-up period compared to baseline

Variation in HbA1c value (in %) during the treatment period and during the 1-year follow-up period compared to Baseline at month 12up to month 12

Variation in HbA1c value (in %) during the treatment period and during the 1-year follow-up period compared to baseline

Variation in HbA1c value (in %) during the treatment period and during the 1-year follow-up period compared to Baseline at month 18up to month 18

Variation in HbA1c value (in %) during the treatment period and during the 1-year follow-up period compared to baseline

Variation in HbA1c value (in %) during the treatment period and during the 1-year follow-up period compared to Baseline at month 24up to month 24

Variation in HbA1c value (in %) during the treatment period and during the 1-year follow-up period compared to baseline

Change in Area under curve (AUC (T0-T120) of serum C-peptide at month 6up to month 6

Change in Area under curve (AUC (T0-T120) of serum C-peptide after mixed-meal tolerance test compared to baseline

2. Variation in HbA1c value (in %) 3. Variation in IDAA1c score (IDAA1c score = HbA1c (in %) + \[4 × insulin dose (in international units per kilogram per 24 h)\].

4. Change in Insulin requirement (insulin dose in international units per kilogram per 24 h 5. Change in Tregs values (in %/CD4+) at month 3, month 6, month 9, month 12 and after treatment interruption at month 18 and month 24 compared to baseline and post-cyclosporin values (Day 63)

Variation in HbA1c value (in %) during the treatment period and during the 1-year follow-up period compared to Baseline at day 63,up to day 63

Variation in HbA1c value (in %) during the treatment period and during the 1-year follow-up period compared to baseline

Variation in IDAA1c scoreduring the treatment period and during the 1 year follow-up period up to month 3up to month 3

Variation in IDAA1c score (IDAA1c score = HbA1c (in %) + \[4 × insulin dose (in international units per kilogram per 24 h)\] during the treatment period and during the 1 year follow-up period compared to baseline

Variation in IDAA1c scoreduring the treatment period and during the 1 year follow-up period at month 6up to month 6

Variation in IDAA1c score (IDAA1c score = HbA1c (in %) + \[4 × insulin dose (in international units per kilogram per 24 h)\] during the treatment period and during the 1 year follow-up period compared to baseline

Variation in IDAA1c scoreduring the treatment period and during the 1 year follow-up period at month 9up to month 9

Variation in IDAA1c score (IDAA1c score = HbA1c (in %) + \[4 × insulin dose (in international units per kilogram per 24 h)\] during the treatment period and during the 1 year follow-up period compared to baseline

Variation in IDAA1c scoreduring the treatment period and during the 1 year follow-up period at month 12up to month 12

Variation in IDAA1c score (IDAA1c score = HbA1c (in %) + \[4 × insulin dose (in international units per kilogram per 24 h)\] during the treatment period and during the 1 year follow-up period compared to baseline

Variation in IDAA1c scoreduring the treatment period and during the 1 year follow-up period at day month 18up to month 18

Variation in IDAA1c score (IDAA1c score = HbA1c (in %) + \[4 × insulin dose (in international units per kilogram per 24 h)\] during the treatment period and during the 1 year follow-up period compared to baseline

Change in Area under curve (AUC (T0-T120) of serum C-peptide at month 24up to month 24

Change in Area under curve (AUC (T0-T120) of serum C-peptide after mixed-meal tolerance test compared to baseline

2. Variation in HbA1c value (in %) 3. Variation in IDAA1c score (IDAA1c score = HbA1c (in %) + \[4 × insulin dose (in international units per kilogram per 24 h)\].

4. Change in Insulin requirement (insulin dose in international units per kilogram per 24 h 5. Change in Tregs values (in %/CD4+) at month 3, month 6, month 9, month 12 and after treatment interruption at month 18 and month 24 compared to baseline and post-cyclosporin values (Day 63)

Variation in IDAA1c scoreduring the treatment period and during the 1 year follow-up period at day 63up to day 63

Variation in IDAA1c score (IDAA1c score = HbA1c (in %) + \[4 × insulin dose (in international units per kilogram per 24 h)\] during the treatment period and during the 1 year follow-up period compared to baseline

Variation in IDAA1c scoreduring the treatment period and during the 1 year follow-up period at month 24up to month 24

Variation in IDAA1c score (IDAA1c score = HbA1c (in %) + \[4 × insulin dose (in international units per kilogram per 24 h)\] during the treatment period and during the 1 year follow-up period compared to baseline

Change in Insulin requirement (insulin dose in international units per kilogram per 24 h) during the treatment period and during the 1 year follow-up period at day 30up to day 30

Change in Insulin requirement (insulin dose in international units per kilogram per 24 h) during the treatment period and during the 1 year follow-up period compared to baseline

Change in Insulin requirement (insulin dose in international units per kilogram per 24 h) during the treatment period and during the 1 year follow-up period at day 63up to day 63

Change in Insulin requirement (insulin dose in international units per kilogram per 24 h) during the treatment period and during the 1 year follow-up period compared to baseline

Change in Insulin requirement (insulin dose in international units per kilogram per 24 h) during the treatment period and during the 1 year follow-up period at month 3up to month 3

Change in Insulin requirement (insulin dose in international units per kilogram per 24 h) during the treatment period and during the 1 year follow-up period compared to baseline

Change in Insulin requirement (insulin dose in international units per kilogram per 24 h) during the treatment period and during the 1 year follow-up period at month 6up to month 6

Change in Insulin requirement (insulin dose in international units per kilogram per 24 h) during the treatment period and during the 1 year follow-up period compared to baseline

Change in Insulin requirement (insulin dose in international units per kilogram per 24 h) during the treatment period and during the 1 year follow-up period at month 9up to month 9

Change in Insulin requirement (insulin dose in international units per kilogram per 24 h) during the treatment period and during the 1 year follow-up period compared to baseline

Change in Insulin requirement (insulin dose in international units per kilogram per 24 h) during the treatment period and during the 1 year follow-up period at month 12up to month 12

Change in Insulin requirement (insulin dose in international units per kilogram per 24 h) during the treatment period and during the 1 year follow-up period compared to baseline

Change in Insulin requirement (insulin dose in international units per kilogram per 24 h) during the treatment period and during the 1 year follow-up period at month 18up to month 18

Change in Insulin requirement (insulin dose in international units per kilogram per 24 h) during the treatment period and during the 1 year follow-up period compared to baseline

Change in Insulin requirement (insulin dose in international units per kilogram per 24 h) during the treatment period and during the 1 year follow-up period at month 24up to month 24

Change in Insulin requirement (insulin dose in international units per kilogram per 24 h) during the treatment period and during the 1 year follow-up period compared to baseline

Change in Tregs values (in %/CD4+) after treatment interruption and post-cyclosporin values at day 30up to day 30

Change in Tregs values (in %/CD4+) after treatment interruption compared to baseline and post-cyclosporin values

Change in Tregs values (in %/CD4+) after treatment interruption and post-cyclosporin values at month 18up to month 18

Change in Tregs values (in %/CD4+) after treatment interruption compared to baseline and post-cyclosporin values

Change in Tregs values (in %/CD4+) after treatment interruption and post-cyclosporin values at month 24up to month 24

Change in Tregs values (in %/CD4+) after treatment interruption compared to baseline and post-cyclosporin values

incidence of adverse events at day 30up to day 30

incidence of adverse events throughout the study (according to NCI-CTC AE classification) to the Baseline to month 24

incidence of adverse events at day 63up to day 63

incidence of adverse events throughout the study (according to NCI-CTC AE classification) to the Baseline to month 24

Change in Tregs values (in %/CD4+) after treatment interruption and post-cyclosporin values at day 63up to day 63

Change in Tregs values (in %/CD4+) after treatment interruption compared to baseline and post-cyclosporin values

Change in Tregs values (in %/CD4+) after treatment interruption and post-cyclosporin values at month 3up to month 3

Change in Tregs values (in %/CD4+) after treatment interruption compared to baseline and post-cyclosporin values

Change in Tregs values (in %/CD4+) after treatment interruption and post-cyclosporin values at month 6up to month 6

Change in Tregs values (in %/CD4+) after treatment interruption compared to baseline and post-cyclosporin values

Change in Tregs values (in %/CD4+) after treatment interruption and post-cyclosporin values at month 9up to month 9

Change in Tregs values (in %/CD4+) after treatment interruption compared to baseline and post-cyclosporin values

Change in Tregs values (in %/CD4+) after treatment interruption and post-cyclosporin values at month 12up to month 12

Change in Tregs values (in %/CD4+) after treatment interruption compared to baseline and post-cyclosporin values

incidence of adverse events at month 3up to month 3

incidence of adverse events throughout the study (according to NCI-CTC AE classification) to the Baseline to month 24

incidence of adverse events at month 6up to month 6

incidence of adverse events throughout the study (according to NCI-CTC AE classification) to the Baseline to month 24

incidence of adverse events at month 9up to month 9

incidence of adverse events throughout the study (according to NCI-CTC AE classification) to the Baseline to month 24

incidence of adverse events at month 12up to month 12

incidence of adverse events throughout the study (according to NCI-CTC AE classification) to the Baseline to month 24

incidence of adverse events at month 18up to month 18

incidence of adverse events throughout the study (according to NCI-CTC AE classification) to the Baseline to month 24

incidence of adverse events at month 24up to month 24

incidence of adverse events throughout the study (according to NCI-CTC AE classification) to the Baseline to month 24

Trial Locations

Locations (1)

Lorenzon Roberta

🇫🇷

Paris, France

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