IMCY-0098 Proof of ACtion in Type 1 Diabetes (IMPACT Study)

NCT04524949 | Terminated Phase 2 DMC FDA Drug

• 2 other identifiers: IMCY-T1D-0032020-001317-20
• Study Type: interventional
• Target: 110
• Locations: 8 countries
• Sites: 29

Brief Summary

The IMPACT study is a study to test a new experimental drug, IMCY-0098, for the treatment of type 1 diabetes (T1D). In most people with type 1 diabetes, the pancreas loses its ability to make insulin because some cells of the body's own immune system mistakenly attack and destroy the cells in the pancreas that produce insulin (islet beta-cells). The study drug IMCY-0098 is being developed to stop the body's own immune system attacking and destroying the insulin-producing cells. When injected, it will induce new immune cells that will specifically destroy the bad immune cells responsible for the damage to the pancreas. IMCY-0098 has previously been tested on recently diagnosed type 1 diabetes patients in the first clinical study between 2017 and 2019 to collect information on the safety of IMCY-0098. The next step is to test the best dose and the best number of injections that show the drug can give a benefit. Two doses of IMCY-0098 will be tested and they will be compared to a placebo. Safety information will also be collected during the study for all the participants.

Conditions

Diabetes Mellitus, Type 1

Keywords

Diabetes Mellitus Type 1; Autoimmune disease; Immunotherapy; Diabetes treatment

Outcome Measures

Primary Outcomes (1)

• Change in stimulated C-peptide response during the first two hours of a mixed meal tolerance test (MMTT) from baseline to 48 weeks between IMCY-0098 and placebo groups

  The area under the stimulated C-peptide response curve over the first two hours of a MMTT

  From baseline to 48 weeks

Secondary Outcomes (12)

• Changes in stimulated C-peptide response during the first two hours of a MMTT for the two doses of IMCY-0098 versus placebo

  From baseline to 24 months

• Difference in Dried Blood Spots (DBS) fasted C-peptide between treatment and placebo groups

  From baseline to 48 weeks

• Changes in DBS C-peptide measurements at each visit comparing each dose with placebo

  From baseline to 24 months

• Effects of each dose of IMCY-0098 on HbA1c

  From baseline to 24 months

• Effects of each dose of IMCY-0098 on hypoglycaemic events

  From baseline to 24 months

Study Arms (3)

IMCY-0098, low dose

EXPERIMENTAL

The dose A (Cohort 1) will consist of subcutaneous administrations of 450 µg of the peptide in two separate injections of 225 µg each (500 µL each).

Drug: IMCY-0098 450 μg

IMCY-0098, high dose

EXPERIMENTAL

The dose B (Cohort 2) will consist of subcutaneous administrations of 1350 µg of the peptide in two separate injections of 675 µg each (500 µL each).

Drug: IMCY-0098 1350 μg

Placebo

PLACEBO COMPARATOR

Participants randomized to placebo will receive subcutaneous administrations of identical volumes of placebo solution to maintain study blind.

Drug: Placebo

Interventions

IMCY-0098 450 μg DRUG

Small synthetic peptide for SC admin. Solvent: alum hydroxide

Also known as: Imotope

IMCY-0098, low dose

IMCY-0098 1350 μg DRUG

Small synthetic peptide for SC admin. Solvent: alum hydroxide

Also known as: Imotope

IMCY-0098, high dose

Placebo DRUG

Solvent: alum hydroxide

Placebo

Eligibility Criteria

• Age: 18 Years - 44 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Have given written informed consent.
• Participants aged ≥ 18 years and \< 45 years at the time of consent
• Have a diagnosis of T1D within maximum 9 weeks at screening (date of the first insulin injection)
• Must have at least one or more diabetes-related autoantibodies present at screening (GAD65, IA-2, or ZnT8)
• Must have random C-peptide levels ≥ 200 pmol/L measured at screening
• Must be Human Leukocyte Antigen (HLA) DR4 positive to participate in the main study OR HLA DR4 negative but HLA DR3 positive to participate in the substudy
• Be willing to comply with intensive diabetes management
• Be treated with insulin therapy in accordance with the local standard of care
• Males with reproductive potential must agree to use adequate contraception up to 90 days after the completion of the last treatment. This includes:
• Barrier contraception (condom and spermicide) or
• True abstinence (where this is in accordance with the participants preferred and usual lifestyle)
• All females must have a negative serum pregnancy test at screening. Women sexually active and of childbearing potential must agree to use a highly effective contraception method from screening up to 90 days after last treatment with the investigational product
• (US ONLY) Have HbA1c levels ≤ 9.5% prior to randomization

You may not qualify if:

• Clinically significant abnormal full blood count (FBC), renal function or liver function at screening including
• Be immunodeficient or have any clinically significant chronic lymphopenia: Leukopenia (\< 3,000 leukocytes /μL), neutropenia (\<1,500 neutrophils/μL), lymphopenia (\<800 lymphocytes/μL), or thrombocytopenia (\<100,000 platelets/μL)
• Evidence of renal dysfunction with serum creatinine greater than 1.5 times the upper limit of normal OR (US ONLY) estimated Glomerular Filtration Rate (eGFR) calculated by Chronic Kidney Disease Epidemiology Collaboration (CKD-Epi) \<90 mL/min per 1.73 m2 in absence of other signs of CKD or rapidly progressing renal disease
• Evidence of liver dysfunction with aspartate aminotransferase (AST) or alanine transaminase (ALT) greater than 3 times the upper limits of normal or (US ONLY) total bilirubin ≥ 2x Upper Limit of Normal (ULN) or Alkaline phosphatase ≥ 2x ULN. For participants presenting with values above ULN but below above threshold for these parameters, the underlying reason should be investigated by the site team to exclude liver disease. Patients for which a liver disease would be diagnosed will be excluded from the study.
• Participants with elevated unconjugated bilirubin (Gilbert's syndrome) are eligible if bilirubin is ≤ 3 times the upper limits of normal and hepatic enzymes and function are otherwise normal (AST/ALT/Alkaline phosphatase within ULN), and there is no evidence of hemolysis
• Have signs or symptoms of serious active infection requiring IV antibiotics and/or hospitalization at study entry
• Have signs or symptoms of active COVID infection or a positive COVID PCR test during the screening period
• Have received any live attenuated vaccine within 3 months prior to the first planned administration of the study product (which includes, but is not limited to: oral poliomyelitis vaccine, measles-mumps-rubella vaccine, yellow fever vaccine, Japanese encephalitis vaccine, dengue vaccine, rotavirus vaccine, varicella vaccine, live-attenuated zoster vaccine, Bacillus Calmette-Guérin \[BCG\] vaccine, oral typhoid vaccine)
• Be currently pregnant or lactating, or anticipate getting pregnant until at least 24 weeks after last study drug administration
• Require the use of immunosuppressive agents including chronic use of systemic steroids. Topical, inhalational or intranasal corticosteroids are allowed
• Have evidence of current or past human immunodeficiency virus (HIV), Hepatitis B or Hepatitis C infection
• Presence of any uncontrolled disease (including uncontrolled autoimmune disease) or abnormal clinical laboratory results that may interfere with study conduct as judged by the investigator
• History of, or current malignancy (except excised basal cell skin cancer)
• Current or ongoing use of non-insulin pharmaceuticals that affect glycaemic control within 7 days prior to screening visit
• Active participation in another T1D treatment study or any investigational intervention study in the previous 30 days or (US ONLY) received gene therapy in the past

Sponsors & Collaborators

• Imcyse SA: lead

Study Sites (29)

University of Alabama at Birmingham

Birmingham, Alabama, 35205, United States

Location

Barbara Davis Center

Aurora, Colorado, 80045, United States

Location

University of Chicago

Chicago, Illinois, 60637, United States

Location

Joslin Diabetes Center

Boston, Massachusetts, 02215, United States

Location

Princess Alexandra Hospital

Brisbane, Australia

Location

Royal Melbourne Hospital

Melbourne, Australia

Location

St. Vincent's Hospital

Melbourne, Australia

Location

Royal North Shore Hospital

Sydney, Australia

Location

Université Libre de Bruxelles - Hôpital Erasme - ULB

Brussels, Belgium

Location

UZ Brussels

Brussels, Belgium

Location

Katholieke Universiteit Leuven UZ Gasthuisberg

Leuven, Belgium

Location

Ospedale San Raffaele S.r.l.

Milan, Italy

Location

AOU Pisana - Ospedale Cisanello

Pisa, Italy

Location

Hospital of Lithuanian University of Health Sciences Kauno Klinikos

Kaunas, Lithuania

Location

Klaipeda university hospital

Klaipėda, Lithuania

Location

Vilnius university hospital Santaros klinikos

Vilnius, Lithuania

Location

UMC - University Children's Hospital

Ljubljana, Slovenia

Location

Department of clinical sciences, CRC/Malmö, Lund University

Lund, Sweden

Location

Addenbrooke's Hospital

Cambridge, United Kingdom

Location

University Hospital of Wales

Cardiff, United Kingdom

Location

Royal Infirmary of Edinburgh

Edinburgh, United Kingdom

Location

Royal Devon and Exeter Hospital

Exeter, United Kingdom

Location

St James´s University Hospital

Leeds, United Kingdom

Location

Leicester General Hospital

Leicester, United Kingdom

Location

Guy's and St Thomas' Hospital

London, United Kingdom

Location

Royal London Hospital

London, United Kingdom

Location

St George's Hospital

London, United Kingdom

Location

Royal Victoria Infirmary

Newcastle, United Kingdom

Location

Churchill Hospital

Oxford, United Kingdom

Location

MeSH Terms

Conditions

Diabetes Mellitus, Type 1; Autoimmune Diseases

Condition Hierarchy (Ancestors)

Diabetes Mellitus; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases; Immune System Diseases

Study Officials

• Jean Van Rampelbergh

  Imcyse SA

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 11, 2020
• First Posted: August 24, 2020
• Study Start: December 29, 2020
• Primary Completion: January 26, 2024
• Study Completion: May 29, 2024
• Last Updated: June 24, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

BE (3); IT (2); SE (1); LI (3); GB (11); AU (4); SL (1); US (4)