Baricitinib in New-onset Type 1 Diabetes

NCT ID: NCT04774224

Last Updated: 2024-12-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

91 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-10-30

Study Completion Date

2024-05-06

Brief Summary

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Type 1 diabetes (T1D) results from the killing of insulin-producing pancreatic beta cells by cells of the immune system. The study aims to slow the progressive, immune-mediated loss of insulin-producing beta cells that occurs after clinical presentation. The investigators have identified a pathway that is important for immune cells to kill beta cells, and a drug that will block this pathway and prevent beta cell death. This drug, baricitinib, is already in clinical use for rheumatoid arthritis, and is currently in clinical trials for other diseases, including childhood autoimmune diseases. It is hypothesized that baricitinib treatment for 48 weeks will preserve beta cell function in children and young adults with recently-diagnosed T1D.

The trial aims to recruit 83 participants aged 10-30 years who have been recently diagnosed with T1D. Two thirds of the participants will be randomly assigned to receive baricitinib, one third will receive placebo. The trial will test if baricitinib can slow the progressive loss of insulin-producing beta cells in these patients. The primary objective is to determine if baricitinib can reduce the loss of meal-stimulated plasma C-peptide, a measure of beta-cell function. Maintaining endogenous insulin in recent-onset T1D improves glucose control and may lead to long-term improvements in glucose and lower rates of serious diabetes complications and death.

Detailed Description

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Conditions

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Type 1 Diabetes

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Randomised placebo controlled trial
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
Prior to study initiation, an independent researcher is responsible for generating the randomization schedule. The randomization schedule including treatment group assignment (active or placebo) will be provided to the pharmacists at each one of the study sites. The study pharmacists will conceal the group assignment from study participants, investigators and study staff including the statistician.

Study Groups

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Baricitinib

Baricitinib is an oral JAK1/JAK2-selective inhibitor. Dosage: The dose of baricitinib is 1 x 4mg tablet once daily Duration of administration: 48 weeks Mode of administration: Orally, with or without food

Group Type ACTIVE_COMPARATOR

Baricitinib

Intervention Type DRUG

Participants will take one tablet of study drug per day for 48 weeks, and will be followed up for 48 weeks after study drug treatment has finished. Two-thirds of participants will receive Baricitinib.

Placebo

One placebo tablet once daily for a duration of 48 weeks. Placebo tablets contain lactose monohydrate, microcrystalline cellulose, croscarmellose sodium and magnesium stearate.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Participants will take one tablet of study drug per day for 48 weeks, and will be followed up for 48 weeks after study drug treatment has finished. One-third of participants will receive Placebo.

Interventions

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Baricitinib

Participants will take one tablet of study drug per day for 48 weeks, and will be followed up for 48 weeks after study drug treatment has finished. Two-thirds of participants will receive Baricitinib.

Intervention Type DRUG

Placebo

Participants will take one tablet of study drug per day for 48 weeks, and will be followed up for 48 weeks after study drug treatment has finished. One-third of participants will receive Placebo.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Male or female aged between 10 and 30 years (inclusive) at screening;
2. Diagnosis of T1D according to ADA criteria within 100 days prior to starting study drug;
3. Islet autoantibody positivity (one or more of: GADA, IA-2A, IAA (assessed within one week of commencing insulin therapy), ZnT8A);
4. Stimulated (peak or 90 min) C-peptide \>0.2 nM during a 2-hour MMTT at the screening visit; or random C-peptide result \>0.3 nM during the screening period.
5. Participants of childbearing age who are sexually active must agree to use of effective birth control until the end of the study;
6. Be able to read, understand and give written informed consent;
7. Be willing to comply with intensive diabetes management.

Exclusion Criteria

1. Use of immunosuppressive or immunomodulatory therapies other than inhaled or topical glucocorticoids;
2. Current or past history of deep vein thrombosis or pulmonary embolism;
3. Impaired renal function defined by estimated glomerular filtration rate (according to the CKD-EPI) of \< 60 mL/min/1.73 m2;
4. LDL cholesterol \>4mmol/l;
5. Elevated liver function tests at screening:

1. Aspartate aminotransferase 2x ULN
2. Alanine aminotransferase 2 x ULN;
6. Clinically significant abnormal laboratory parameters at screening including but not limited to:

1. Hemoglobin \< 8 g/L;
2. White blood cells \<2500 cells/µl;
3. Lymphocyte count \<750 cells/µl;
4. Platelets \<50,000 cells/µl;
5. Neutrophils \<1200cells/µL;
7. Known hypersensitivity to baricitinib;
8. Known malignancy with the exception of successfully treated non- metastatic basal cell and squamous cell carcinoma;
9. Pregnancy, a desire for pregnancy, breast feeding, or a desire to father a child during the study;
10. Patients with current or recent (within 12 weeks of screening) clinically significant comorbidity, including clinically serious viral, bacterial, fungal, or parasitic infection. Viral infections include HBV, HCV, EBV, HIV, recent herpes zoster and TB;
11. Treatment with any investigational product within 30 days or 5 half - lives (whichever is longer) prior to baseline visit, or concurrent participation in a clinical trial with an investigational product or device;
12. Experienced any of the following within 12 weeks of screening: myocardial infarction, unstable ischemic heart disease, stroke, or New York Heart Association Stage IV heart failure;
13. Any serious medical condition within the previous 4 weeks which places the participant at an unacceptable risk if he or she were to participate in the study or confounds the ability to interpret data from the study, including, but not limited to, symptomatic cardiovascular, renal, respiratory, hepatic, gastrointestinal, endocrine, haematological and neurological conditions or psychiatric illness/social situations that would limit compliance with study requirements;
14. Have had any major surgery within 8 weeks prior to screening or will require major surgery during the study that, in the opinion of the investigator would pose an unacceptable risk to the participant;
15. History of chronic alcohol abuse or IV drug abuse or other illicit drug abuse within 2 years prior to screening.
Minimum Eligible Age

10 Years

Maximum Eligible Age

30 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Juvenile Diabetes Research Foundation Australia

UNKNOWN

Sponsor Role collaborator

Juvenile Diabetes Research Foundation

OTHER

Sponsor Role collaborator

St Vincent's Institute of Medical Research

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Tom Kay, Prof

Role: PRINCIPAL_INVESTIGATOR

SVI

Locations

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Women's and Children's Hospital Adelaide

North Adelaide, South Australia, Australia

Site Status

St Vincent's Hospital Melbourne

Fitzroy, Victoria, Australia

Site Status

Royal Melbourne Hospital

Parkville, Victoria, Australia

Site Status

Royal Children's Hospital Melbourne

Parkville, Victoria, Australia

Site Status

Countries

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Australia

References

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Waibel M, Thomas HE, Wentworth JM, Couper JJ, MacIsaac RJ, Cameron FJ, So M, Krishnamurthy B, Doyle MC, Kay TW. Investigating the efficacy of baricitinib in new onset type 1 diabetes mellitus (BANDIT)-study protocol for a phase 2, randomized, placebo controlled trial. Trials. 2022 May 23;23(1):433. doi: 10.1186/s13063-022-06356-z.

Reference Type BACKGROUND
PMID: 35606820 (View on PubMed)

Waibel M, Wentworth JM, So M, Couper JJ, Cameron FJ, MacIsaac RJ, Atlas G, Gorelik A, Litwak S, Sanz-Villanueva L, Trivedi P, Ahmed S, Martin FJ, Doyle ME, Harbison JE, Hall C, Krishnamurthy B, Colman PG, Harrison LC, Thomas HE, Kay TWH; BANDIT Study Group. Baricitinib and beta-Cell Function in Patients with New-Onset Type 1 Diabetes. N Engl J Med. 2023 Dec 7;389(23):2140-2150. doi: 10.1056/NEJMoa2306691.

Reference Type RESULT
PMID: 38055252 (View on PubMed)

Related Links

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Other Identifiers

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SVI-BARI-01

Identifier Type: -

Identifier Source: org_study_id