Type 1 DiabetesPhase 2BANDITNCT04774224
Baricitinib in New-onset Type 1 Diabetes
A Phase 2 study testing Baricitinib in people with Type 1 Diabetes, run by St Vincent's Institute of Medical Research at 4 sites in Australia.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 10 to 30 years
- Conditions the study is for
- Type 1 Diabetes
- What is being tested?
- Baricitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 91 participants
- Where
- 4 sitesAustralia
- Study dates
- 30 Oct 2020to 6 May 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male or female aged between 10 and 30 years (inclusive) at screening;
- Diagnosis of T1D according to ADA criteria within 100 days prior to starting study drug;
- Islet autoantibody positivity (one or more of: GADA, IA-2A, IAA (assessed within one week of commencing insulin therapy), ZnT8A);
- 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.
- Participants of childbearing age who are sexually active must agree to use of effective birth control until the end of the study;
- Be able to read, understand and give written informed consent;
- Be willing to comply with intensive diabetes management.
Who cannot join
- Use of immunosuppressive or immunomodulatory therapies other than inhaled or topical glucocorticoids;
- Current or past history of deep vein thrombosis or pulmonary embolism;
- Impaired renal function defined by estimated glomerular filtration rate (according to the CKD-EPI) of < 60 mL/min/1.73 m2;
- LDL cholesterol >4mmol/l;
- Elevated liver function tests at screening:
- Aspartate aminotransferase 2x ULN
Show all 22Show fewer
- Alanine aminotransferase 2 x ULN;
- Clinically significant abnormal laboratory parameters at screening including but not limited to:
- Hemoglobin < 8 g/L;
- White blood cells <2500 cells/µl;
- Lymphocyte count <750 cells/µl;
- Platelets <50,000 cells/µl;
- Neutrophils <1200cells/µL;
- Known hypersensitivity to baricitinib;
- Known malignancy with the exception of successfully treated non- metastatic basal cell and squamous cell carcinoma;
- Pregnancy, a desire for pregnancy, breast feeding, or a desire to father a child during the study;
- 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;
- 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;
- 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;
- 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;
- 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;
- History of chronic alcohol abuse or IV drug abuse or other illicit drug abuse within 2 years prior to screening.
Trial locations and contacts
4 sites in 1 country.
Australia4 sites
St Vincent's Hospital Melbourne
Fitzroy, Victoria, 3065
Women's and Children's Hospital Adelaide
North Adelaide, South Australia, 5006
Royal Melbourne Hospital
Parkville, Victoria, 3050
Royal Children's Hospital Melbourne
Parkville, Victoria, 3052
About this study
Official title: A Phase 2, Randomised, Placebo Controlled Study Investigating the Efficacy of Baricitinib in New Onset Type 1 Diabetes Mellitus
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.
- Conditions
- Type 1 Diabetes
- Being tested
- Baricitinib, Placebo
- Keywords
- newly diagnosed
- Medical subject headings
- Diabetes Mellitus, Type 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BaricitinibDrug
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.
PlaceboDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Baricitinib | Active comparator | 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 Receives: Drug: Baricitinib |
| Placebo | Placebo comparator | One placebo tablet once daily for a duration of 48 weeks. Placebo tablets contain lactose monohydrate, microcrystalline cellulose, croscarmellose sodium and magnesium stearate. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary endpoint of the study is the change from baseline of plasma C-peptide area under the curve (AUC) over 2 hours following a mixed meal.
Measured: Measured at 48 weeks post commencement of intervention.
Secondary outcomes (7)
Change from baseline in plasma C-peptide AUC over 2 hours following a mixed meal.
Measured: Measured at weeks 12, 24, 72 and 96 post commencement of intervention.
Change from baseline in mean daily insulin use over 7 consecutive days.
Measured: Measured during the 2 weeks prior to the assessment at weeks 12, 24, 48, 72 and 96 post commencement of intervention.
Change from baseline in glycosylated haemoglobin (HbA1c) levels.
Measured: Measured at weeks 12, 24, 48, 72 and 96 post commencement of intervention.
Number of participants with responder status. Responder status is defined as glycosylated haemoglobin (HbA1c) less than or equal to 6.5 percent and mean daily insulin use less than 0.5 international units per kilogram per day (IU/kg/day).
Measured: Measured at weeks 12, 24, 48, 72 and 96 post commencement of intervention.
Change in estimated C-peptide (CPEST) from baseline. CPEST is calculated based on six variable routine measures: disease duration, BMI, insulin dose, HbA1c, fasting plasma C-peptide and fasting plasma glucose.
Measured: Measured at weeks 12, 24, 48, 72 and 96 post commencement of intervention.
Continuous glucose monitoring (CGM).
Measured: Measured at baseline, 12, 24, 48 and 96 weeks post commencement of intervention.
The composite outcome assessing both the frequency, and when relevant, the severity of all adverse events.
Measured: Adverse events will be monitored throughout the entire study duration at weeks 0, 2, 4, 8, 12, 16, 20, 24, 36, 48, 72 and 96.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Mar 2021
- Study start30 Oct 2020
- Primary completion30 Jan 2023
- Study completion6 May 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Type 1 DiabetesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Randomised placebo controlled trial
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 91 participants (actual)
Sponsor and investigators
- Lead sponsor
- St Vincent's Institute of Medical Research
- Sponsor type
- University, hospital or other
- Collaborators
- Juvenile Diabetes Research Foundation Australia, Juvenile Diabetes Research Foundation
- Responsible party
- Sponsor
- Investigators
- Tom Kay, Prof, Principal investigator, SVI
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04774224
- Sponsor's protocol ID
- SVI-BARI-01
- Acronym
- BANDIT
- Status last verified
- Nov 2024
- First submitted
- 22 Feb 2021
- Last updated
- 3 Dec 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: All of the individual participant data collected during the trial, after de-identification.
Publications
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.
PubMed 35606820Waibel 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.
PubMed 38055252
Study at a glance
From the registry record, last updated by the sponsor on 3 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomised, Placebo Controlled Study Investigating the Efficacy of Baricitinib in New Onset Type 1 Diabetes Mellitus
- ClinicalTrials.gov ID
- NCT04774224
- Acronym / sponsor ID
- BANDIT / SVI-BARI-01
- Conditions
- Type 1 Diabetes
- Keywords
- newly diagnosed
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 91 participants (actual)
- Lead sponsor
- St Vincent's Institute of Medical ResearchUniversity, hospital or other
- Collaborators
- Juvenile Diabetes Research Foundation Australia, Juvenile Diabetes Research Foundation
- Study start
- 30 Oct 2020 (actual)
- Primary completion
- 30 Jan 2023 (actual)
- Study completion
- 6 May 2024 (actual)
- First posted
- 1 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Randomised placebo controlled trial
| Arm | Type | Receives |
|---|---|---|
| Baricitinib | Active comparator | Drug: BaricitinibBaricitinib 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 |
| Placebo | Placebo comparator | Drug: PlaceboOne placebo tablet once daily for a duration of 48 weeks. Placebo tablets contain lactose monohydrate, microcrystalline cellulose, croscarmellose sodium and magnesium stearate. |
Who may qualify
- Ages
- 10 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Male or female aged between 10 and 30 years (inclusive) at screening;
- Diagnosis of T1D according to ADA criteria within 100 days prior to starting study drug;
- Islet autoantibody positivity (one or more of: GADA, IA-2A, IAA (assessed within one week of commencing insulin therapy), ZnT8A);
- 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.
- Participants of childbearing age who are sexually active must agree to use of effective birth control until the end of the study;
Key exclusion criteria (22 listed)
- Use of immunosuppressive or immunomodulatory therapies other than inhaled or topical glucocorticoids;
- Current or past history of deep vein thrombosis or pulmonary embolism;
- Impaired renal function defined by estimated glomerular filtration rate (according to the CKD-EPI) of < 60 mL/min/1.73 m2;
- LDL cholesterol >4mmol/l;
- Elevated liver function tests at screening:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BaricitinibParticipants 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. | Drug | Baricitinib |
| PlaceboParticipants 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. | Drug | Placebo |
Primary outcome
The primary endpoint of the study is the change from baseline of plasma C-peptide area under the curve (AUC) over 2 hours following a mixed meal.
Time frame: Measured at 48 weeks post commencement of intervention.
7 secondary outcomes
Change from baseline in plasma C-peptide AUC over 2 hours following a mixed meal.
Time frame: Measured at weeks 12, 24, 72 and 96 post commencement of intervention.
Change from baseline in mean daily insulin use over 7 consecutive days.
Time frame: Measured during the 2 weeks prior to the assessment at weeks 12, 24, 48, 72 and 96 post commencement of intervention.
Change from baseline in glycosylated haemoglobin (HbA1c) levels.
Time frame: Measured at weeks 12, 24, 48, 72 and 96 post commencement of intervention.
Number of participants with responder status. Responder status is defined as glycosylated haemoglobin (HbA1c) less than or equal to 6.5 percent and mean daily insulin use less than 0.5 international units per kilogram per day (IU/kg/day).
Time frame: Measured at weeks 12, 24, 48, 72 and 96 post commencement of intervention.
Change in estimated C-peptide (CPEST) from baseline. CPEST is calculated based on six variable routine measures: disease duration, BMI, insulin dose, HbA1c, fasting plasma C-peptide and fasting plasma glucose.
Time frame: Measured at weeks 12, 24, 48, 72 and 96 post commencement of intervention.
Continuous glucose monitoring (CGM).
Time frame: Measured at baseline, 12, 24, 48 and 96 weeks post commencement of intervention.
The composite outcome assessing both the frequency, and when relevant, the severity of all adverse events.
Time frame: Adverse events will be monitored throughout the entire study duration at weeks 0, 2, 4, 8, 12, 16, 20, 24, 36, 48, 72 and 96.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Dec 2024.
- Background: PubMed 35606820Waibel 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.
- Results publication: PubMed 38055252Waibel 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.
- Trial websiteLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. All of the individual participant data collected during the trial, after de-identification.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Tom Kay, Prof principal investigator
SVI
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Baricitinib in people with Type 1 Diabetes, run by St Vincent's Institute of Medical Research at 4 sites in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The primary endpoint of the study is the change from baseline of plasma C-peptide area under the curve (AUC) over 2 hours following a mixed meal. (time frame: Measured at 48 weeks post commencement of intervention.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04774224, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Dec 2024; AzTrials last refreshed its copy on 6 Nov 2026.