Type 1 Diabetes MellitusPhase 2/3UST1D2NCT03941132

Clinical Phase II/III Trial of Ustekinumab to Treat Type 1 Diabetes (UST1D2)

A Phase 2/3 study testing Ustekinumab in people with Type 1 Diabetes Mellitus, run by University of British Columbia at 2 sites in Canada.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record

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Trial summary

Who is it for?: Ages 18 to 35 years
Conditions the study is for: Type 1 Diabetes Mellitus
What is being tested?: Ustekinumab
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 66 participants
Where: 2 sitesCanada
Study dates: 4 Jan 2021to 1 Nov 2026
Phase: Phase 2/3
Are there results?: Not yet: the study is under way

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 18 to 35 years
All sexes
No healthy volunteers
Adult

Who may be able to join

A diagnosis of type 1 diabetes mellitus in accordance with the ADA/CDA criteria.
An interval of ≤100 days between the diagnosis and the first dose of the study drug.
Ability to provide documented informed consent.
Male or female, aged 18-35 years inclusive, at the time of the anticipated first dose of the study drug.
Evidence of residual functioning β cells. This will be assessed by a C-peptide level over 0.2nmol/L in the MMTT test.
Positive for at least one diabetes-related autoantibody.
Willing to record all insulin taken and blood glucose levels that are required for monitoring during the study, including reporting any hypoglycaemic events.

Who cannot join

No condition that, in the investigators' judgment, is likely to cause the subject to not be able to understand information in order to provide informed consent.
History of malignancy.
No significant and/or active disease in any body system that is likely to increase the risk to the subject or interfere with the subject's participation in the study.
No significant systemic infection during the 6 weeks before the first dose of the study drug.
No history of current or past active tuberculosis infection and no latent tuberculosis as per CDC guidelines.
Have used any other investigational drug within the 3 months prior to the first dose and/or intend on using any investigational drug for the duration of the study.

Show all 15Show fewer

Prior or current treatment that is known to cause a significant, ongoing change in the course of T1D or immunological status.
Current or prior (within 30 days prior to first study drug dose) use of medications known to influence glucose tolerance.
No significant abnormal laboratory values during the screening period, other than those due to T1D.
Not pregnant, breastfeeding or planning to become pregnant during the 60 days after the last dose of the study drug.
Have not received any live vaccines within 30 days prior to the first study drug dose and are not expected to need to receive a vaccine during the study.
No prior allergic reaction, including anaphylaxis, to any component of the study drug product.
No prior allergic reaction, including anaphylaxis, to any human, humanized, chimeric or rodent antibody treatment.
Have not undergone any major surgery within the 30 day period prior to the first drug dose and not anticipating requiring surgery during the study period.
Negative results for Hepatitis B surface antigen and for antibodies to Hepatitis B core antigen, or evidence of Hepatitis B surface antibody > 10 IU, and negative for Hepatitis C. Negative results for HIV and not considered by the investigator to be at high risk for HIV infection.

Trial locations and contacts

2 sites in 1 country.

Canada2 sites

Mount Sinai Hospital/UHN
Toronto, British Columbia, M5T 3L9
BCDiabetes
Vancouver, British Columbia, V5Y 3W2

Other trials in Canada

About this study

In type 1 diabetes (T1D), immune defense cells in the body attack and destroy insulin-producing beta cells leaving affected people with a lifelong need for daily insulin injections. Even with insulin injections, blood glucose (sugar) control is imperfect and leads to many health complications and a shortened life span. Our pilot study (NCT02117765) has informed us that Ustekinumab is safe in the treatment of participants with recent-onset T1D. Ustekinumab is currently licensed for use in psoriasis where it has proven to be both highly effective and safe. The investigators hope that if the drug can block immune cells soon after the development of diabetes, any remaining insulin-producing cells may be protected, and regenerate, thus producing more insulin so that individuals may be insulin free, or require less insulin. This trial will assess the efficacy of Ustekinumab in decreasing C-peptide decline (proxy for endogenous insulin production) in participants with recent onset T1D.

Read the detailed description

This is a randomized, placebo-controlled, double-blinded, multi-centre phase II/II study to assess efficacy and safety of Ustekinumab (STELARA®) in patients with T1D. The investigators will perform a phase II/III clinical trial with a total of 66 adult (18-35 years old) subjects with recent-onset T1D. There will be two study cohorts, with a drug:placebo ratio of 2:1. Patients receiving the study drug will receive a loading dose of 6mg/kg Ustekinumab IV given at week 0. Thereafter, 90mg Ustekinumab subcutaneously given at weeks 8, 16, 24, 32, 40, 48 (total of 7 doses). Patients randomized to receive placebo will receive respective amounts of a saline-placebo. An additional non-dosing visit at the midpoint (week 28) is required to measure 2-hour C-peptide during a MMTT. Patients will be followed for 78 weeks following the first dose. There will be a total of 10 study visits over 78 weeks, three of which are non-dosing and follow-up visits. Recruitment and screening for the study will be completed within the first 24 months. The follow up period is 1 and 1.5 years from the first dose.

Conditions: Type 1 Diabetes Mellitus
Being tested: Ustekinumab, Placebo
Keywords: T1D, Ustekinumab, new-onset, diabetes
Medical subject headings: Diabetes Mellitus, Type 1, Diabetes Mellitus

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

UstekinumabDrug
Also called Stelara
Week 0: Loading dose of 6mg/kg Ustekinumab Intravenously.
Weeks 8, 16, 24, 32, 40, and 48 (6 visits): 90mg Ustekinumab subcutaneously.
PlaceboDrug
Also called Saline solution
Patients allocated to receive placebo will receive respective amounts of a saline-placebo at the same intervals.
Week 0: Loading dose of 6mg/kg saline intravenously.
Weeks 8, 16, 24, 32, 40, and 48 (6 visits): 90mg saline subcutaneously.

Study groups in NCT03941132
Study group	Role	What they receive
Ustekinumab	Experimental	Week 0: Loading dose of 6mg/kg Ustekinumab Intravenously. Weeks 8, 16, 24, 32, 40, and 48 (6 visits): 90mg Ustekinumab subcutaneously. Weeks 28, 52, 78: Non-dosing visits where a Mixed Meal Tolerance Test will be administered. Total of 11 visits Receives: Drug: Ustekinumab
Saline Solution - Placebo	Placebo comparator	Patients allocated to receive placebo will receive respective amounts of a saline-placebo at the same intervals. Week 0: Loading dose of 6mg/kg saline intravenously. Weeks 8, 16, 24, 32, 40, and 48 (6 visits): 90mg saline subcutaneously. Weeks 28, 52, 78: Non-dosing visits where a Mixed Meal Tolerance Test will be administered. Total of 11 visits Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Baseline change in 2-hour mixed meal-stimulated C-peptide AUC at week 52.
Measured: Week 52
Rate, frequency and severity of all adverse events including; hypoglycemic episodes; injection reactions; hypersensitivity reactions; evidence of infection and posterior leukoencephalopathy syndrome.
Measured: Week 52

Secondary outcomes (13)

2-hour MMTT-stimulated C-peptide AUC at weeks 28 and 78)
Measured: Weeks 28 and 78
HbA1C and insulin use in units per kg body weight per day at weeks 0, 8, 16, 24, 28, 32, 40, 48, 52, 78.
Measured: 78 Weeks
Immune phenotyping via flow cytometry of all IL-12, IL-23, IL-17, IFN-γ secreting immune subsets at weeks 0, 32, 52, 78).
Measured: 78 Weeks
Basic immune phenotyping of WBC subsets
Measured: 78 Weeks
HLA- A, B, C, DR, DP, DQ typing at weeks 0, 8 ,16, 32, 52, 78)
Measured: 78 Weeks
Fluorospot (ELISpot) analysis for IL-17 and IFN-γ secretion in response to whole insulin and antigens for CD8+ and CD4+ T cells.
Measured: 78 Weeks
Luminex/Mesoscale assessment of serum cytokines IL-17, IFN-γ, IL-12p40, IL-12p70 and IL-23.
Measured: 78 Weeks
Regulatory T cell (CD4+ FOXP3+): Effector T cell (CD4+ FOXP3-CD25+) ratio.
Measured: 78 Weeks
CD154 and CD134 (OX40) based assays to determine diabetogenic antigen specific responses of T helper cells.
Measured: 52 Weeks
Nanostring assessment of whole blood and PBMC RNA gene expression of IL-17 and IFN-γ family genes.
Measured: 78 Weeks
Epigenetic assessment of Treg phenotype and function.
Measured: 78 Weeks
Sequencing and profiling of microbiome.
Measured: 78 Weeks
Glycaemic variability in continuous glucose monitoring and hypoglycaemia rates.
Measured: 78 Weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

4 Jan 20211 Nov 2026

First posted7 May 2019
Study start4 Jan 2021
Primary completion1 Feb 2026 (estimated)
Study completion1 Nov 2026 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Type 1 Diabetes MellitusStudies of the same condition, open and under way.
More trials run by University of British ColumbiaThe sponsor's other registered studies.
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 2/3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Model description: There will be a total of 60 patients enrolled in the study. The study participants will be enrolled at 2 centers (Vancouver and Toronto), and will be referred by adult or pediatric endocrinologists. This sample size estimation is based on results from week 52 C-peptide AUC values observed in the pilot UST1D study and the expected 1-year C-peptide decline in adult-onset T1D patients. Using a 2:1 Ustekinumab vs. placebo randomized assignment, a sample size of 60 yields 85% power to detect improvement in C-peptide function (alpha = 0.05) in the Ustekinumab group for an unstratified analysis at 12 months. Sixty-six participants (44 active: 22 placebo) will be recruited to allow for an approximate 10% loss to follow-up.
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 66 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03941132
Sponsor's protocol ID: H19-00411
Acronym: UST1D2
Status last verified: Feb 2025
First submitted: 3 May 2019
Last updated: 24 Feb 2025
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: Undecided

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Type 1 Diabetes Mellitus trials

Study at a glance

From the registry record, last updated by the sponsor on 24 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Clinical Phase II/III Trial of Ustekinumab to Treat Type 1 Diabetes (UST1D2)
ClinicalTrials.gov ID: NCT03941132
Acronym / sponsor ID: UST1D2 / H19-00411
Conditions: Type 1 Diabetes Mellitus
Keywords: T1D, Ustekinumab, new-onset, diabetes
Phase: Phase 2/3
Study type: Interventional
Recruitment status: Active, not recruiting
Enrolment: 66 participants (estimated)
Lead sponsor: University of British ColumbiaUniversity, hospital or other
Collaborators: Juvenile Diabetes Research Foundation, Janssen, LP
Study start: 4 Jan 2021 (actual)
Primary completion: 1 Feb 2026 (estimated)
Study completion: 1 Nov 2026 (estimated)
First posted: 7 May 2019 (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.

Detailed description

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: There will be a total of 60 patients enrolled in the study. The study participants will be enrolled at 2 centers (Vancouver and Toronto), and will be referred by adult or pediatric endocrinologists. This sample size estimation is based on results from week 52 C-peptide AUC values observed in the pilot UST1D study and the expected 1-year C-peptide decline in adult-onset T1D patients. Using a 2:1 Ustekinumab vs. placebo randomized assignment, a sample size of 60 yields 85% power to detect improvement in C-peptide function (alpha = 0.05) in the Ustekinumab group for an unstratified analysis at 12 months. Sixty-six participants (44 active: 22 placebo) will be recruited to allow for an approximate 10% loss to follow-up.

Arm	Type	Receives
Ustekinumab	Experimental	Drug: UstekinumabWeek 0: Loading dose of 6mg/kg Ustekinumab Intravenously. Weeks 8, 16, 24, 32, 40, and 48 (6 visits): 90mg Ustekinumab subcutaneously. Weeks 28, 52, 78: Non-dosing visits where a Mixed Meal Tolerance Test will be administered. Total of 11 visits
Saline Solution - Placebo	Placebo comparator	Drug: PlaceboPatients allocated to receive placebo will receive respective amounts of a saline-placebo at the same intervals. Week 0: Loading dose of 6mg/kg saline intravenously. Weeks 8, 16, 24, 32, 40, and 48 (6 visits): 90mg saline subcutaneously. Weeks 28, 52, 78: Non-dosing visits where a Mixed Meal Tolerance Test will be administered. Total of 11 visits

Who may qualify

Ages: 18 to 35 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult

Key inclusion criteria (7 listed)

A diagnosis of type 1 diabetes mellitus in accordance with the ADA/CDA criteria.
An interval of ≤100 days between the diagnosis and the first dose of the study drug.
Ability to provide documented informed consent.
Male or female, aged 18-35 years inclusive, at the time of the anticipated first dose of the study drug.
Evidence of residual functioning β cells. This will be assessed by a C-peptide level over 0.2nmol/L in the MMTT test.

Key exclusion criteria (15 listed)

No condition that, in the investigators' judgment, is likely to cause the subject to not be able to understand information in order to provide informed consent.
History of malignancy.
No significant and/or active disease in any body system that is likely to increase the risk to the subject or interfere with the subject's participation in the study.
No significant systemic infection during the 6 weeks before the first dose of the study drug.
No history of current or past active tuberculosis infection and no latent tuberculosis as per CDC guidelines.

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
UstekinumabAlso: StelaraWeek 0: Loading dose of 6mg/kg Ustekinumab Intravenously. Weeks 8, 16, 24, 32, 40, and 48 (6 visits): 90mg Ustekinumab subcutaneously.	Drug	Ustekinumab
PlaceboAlso: Saline solutionPatients allocated to receive placebo will receive respective amounts of a saline-placebo at the same intervals. Week 0: Loading dose of 6mg/kg saline intravenously. Weeks 8, 16, 24, 32, 40, and 48 (6 visits): 90mg saline subcutaneously.	Drug	Saline Solution - Placebo

Primary outcome

Baseline change in 2-hour mixed meal-stimulated C-peptide AUC at week 52.
Time frame: Week 52
Rate, frequency and severity of all adverse events including; hypoglycemic episodes; injection reactions; hypersensitivity reactions; evidence of infection and posterior leukoencephalopathy syndrome.
Time frame: Week 52

13 secondary outcomes

2-hour MMTT-stimulated C-peptide AUC at weeks 28 and 78)
Time frame: Weeks 28 and 78
HbA1C and insulin use in units per kg body weight per day at weeks 0, 8, 16, 24, 28, 32, 40, 48, 52, 78.
Time frame: 78 Weeks
Immune phenotyping via flow cytometry of all IL-12, IL-23, IL-17, IFN-γ secreting immune subsets at weeks 0, 32, 52, 78).
Time frame: 78 Weeks
Basic immune phenotyping of WBC subsets
Time frame: 78 Weeks
HLA- A, B, C, DR, DP, DQ typing at weeks 0, 8 ,16, 32, 52, 78)
Time frame: 78 Weeks
Fluorospot (ELISpot) analysis for IL-17 and IFN-γ secretion in response to whole insulin and antigens for CD8+ and CD4+ T cells.
Time frame: 78 Weeks
Luminex/Mesoscale assessment of serum cytokines IL-17, IFN-γ, IL-12p40, IL-12p70 and IL-23.
Time frame: 78 Weeks
Regulatory T cell (CD4+ FOXP3+): Effector T cell (CD4+ FOXP3-CD25+) ratio.
Time frame: 78 Weeks
CD154 and CD134 (OX40) based assays to determine diabetogenic antigen specific responses of T helper cells.
Time frame: 52 Weeks
Nanostring assessment of whole blood and PBMC RNA gene expression of IL-17 and IFN-γ family genes.
Time frame: 78 Weeks
Epigenetic assessment of Treg phenotype and function.
Time frame: 78 Weeks
Sequencing and profiling of microbiome.
Time frame: 78 Weeks
Glycaemic variability in continuous glucose monitoring and hypoglycaemia rates.
Time frame: 78 Weeks

Results and safety

This study is still under way. Results are not available in the source record yet.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 24 Feb 2025.
Individual participant dataSharing planned: Undecided

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Estimated Risk of Preeclampsia in the First Trimester and Its Association With Subclinical Atherosclerosis and Cardiovascular Risk in Women With Type 1 Diabetes
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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.

Jan Dutz, MD FRCPC principal investigator

University of British Columbia

Betina F Rasmussen, MSc study director

University of British Columbia

2 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Ustekinumab in people with Type 1 Diabetes Mellitus, run by University of British Columbia at 2 sites in Canada.

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 15 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: under way, not looking for participants, as of the sponsor's last update on 24 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in Canada. 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 combined 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.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

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?

Baseline change in 2-hour mixed meal-stimulated C-peptide AUC at week 52. (time frame: Week 52). The study lists 2 primary outcomes in all. 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 NCT03941132.

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 24 Feb 2025.