Diabetes Mellitus, Type 1Phase 1/2NCT03243058
Low-dose IL-2 in Established T1D - The "PROREG" Study
A Phase 1/2 study testing ILT-101 (Aldesleukin; IL-2) in people with Diabetes Mellitus, Type 1 and Diabetes, run by Jay S. Skyler at 1 site in United States.
Trial summary
Randomized, controlled, double-blinded, multicenter, phase I/II clinical trial to evaluate the safety of low-dose IL-2 and determine whether low-dose IL-2 therapy for one year, can prevent further loss of beta-cell function in patients with established T1D, (primary outcome). Read more about this study
- Who is it for?
- Ages 8 to 21 years
- Conditions the study is for
- Diabetes Mellitus, Type 1, Diabetes, Diabetes, Autoimmune
- What is being tested?
- ILT-101 (Aldesleukin; IL-2)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2023to Dec 2028
- Phase
- Phase 1/2
- 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 8 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 8-21 years of age
- T1D, demonstrated by at least one islet autoantibody
- T1D duration 4-12 months at the time of the first dose
- Peak stimulated C-peptide >0.2 nmol/L during a 4-hour MMTT
Who cannot join
- Treatment with oral anti-diabetic agents
- Illnesses that would preclude use of low-dose IL-2
Trial locations and contacts
1 site in 1 country.
United States1 site
Diabetes Research Institute, University of Miami Miller School of Medicine
Miami, Florida, 33136
About this study
Official title: A Randomized, Double Blind, Phase I/II Trial of Low-Dose Interlekin-2 Immunotherapy in Established Type 1 Diabetes
Randomized, controlled, double-blinded, multicenter, phase I/II clinical trial to evaluate the safety of low-dose IL-2 and determine whether low-dose IL-2 therapy for one year, can prevent further loss of beta-cell function in patients with established T1D, (primary outcome). The study will carefully examine various effects of low-dose IL-2 on the immune system in patients with T1D, including effects on Treg and other cell subsets, and disease-specific autoimmune responses.
Read the detailed description
Randomized, controlled, double-blinded, multicenter, phase I/II clinical trial to evaluate the safety of low-dose IL-2 and determine whether low-dose IL-2 therapy for one year, can prevent further loss of beta-cell function in patients with established T1D, (primary outcome). The study will carefully examine various effects of low-dose IL-2 on the immune system in patients with T1D, including effects on Treg and other cell subsets, and disease-specific autoimmune responses.
Patients will be treated with ILT-101 or placebo. ILT-101 will be given at doses of 0.5 million IU (body surface area <2 m2) or 1 million IU (body surface area >2 m2), via subcutaneous (s.c.) injections. Patients will receive a course of 5 daily injections (days 1-5. Starting on day 15, patients will receive an s.c. injection (same dose) every 15 days for 1 year. Thus, patients will receive 29 injections during the first year of treatment. At the end of the first year, approximately half of those randomized to ILT-101 will continue receiving treatment every 15 days, until the end of the second year (23 doses). The other half will stop therapy and will be switched to a placebo.
A group of patients will be randomly assigned to a placebo for the duration of the study. Patients to be included in this study are those diagnosed with T1D who would have had T1D from 4 months to 1 year at the time of randomization, who have a current or past demonstration of autoimmunity (using autoantibodies), and maintain preserved β-cell function, defined as an MMTT stimulated C-peptide >0.2 nmol/L. This population is chosen because it will extend the scope of therapy beyond the immediate time following diagnosis when most previous studies of immunotherapy in T1D have been conducted. This trial can further impact the field if a therapeutic benefit is shown when the disease is more established.
- Conditions
- Diabetes Mellitus, Type 1, Diabetes, Diabetes, Autoimmune
- Being tested
- ILT-101 (Aldesleukin; IL-2), Treatment-Placebo Arm, Placebo Arm
- Keywords
- Diabetes, Type 1 Diabetes, Autoimmune, Diabetes, Autoimmune, Interleukin-2, Interleukin, IL-2
- 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.
ILT-101 (Aldesleukin; IL-2)Biological
Also called IL-2, Interleukin-2
Administration of Low-Dose Interleukin-2 (ILT-101) for two years
Treatment-Placebo ArmOther
Administration of Low-Dose Interleukin-2 (ILT-101) for one year, and then placebo for the second year.
Placebo ArmOther
Participants in this group will receive a placebo injection for two years.
| Study group | Role | What they receive |
|---|---|---|
| Treatment Arm | Active comparator | ILT-101 (Aldesleukin; IL-2), 0.5 million IU/m2 (up to a maximum of 1 million IU), or placebo, will be given for 5 consecutive days (days 1-5), and then on day 15 and every 15 days thereafter, for two years. Receives: Biological: ILT-101 (Aldesleukin; IL-2) |
| Treatment-Placebo Arm | Experimental | Participants in this group will receive study drug ILT-101 for one year, and then placebo for the second year. Receives: Biological: ILT-101 (Aldesleukin; IL-2); Other: Treatment-Placebo Arm |
| Placebo | Placebo comparator | Participants in this group will receive a placebo injection for two years. Receives: Other: Placebo Arm |
What is being measured
The primary outcome is the main question the study is designed to answer.
C-peptide response
Preservation of insulin secretion (measured as c-peptide nmol/L) based on stimulated area under the curve (AUC) during a 4-hour MMTT at 1 year
Measured: 1 year primary outcome
Secondary outcomes (1)
C-peptide response
Preservation of insulin secretion (measured as c-peptide nmol/L) based on stimulated area under the curve (AUC) during a 4-hour MMTT at 2 years
Measured: 2 year secondary outcome
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2017
- Study startJun 2023 (estimated)
- Primary completionDec 2026 (estimated)
- Study completionDec 2028 (estimated)
Why it stopped: Funding withdrawal and COVID-19 environment.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Diabetes Mellitus, Type 1Studies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- ILT-101 (aldesleukin; Interleukin-2, IL-2), 0.5 million IU (body surface area \<2 m2) or 1 million IU (body surface area \>2 m2), or placebo, will be given for 5 consecutive days (days 1-5), and then on day 15 and every 15 days thereafter, for one year; at the one-year endpoint, approximately half the subjects on ILT-101 will continue therapy and the other half will switch to placebo for 1 more year.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jay S. Skyler
- Sponsor type
- University, hospital or other
- Collaborators
- Diabetes Research Institute Foundation, University of Florida, University of California, San Francisco
- Responsible party
- Sponsor-investigator, Jay S. Skyler, University of Miami
- Investigators
- Jay S Skyler, M.D., Principal investigator, Professor of MedicineAlberto Pugliese, M.D., Principal investigator, Professor of MedicineDavid A Baidal, M.D., Principal investigator, Assistant Professor of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03243058
- Sponsor's protocol ID
- 20170301
- Other IDs
- 20170301
- Status last verified
- May 2024
- First submitted
- 20 Jul 2017
- Last updated
- 22 May 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: A participant's privacy and confidentiality will be respected throughout the study. Each participant will be assigned a unique identification number and these numbers rather than names will be used to collect, store, and report participant information. Site personnel will not transmit documents containing personal health identifiers (PHI) to the study sponsor or their representatives.
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double Blind, Phase I/II Trial of Low-Dose Interlekin-2 Immunotherapy in Established Type 1 Diabetes
- ClinicalTrials.gov ID
- NCT03243058
- Acronym / sponsor ID
- 20170301
- Conditions
- Diabetes Mellitus, Type 1, Diabetes, Diabetes, Autoimmune
- Keywords
- Diabetes, Type 1 Diabetes, Autoimmune, Diabetes, Autoimmune, Interleukin-2, Interleukin, IL-2
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Funding withdrawal and COVID-19 environment.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Jay S. SkylerUniversity, hospital or other
- Collaborators
- Diabetes Research Institute Foundation, University of Florida, University of California, San Francisco
- Study start
- Jun 2023 (estimated)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 8 Aug 2017 (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.
Randomized, controlled, double-blinded, multicenter, phase I/II clinical trial to evaluate the safety of low-dose IL-2 and determine whether low-dose IL-2 therapy for one year, can prevent further loss of beta-cell function in patients with established T1D, (primary outcome). The study will carefully examine various effects of low-dose IL-2 on the immune system in patients with T1D, including effects on Treg and other cell subsets, and disease-specific autoimmune responses.
Detailed description
Randomized, controlled, double-blinded, multicenter, phase I/II clinical trial to evaluate the safety of low-dose IL-2 and determine whether low-dose IL-2 therapy for one year, can prevent further loss of beta-cell function in patients with established T1D, (primary outcome). The study will carefully examine various effects of low-dose IL-2 on the immune system in patients with T1D, including effects on Treg and other cell subsets, and disease-specific autoimmune responses.
Patients will be treated with ILT-101 or placebo. ILT-101 will be given at doses of 0.5 million IU (body surface area <2 m2) or 1 million IU (body surface area >2 m2), via subcutaneous (s.c.) injections. Patients will receive a course of 5 daily injections (days 1-5. Starting on day 15, patients will receive an s.c. injection (same dose) every 15 days for 1 year. Thus, patients will receive 29 injections during the first year of treatment. At the end of the first year, approximately half of those randomized to ILT-101 will continue receiving treatment every 15 days, until the end of the second year (23 doses). The other half will stop therapy and will be switched to a placebo.
A group of patients will be randomly assigned to a placebo for the duration of the study. Patients to be included in this study are those diagnosed with T1D who would have had T1D from 4 months to 1 year at the time of randomization, who have a current or past demonstration of autoimmunity (using autoantibodies), and maintain preserved β-cell function, defined as an MMTT stimulated C-peptide >0.2 nmol/L. This population is chosen because it will extend the scope of therapy beyond the immediate time following diagnosis when most previous studies of immunotherapy in T1D have been conducted. This trial can further impact the field if a therapeutic benefit is shown when the disease is more established.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
- Model description
- ILT-101 (aldesleukin; Interleukin-2, IL-2), 0.5 million IU (body surface area <2 m2) or 1 million IU (body surface area >2 m2), or placebo, will be given for 5 consecutive days (days 1-5), and then on day 15 and every 15 days thereafter, for one year; at the one-year endpoint, approximately half the subjects on ILT-101 will continue therapy and the other half will switch to placebo for 1 more year.
| Arm | Type | Receives |
|---|---|---|
| Treatment Arm | Active comparator | Biological: ILT-101 (Aldesleukin; IL-2)ILT-101 (Aldesleukin; IL-2), 0.5 million IU/m2 (up to a maximum of 1 million IU), or placebo, will be given for 5 consecutive days (days 1-5), and then on day 15 and every 15 days thereafter, for two years. |
| Treatment-Placebo Arm | Experimental | Biological: ILT-101 (Aldesleukin; IL-2), Other: Treatment-Placebo ArmParticipants in this group will receive study drug ILT-101 for one year, and then placebo for the second year. |
| Placebo | Placebo comparator | Other: Placebo ArmParticipants in this group will receive a placebo injection for two years. |
Who may qualify
- Ages
- 8 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 8-21 years of age
- T1D, demonstrated by at least one islet autoantibody
- T1D duration 4-12 months at the time of the first dose
- Peak stimulated C-peptide >0.2 nmol/L during a 4-hour MMTT
Key exclusion criteria (2 listed)
- Treatment with oral anti-diabetic agents
- Illnesses that would preclude use of low-dose IL-2
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 |
|---|---|---|
| ILT-101 (Aldesleukin; IL-2)Also: IL-2, Interleukin-2Administration of Low-Dose Interleukin-2 (ILT-101) for two years | Biological | Treatment Arm, Treatment-Placebo Arm |
| Treatment-Placebo ArmAdministration of Low-Dose Interleukin-2 (ILT-101) for one year, and then placebo for the second year. | Other | Treatment-Placebo Arm |
| Placebo ArmParticipants in this group will receive a placebo injection for two years. | Other | Placebo |
Primary outcome
C-peptide response
Time frame: 1 year primary outcome
Preservation of insulin secretion (measured as c-peptide nmol/L) based on stimulated area under the curve (AUC) during a 4-hour MMTT at 1 year
1 secondary outcome
C-peptide response
Time frame: 2 year secondary outcome
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2024.
- Individual participant dataSharing planned: No. A participant's privacy and confidentiality will be respected throughout the study. Each participant will be assigned a unique identification number and these numbers rather than names will be used to collect, store, and report participant information. Site personnel will not transmit documents containing personal health identifiers (PHI) to the study sponsor or their representatives.
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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.
Jay S Skyler, M.D. principal investigator
Professor of Medicine
Alberto Pugliese, M.D. principal investigator
Professor of Medicine
David A Baidal, M.D. principal investigator
Assistant Professor of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ILT-101 (Aldesleukin; IL-2) in people with Diabetes Mellitus, Type 1 and Diabetes, run by Jay S. Skyler at 1 site in United States.
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 4 inclusion and 2 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: withdrawn, as of the sponsor's last update on 22 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 was withdrawn before enrolling participants, so there are no results.
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?
C-peptide response (time frame: 1 year primary outcome). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 22 May 2024.