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Diabetes Mellitus, Type 1Phase 2MONORAPANCT02803892

Monotherapy With Rapamycin in Long-standing Type 1 Diabetes

A Phase 2 study testing rapamycin and Vildagliptin in people with Diabetes Mellitus, Type 1, run by Piemonti Lorenzo at 1 site in Italy.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This study is a phase 2, single-center, prospective, randomized, double-blind, placebo-controlled, 3-arm parallel group (1:1:1) intervention trial to determine the efficacy of 4 weeks rapamycin treatment and 4 weeks rapamycin treatment plus 3 months vildagliptin treatment versus placebo in increasing endogenous… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Diabetes Mellitus, Type 1
What is being tested?
rapamycin and Vildagliptin
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
55 participants
Where
1 siteItaly
Study dates
May 2016to Mar 2019
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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Male or female aged >18 years, inclusive
  • Clinical history compatible with T1D with onset of disease at < 40 years of age, insulin dependence for ≥ 5 years at the time of enrolment
  • C-peptide concentrations under the threshold of preserved beta cell function: fasting C peptide <0.23 ng/ml
  • Detectable fasting proinsulin concentrations (>0.5 pmol/l)
  • Ability to provide written informed consent
  • Mentally stable and able to comply with the protocol procedures for the duration of the study, including scheduled follow-up visits and examinations

Who cannot join

  • Body mass index (BMI) >30 kg/m2 or patient with body weight ≤40kg;
  • Insulin requirement >1.0 IU/kg/day or <10 U/day;
  • HbA1c >11% (normal value: 3.5-6.0%) at the time of enrolment
  • estimated glomerular filtration rate <60 mL/min/1.73m2 calculated using the subject's measured serum creatinine and the Modification of Diet in Renal Disease [MDRD] study estimation formula)
  • Presence or history of macroalbuminuria (>300mg/g creatinine)
  • For female subjects: positive pregnancy test, presently breast-feeding, or unwillingness to use effective contraceptive measures for the duration of the study and 4 months after discontinuation of treatment
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  • Active infection including hepatitis B, hepatitis C, HIV, or tuberculosis (TB) as determined by a positive skin test or clinical presentation, or under treatment for suspected TB
  • Any history of malignancy except for completely resected squamous or basal cell carcinoma of the skin
  • Lymphopenia (<1,000/μL), neutropenia (<1,500/μL), or thrombocytopenia (platelets <100,000/μL).
  • Severe unremitting diarrhea, vomiting or other gastrointestinal disorders potentially interfering with the ability to absorb oral medications
  • Any medical condition that will interfere with safe participation in the trial;
  • Any immunosuppressive treatment at the time of enrollment.
  • Allergy to active ingredients or to any of excipients

Trial locations and contacts

1 site in 1 country.

Italy1 site

  • IRCCS San Raffaele Scientific Institute

    Milan, 20132

Other trials in Italy

About this study

Official title: Evaluation of the Efficacy of Rapamycin and a Dipeptidyl Peptidase-4 Inhibitor (Vildagliptin) in Improving Beta Cell Function in Type 1 Diabetes of Long Duration, a Perspective Randomized Study

This study is a phase 2, single-center, prospective, randomized, double-blind, placebo-controlled, 3-arm parallel group (1:1:1) intervention trial to determine the efficacy of 4 weeks rapamycin treatment and 4 weeks rapamycin treatment plus 3 months vildagliptin treatment versus placebo in increasing endogenous insulin production and correcting glycemic lability. It will involve 60 patients with long standing type 1 diabetes (T1D). Patients will receive for one month placebo (Group 1), rapamycin plus placebo (Group 2), or rapamycin plus Vildagliptin (Group 3). Rapamycin will be administered at an initial dose 0.2 mg/kg orally on day 0 followed by 0.1 mg/kg/die (target trough levels: 8-10 ng/ml). Vildagliptin will be administered at a dose of 50 mg x2/die starting from day 0. After 4 weeks of treatment (period A), patients will discontinue rapamycin or relevant placebo treatment, but continue Vildagliptin or placebo for a further 8 weeks and be monitored over this period (period B).

Being tested
rapamycin, Vildagliptin, Placebo 1, Placebo 2
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.

  • rapamycinDrug

    Also called Rapamune®

    Rapamycin will be administered at an initial dose 0.2 mg/kg on day 0, followed by 0.1 mg/kg/die. The daily dose will be adjusted to the whole blood 24-hr trough to target, as tolerated, 8-10 ng/mL

  • VildagliptinDrug

    Also called GALVUS

    Vildagliptin will be administered at a dose of 50 mg x2/die starting from day 0.

  • Placebo 1Drug

    Placebo 1 will be titrated according to a random schedule alternating plausible doses of placebo. After 4 weeks of treatment patients will discontinue placebo 1

  • Placebo 2Drug

    Placebo 2 will be administered BID starting from day 0. After 8 weeks of treatment patients will discontinue placebo 2

Study groups in NCT02803892
Study groupRoleWhat they receive
Group 1: PlaceboPlacebo comparatorEligible participants will be randomized to one of three treatment arms. In this arm patients will received placebo x 2 placebo (Group 1) After 4 weeks of treatment, patients will discontinue relevant placebo treatment, but continue the second placebo for a further 8 weeks
Receives: Drug: Placebo 1; Drug: Placebo 2
Group 2: Rapamycin plus PlaceboExperimentalEligible participants will be randomized to one of three treatment arms. In this arm patients will received rapamycin plus placebo. After 4 weeks of treatment, patients will discontinue rapamycin, but continue the second placebo for a further 8 weeks
Receives: Drug: rapamycin; Drug: Vildagliptin
Group 3: Rapamycin plus VildagliptinExperimentalEligible participants will be randomized to one of three treatment arms. In this arm patients will received rapamycin plus vildagliptin. After 4 weeks of treatment, patients will discontinue rapamycin , but continue Vildagliptin o for a further 8 weeks
Receives: Drug: rapamycin; Drug: Vildagliptin

What is being measured

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

  1. Change from Baseline C-peptide response in the MMTT

    the proportion of participants with a positive response to the MMTT defined as C-peptide at 90 min >0.6 ng/ml.

    Measured: week 4±1, week 12±2

  2. Change from Baseline C-peptide after the MMTT

    change in the area under the curve of C-peptide after the MMTT vs baseline

    Measured: week 4±1, week 12±2

Secondary outcomes (5)

  1. Change from Baseline insulin requirement

    change in insulin requirement vs baseline

    Measured: week 4±1, week 12±2

  2. Change from Baseline fasting C-peptide

    change in fasting C-peptide vs baseline

    Measured: week 4±1, week 12±2

  3. Change from Baseline HbA1c

    change in HbA1c vs baseline

    Measured: week 4±1, week 12±2

  4. Adverse Events (AEs) related to the immunosuppression

    the incidence and severity of Adverse Events (AEs) related to the immunosuppressive treatment

    Measured: week 4±1, week 12±2

  5. Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Measured: week 4±1, week 12±2

Timeline

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

  1. First posted17 Jun 2016 (estimated)
  2. Study startMay 2016
  3. Primary completionDec 2018
  4. Study completionMar 2019

Resources

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
Masking
Double blind
Who is masked
Participant, Care provider
Enrolment
55 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02803892
Sponsor's protocol ID
DRI-2/2014 MONORAPA
Acronym
MONORAPA
Status last verified
Nov 2020
First submitted
10 Jun 2016
Last updated
4 Nov 2020
Results posted
No
Individual data sharing
Yes

Publications

  • Bolla AM, Gandolfi A, Borgonovo E, Laurenzi A, Caretto A, Molinari C, Catalano RS, Bianconi E, Monti P, Sordi V, Pellegrini S, Lampasona V, Costa S, Scavini M, Bosi E, Piemonti L. Rapamycin Plus Vildagliptin to Recover beta-Cell Function in Long-Standing Type 1 Diabetes: A Double-Blind, Randomized Trial. J Clin Endocrinol Metab. 2021 Jan 23;106(2):e507-e519. doi: 10.1210/clinem/dgaa791.

    PubMed 33124663

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing rapamycin and Vildagliptin in people with Diabetes Mellitus, Type 1, run by Piemonti Lorenzo at 1 site in Italy.

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 6 inclusion and 13 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 4 Nov 2020. 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 Italy. 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?

Change from Baseline C-peptide response in the MMTT (time frame: week 4±1, week 12±2). 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 NCT02803892, which also lists 1 linked publication.

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 4 Nov 2020; AzTrials last refreshed its copy on 3 Nov 2026.

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