Efficacy of Extracorporeal Photopheresis (ECP) in the Treatment of Type 1 Diabetes Mellitus
- Conditions
- Type 1 Diabetes
- Interventions
- Combination Product: ECP regular-intensity armCombination Product: ECP accelerated-intensity armBiological: T1DM standard of care
- Registration Number
- NCT05413005
- Lead Sponsor
- Abu Dhabi Stem Cells Center
- Brief Summary
OPERA Study is a randomized, open-label, prospective, pilot, and a monocentric clinical trial involving outpatients within Abu Dhabi Stem Cells Center (ADSCC) with a confirmed diagnosis of type 1 diabetes mellitus (T1DM). The patients will be randomly allocated (1:1) in a parallel assignment involving two groups of participants: Group A (Regular-intensity arm): Extracorporeal Photopheresis (ECP) on a regular-intensity regimen described in the Protocol as add-on T1DM standard of care, or Group B (Accelerated-intensity arm): ECP on an accelerated regimen plus T1DM standard of care.
- Detailed Description
The standard of care is defined as per the "DOH Standards for diagnosis, management and data reporting for diabetes" in combination with the DOH recommended NICE (UK National Institute for Health and Care Excellence) guidelines "Type 1 diabetes in adults: diagnosis and management (NG17)". OPERA Study will be fully conducted in ADSCC, including the patient assessment and inclusion, randomization, ECP procedures, and follow-up consultations, according to this Protocol and GCP principles. All patients will receive T1DM standard of care, plus additional ECP protocols on two regimens of investigational interventions. The primary objective is the safety assessment of ECP, to be assessed by the tolerability to the ECP procedures, incidence of treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), and serious adverse events (SAEs) assessed by the CTCAE v5.0, and the WHO-UMC causality assessment system. The other primary objective is the preliminary efficacy assessment of the ECP as an add-on treatment to the standard of care for T1DM patients, assessed by the exogenous insulin use, HbA1c levels, C-peptide levels, and clinically important hypoglycemic episodes. The secondary objective is the assessment of the immune response profile in T1DM patients receiving standard of care and additional ECP procedures. The trial is approved by the institutional ADSCC Research Ethics Committee (REC) and written informed consent will be obtained from all patients. OPERA Study will be conducted following the principles of the Declaration of Helsinki and ICH-GCP guidelines.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 10
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Group A (ECP regular-intensity arm) ECP regular-intensity arm ECP in a regular-intensity arm plus T1DM standard of care Group A (ECP regular-intensity arm) T1DM standard of care ECP in a regular-intensity arm plus T1DM standard of care Group B (ECP accelerated-intensity arm) T1DM standard of care ECP in an accelerated-intensity arm plus T1DM standard of care Group B (ECP accelerated-intensity arm) ECP accelerated-intensity arm ECP in an accelerated-intensity arm plus T1DM standard of care
- Primary Outcome Measures
Name Time Method Tolerability to ECP procedures Weeks 0 - 24 Rate of tolerability of the ECP will be assessed by the number of ECP discontinuation due to adverse events (AE), or withdrawal of participants of the Study due to serious adverse events (SAEs)
Exogenous insulin use Baseline, months 3, 6, and 12 Rate of modification in exogenous insulin requirements compared with baseline. Marker for efficacy of treatment: reducing insulin dose
Clinically important hypoglycemic episodes Baseline - Month 12 Frequency of clinically important hypoglycemic episodes (described in Protocol).
Marker for efficacy of treatment: requiring decrease of insulin dose)C-peptide levels Baseline, months 3, 6, and 12 Rate of modification in C-Peptide levels compared with baseline. Marker for efficacy of treatment: increasing C-Peptide levels of ≥ 0.7 ng/mL
Incidence of adverse events (AEs) Weeks 0 - 24 Incidence of treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), and serious adverse events (SAEs): Proportion of participants with AEs as assessed by CTCAE v5.0
HbA1c levels Baseline, months 3, 6, and 12 Rate of modification in HbA1c levels compared with baseline. Marker for efficacy of treatment: decreasing for a target HbA1c level of 48 mmol/mol \[6.5%\]
- Secondary Outcome Measures
Name Time Method Immune response profile (cellular) Baseline, months 3, 6, and 12 CD3, CD4, CD8, CD11c, CD14, CD16, CD19, CD20, CD25, CD27, CD28, CD38, CD45, CD45RA, CD45RO, CD56, CD57, CD66b, CD123, CD127, CD161, CD294, CCR4, CCR6, CCR7, CXCR3, CXCR5, will be assessed for identification of immune cells and subsets analyses
Serum IgG levels Baseline, months 3, 6, and 12 Serum IgG concentration will be assessed for characterization of the humoral response profile
Serum IgM levels Baseline, months 3, 6, and 12 Serum IgM concentration will be assessed for characterization of the humoral response profile
Serum IgA levels Baseline, months 3, 6, and 12 Serum IgA concentration will be assessed for characterization of the humoral response profile
Trial Locations
- Locations (1)
Abu Dhabi Stem Cells Center
🇦🇪Abu Dhabi, United Arab Emirates