Efficacy of Extracorporeal Photopheresis (ECP) in the Treatment of Type 1 Diabetes Mellitus

Early Phase 1

Recruiting

• Conditions: Type 1 Diabetes
• Interventions: Combination Product: ECP regular-intensity arm; Combination Product: ECP accelerated-intensity arm; Biological: 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.

Study Timeline

• Study First Submitted: 2022-06-07
• Study First Submitted QC: 2022-06-07
• Study First Posted: 2022-06-09
• Study Start: 2022-09-05
• Status Verified: 2025-07
• Last Update Submitted: 2025-07-01
• Last Update Posted: 2025-07-03
• Primary Completion: 2026-09-01
• Study Completion: 2026-12-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

Not provided

Exclusion Criteria

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)
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 \[6.5%\]
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

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