Recent-Onset Type 1 Diabetes Extension Study Evaluating the Long-Term Safety of Teplizumab

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Provention Bio Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

Trial duration

23 Jul 2021 → ongoing

Decision date (initial)

2024-10-10

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Provention Bio

External identifiers

EU CT number

2024-516543-26-00

EudraCT number

2020-002798-92

WHO UTN

U1111-1310-5137

ClinicalTrials.gov

NCT04598893

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To evaluate the long-term safety of teplizumab in participants who previously completed the PRV-031-001 (PROTECT) study for an additional 42 months of follow-up

Secondary objectives 1

1. To evaluate clinical parameters of diabetes management, including insulin use and HbA1c, during the 42 months of follow-up

Conditions and MedDRA coding

Type 1 Diabetes Mellitus

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-000524-PIP01-08

Plan to share IPD

Yes

IPD plan description

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

1. Completion of the PRV-031-001 (PROTECT) study by completing the Week 78 visit in that study, regardless of how many doses of study drug were administered
2. Provide written informed consent within 12 months of the Week 78 in the PRV- 031-001 study. Written assent will be obtained for participants under 18 years of age at the time of enrollment, according to applicable regulations. If possible, written informed consent and/or assent for the current PROTECT Extension study (PRV-031-003) should be obtained at the final (Week 78) visit in the PROTECT study

Exclusion criteria 1

1. Premature discontinuation from the PRV-031-003 (PROTECT) study for any reason

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Incidence of adverse events (AEs), serious adverse events (SAEs) and adverse events of special interest (AESIs), including infections and malignancies

Secondary endpoints 3

1. The area under the time-versus-concentration curve (AUC) of C-peptide after a 4-hour (4h) mixed-meal tolerance test (MMTT), a measure of endogenous insulin production and β cell function by visit and change from baseline (MMTT), a measure of endogenous insulin production and β cell function
2. Insulin use, defined as a daily average dose in units per kilogram per day (U/kg/day) by visit and change from baseline
3. HbA1c levels(%) by visit and change from baseline

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Provention Bio Inc.

Sponsor organisation

Provention Bio Inc.

Address

100 Morris Street

City

Morristown

Postcode

07960-4563

Country

United States

Scientific contact point

Organisation

Provention Bio Inc.

Contact name

Clinical Sciences and Operations

Public contact point

Organisation

Provention Bio Inc.

Contact name

Clinical Sciences and Operations

Third parties 7

Locations

5 EU/EEA countries · 16 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 45 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

6 submissions — initial application plus substantial / non-substantial modifications