Grafalon®therapy within 12 weeks of Type 1 diabetes (T1D) diagnosis in children and young adults to prevent the loss of residual beta cell function: a randomised, double-blind, placebo-controlled dose finding study Acronym: GIRO T1D

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Institute For Clinical And Experimental Medicine, University Hospital Kralovske Vinohrady

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

4 Jun 2024 → ongoing

Decision date (initial)

2024-01-11

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

Institute of Clinical and Experimental Medicine (IKEM), Prague, CZ · University Hospital of Kralovske Vinohrady (FNKV)

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To assess the efficacy and safety of two different ATLG dosages in the treatment of patients with recent onset Type 1 Diabetes.

Secondary objectives 1

1. To Assess the effects of ATLG on the need of insulin therapy and other markers of diabetes. To Assess the effects of ATLG treatment on the immune system and other biological markers including T-cell populations and markers, T-cell repertoire.

Conditions and MedDRA coding

Type 1 diabetes

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. from the beginning of Screening to Visit 14.

Exclusion criteria 1

1. 1. History of recurrent (e.g., several times a year) of severe (e.g., pneumonia) or chronic infections or conditions predisposing to chronic infections 2. History of severe systemic fungal infection within the past 12 months prior to screening unless treated and resolved with appropriate documented therapy 3. Vaccination within 4 weeks before randomization 4. Positive Viral PCR for EBV, CMV, COVID 5. Patients with HIV or HTLV I/II infection or HepB/C 6. Receipt of any other concomitant medications or herbal products that can influence the immune system within 90 days prior to screening 7. History of pancreatitis (acute or chronic) 8. Any past or current diagnosis of malignant neoplasms 9. Known impairment of the immune system, except for T1D, coeliac disease, alopecia, autoimmune antibodies not considered clinical important (e.g. thyroid antibodies without any clinically important thyroid disease or well-balanced Hashimoto’s disease), and vitiligo 10. Patients with a psychiatric condition (e.g., severe anxiety, psychosis) that would interfere with the study as determined by the primary investigator. Stable psychiatric conditions such as chronic anxiety or depression will be allowed. 11. Patients with known allergy to one or more of the study drug components or rabbit proteins. 12. Female patients who are pregnant, lactating, or who want to get pregnant during the study period. 13. Patients with a history of alcohol or any psychoactive substance abuse or dependence (including alcohol but excluding nicotine and caffeine). 14. Patients with any other significant chronic disease, according to the investigator discretion, exclude rare forms of diabetes (MODY) according to history and clinical judgement. 15. Obesity (BMI more than 35 kg/m2) 16. Other forms of diabetes 17. Patients unable or unwilling to comply with the protocol

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. AUC0-2h for a mixed meal tolerance test (MMTT) stimulated C-peptide concentration-time curve (C-peptideAUC) at 12 months relative to baseline

Secondary endpoints 1

1. Peak MMTT stimulated C-peptide concentration (C-peptidemax) at months 6, 12 and 24 relative to baseline Change in fasting C-peptide from baseline to months 6 and 12 Change in HbA1c from baseline to month 12 HbA1c at 12 and 24 months Change in fasting plasma glucose from baseline to month 12 Total insulin treatment administered over the period of 12 months calculated as AUC (insulinAUC). Total daily insulin dose per kg at 1, 3, 6 and 12 months Percentage of patients that maintain stimulated pea

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Institute For Clinical And Experimental Medicine

Sponsor organisation

Institute For Clinical And Experimental Medicine

Address

Videnska 1958/9 Krc

City

Prague

Postcode

140 00

Country

Czechia

Scientific contact point

Organisation

Institute For Clinical And Experimental Medicine

Contact name

Frantise Saudek

Public contact point

Organisation

Institute For Clinical And Experimental Medicine

Contact name

Frantise Saudek

University Hospital Kralovske Vinohrady

Sponsor organisation

University Hospital Kralovske Vinohrady

Address

Srobarova 1150/50

City

Praha

Postcode

10034 Praha 10

Country

Czechia

Scientific contact point

Organisation

University Hospital Kralovske Vinohrady

Contact name

Jan Vosahlo

Public contact point

Organisation

University Hospital Kralovske Vinohrady

Contact name

Jan Vosahlo

Sponsor responsibilities

Article 77 compliance

Institute For Clinical And Experimental Medicine

Contact point sponsor

Institute For Clinical And Experimental Medicine

Article 77 implementation

Institute For Clinical And Experimental Medicine

Locations

1 EU/EEA country · 2 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 25 files

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

Application history

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