Tirzepatide, a new drug for type 2 diabetes mellitus and obesity, as a possible treatment to modify the clinical evolution of Wolfram syndrome type 1

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Ospedale San Raffaele S.r.l.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hormonal diseases [C19]

Trial duration

16 May 2024 → 12 Sep 2025

Decision date (initial)

2024-08-13

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2024-514909-64-00

EudraCT number

2022-003853-70

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy

To determine the efficacy of tirzepatide in increasing endogenous insulin production in patients with Wolfram syndrome type 1 (WS1)

Conditions and MedDRA coding

Wolfram syndrome type 1

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. A definite diagnosis of Wolfram syndrome, as determined by the following: (a) Documented diabetes mellitus diagnosed under 16 years of age according to WHO or ADA criteria; and (b) Documented functionally relevant recessive mutations on both alleles of the WFS1 gene or dominant mutation on one allele of the WFS1 gene based on historical test results (if available) or from a qualified screening laboratory;
2. aged 5 years or older;
3. The patient, the patient's parents or legally authorized guardian(s) must have voluntarily signed an informed consent form approved by the Institutional Review Board/Independent Ethics Committee after all relevant aspects of the study have been explained and discussed with the patient . Guardian consent and patient consent, if applicable, must be obtained;
4. Women of childbearing age will be included only after a highly sensitive negative urine pregnancy test. If sexually active, they must agree to use a highly effective contraceptive measure;
5. Patient willing to wear a continuous glucose monitor.

Exclusion criteria 11

1. Clinically significant CNS involvement unrelated to Wolfram that is judged by the investigator likely to interfere with the accurate administration and interpretation of protocol assessments;
2. A history of pancreatitis;
3. Pre-existing thyroid disease;
4. A personal or family history of medullary thyroid carcinoma;
5. Multiple endocrine neoplasia type 2 syndrome;
6. Active liver or kidney disease, personal or family history of liver/renal dysfunction related to known genetic disease;
7. Treatment with any investigational drug within 30 days prior to study entry;
8. Ongoing therapy with a GLP-1 agonist or DDP-4 inhibitor or known hypersensitivity to the GLP-1 agonist;
9. Any other medical, psychiatric, social situation or acute or chronic laboratory outcome that, in the judgment of the investigator, would jeopardize the safety of the patient while participating in the study, cause inability to comply with the protocol, or affect the outcome of the study;
10. Breastfeeding;
11. Pre-existing eye disease (corneal or lens disease and any other retinal or optic nerve disease not related to Wolfram).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The percentage of participants with a positive response to MMTT (C-peptide at 90 min >0.6 ng/ml)

Investigational products

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

Test 3

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Ospedale San Raffaele S.r.l.

Sponsor organisation

Ospedale San Raffaele S.r.l.

Address

Via Olgettina 60

City

Milan

Postcode

20132

Country

Italy

Scientific contact point

Organisation

Ospedale San Raffaele S.r.l.

Contact name

Lorenzo Piemonti

Public contact point

Organisation

Ospedale San Raffaele S.r.l.

Contact name

Lorenzo Piemonti

Locations

1 EU/EEA country · 1 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 11 files

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

Application history

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