← Trials/Trial dossier/NCT05659368

UnknownPhase 2

Tirzepatide Monotherapy in Patients With Wolfram Syndrome Type 1

Towards a Personalized Precision Medicine in Rare Disease: Tirzepatide (a Dual Glucose-dependent Insulinotropic Polypeptide and Glucagon-like Peptide-1 Receptor Agonist) Monotherapy in Patients With Wolfram Syndrome Type 1

Asset

Tirzepatide

Subcutaneous · GLP-1 / GIP dual

Listed sites

1

Recruiting sites

1

Enrollment

10

estimated

Study population

Diabetes (other / unspecified), Wolfram Syndrome

Key I/E criterion

Primary endpoint

C-peptide AUC

Footprint

Where this trial recruits

Site locations as reported to ClinicalTrials.gov. Site count is not enrollment count; per-site enrollment is not available from source.

Identifiers

Registered as

NCT IDNCT05659368
Org study IDWolfram Tirzepatide
Secondary ID2022-003853-70
Secondary IDPNRR-MR1-2022-12375914National Recovery and Resilience Plan

Timeline

Milestones

Study first posted2022-12-21actual
Study start2024-01-01actual
Last update posted2024-02-22actual
Primary completion2024-10estimated (month precision)
Study completion2024-12estimated (month precision)

Assets

Investigational agents

Study populations

Who this study enrolls

Diabetes (other / unspecified)Wolfram Syndrome

Eligibility

Who can enroll

Minimum age5 Years
SexAll
Healthy volunteersNot accepted

Inclusion criteria

1. A definitive diagnosis of Wolfram syndrome, as determined by the following:

1. Documented diabetes mellitus diagnosed under 16 completed years according to WHO or ADA criteria AND

2. 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 laboratory at screening;

2. Aged 5 years or older;

3. The patient, patient's parent(s), or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board/Independent Ethics Committee-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient. The guardians' consent and patient's assent, as relevant, must be obtained;

4. Females of child bearing potential will only be included after a negative highly sensitive urine pregnancy test. If sexually active, they must agree to use a highly effective contraception measure;

5. Patient willing to wear a continuous glucose monitor.

Exclusion criteria

1. Clinically significant non-Wolfram related CNS involvement which is judged by the Investigator to be 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 syndrome type 2;

6. Active liver or renal disease, personal or family history of liver/kidney dysfunction related to known genetic disorders;

7. Treatment with any investigational drug within the 30 days prior to Trial entry;

8. Current therapy with of GLP-1 agonist or DDP-4 inhibitor or a known hypersensitivity to GLP-1 agonist;

9. Any other acute or chronic medical, psychiatric, social situation or laboratory result that, based on investigator's judgment, would jeopardize patient safety during trial participation, cause inability to comply with the protocol, or affect the Trial outcome;

10. Breastfeeding;

11. Pre-existing ocular disease (corneal or lens diseases and any other retinal or optic nerve non-Wolfram related diseases).

Endpoints (7)

What's being measured

Protocol endpoints and posted registry outcome measures, grouped into outcome categories. Composite endpoints show their component event types. Standard codes (LOINC, SNOMED CT) are shown where available.

Glycemic / diabetes

7 endpoints
Primary/protocol endpoint

changing in endogenous insulin production

Time frame:[Time Frame: month 6±1, month 12±1]

C-peptide AUC

change from baseline, improvement

Primary/protocol endpoint

changing in insulin production

Time frame:[Time Frame: month 6±1, month 12±1]

C-peptide AUC

change from baseline, improvement

Secondary/protocol endpoint

glucose variability

Time frame:[Time Frame: month 6±1, month 12±1]

change from baseline, improvement

Secondary/protocol endpoint

glycemic variability

Time frame:[Time Frame: month 6±1, month 12±1]

change from baseline, improvement

Secondary/protocol endpoint

change in time in range

Time frame:[Time Frame: month 6±1, month 12±1]

CGM time-in-range

change from baseline, improvement

Secondary/protocol endpoint

change in insulin requirements

Time frame:[Time Frame: month 3±1, month 6±1, month 12±1]

change from baseline, improvement

Secondary/protocol endpoint

change in HbA1c

Time frame:[Time Frame: month 3±1, month 6±1, month 12±1]

HbA1c, change

change from baseline, improvement

LOINC 4548-4

Provenance

Sources

Trial identity, design, statusClinicalTrials.gov API v2
Snapshot dateJuly 1, 2026
Endpoint classificationDelfa endpoint taxonomy v2 (May 13, 2026)
Results tableno registry results posted yet

Trial facts come from public ClinicalTrials.gov records. Endpoint categories are Delfa's classification of those records, not a ClinicalTrials.gov field. All figures reflect the July 1, 2026 snapshot.