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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
Lead sponsor
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
Timeline
Milestones
Assets
Investigational agents
Study populations
Who this study enrolls
Eligibility
Who can enroll
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 endpointschanging in endogenous insulin production
Time frame:[Time Frame: month 6±1, month 12±1]
C-peptide AUC
change from baseline, improvement
changing in insulin production
Time frame:[Time Frame: month 6±1, month 12±1]
C-peptide AUC
change from baseline, improvement
glucose variability
Time frame:[Time Frame: month 6±1, month 12±1]
change from baseline, improvement
glycemic variability
Time frame:[Time Frame: month 6±1, month 12±1]
change from baseline, improvement
change in time in range
Time frame:[Time Frame: month 6±1, month 12±1]
CGM time-in-range
change from baseline, improvement
change in insulin requirements
Time frame:[Time Frame: month 3±1, month 6±1, month 12±1]
change from baseline, improvement
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 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.