Prophylactic Treatment of Graves Ophthalmopathy (GO) in Newly Diagnosed Graves' Disease (GD) with Atorvastatin (ATORGO)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Region Skane

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hormonal diseases [C19], Diseases [C] - Eye Diseases [C11], Diseases [C] - Immune System Diseases [C20]

Decision date (initial)

2026-02-04

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy

The primary objective of this trial is to determine if atorvastatin can prevent the development of active GO in patients newly diagnosed with GD.

Secondary objectives 6

1. To evaluate the progression of GO during the study period
2. To assess the severity of GO during the study period
3. To determine the need for additional treatment for GO (e.g., corticosteroids or biologics).
4. To monitor changes in quality of life
5. To identify changes in biomarkers related to GO
6. To explore genetic factors associated with treatment response

Conditions and MedDRA coding

Graves Ofthalmopathy

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Age 18–85 years
2. Diagnosis of Graves' Disease (GD):TSH < 0.1 μU/L. Elevated free T4 (fT4) and/or free T3 (fT3). Positive TSH receptor antibodies (TRAb).
3. Sufficient knowledge of Swedish to understand medical information and participate in telephone or video calls.

Exclusion criteria 10

1. Pregnancy, planned pregnancy within 12 months or lactation
2. Need of corticosteroid protection for planned radioiodine therapy
3. Mental inability, reluctance or language difficulties that result in difficulty understanding the meaning of participation in the trial
4. Prior treatment with statins, selenium, corticosteroids, or other immunosuppressive medications for more than 7 days within the 3 months preceding GD diagnosis.
5. Existing Graves' Ophthalmopathy (GO) at the time of GD diagnosis.
6. Known hypersensitivity to atorvastatin or other statins.
7. Active liver disease.
8. Alcohol abuse.
9. Elevated liver enzymes (ASAT/ALAT > 3 times the upper normal limit in two consecutive tests).
10. Concurrent use of medications that interact with statins, particularly CYP3A4 inhibitors or inducers.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The proportion of patients developing active GO after 12 months, defined as a Clinical Activity Score (CAS) ≥3. CAS is a standardized method for assessing the activity of GO.

Secondary endpoints 8

1. Proportion of patients developing clinical GO at 3, 6, 9, 15, and 18 months.
2. Time from GD diagnosis to GO onset.
3. Highest CAS recorded during the study period.
4. Proportion of patients requiring interventions such as corticosteroids, orbital irradiation, or biologics (e.g., rituximab or teprotumumab).
5. Change in quality-of-life scores as measured by validated tools, including GO-QoL and Thyr-Pro questionnaires, at baseline, 12 months, and 18 months.
6. Levels of TSH-receptor antibodies (TRAb) at 12 and 18 months
7. Identification of new GO-related biomarkers using proteomics and microarray analysis.
8. Genetic differences between responders and non-responders to atorvastatin, identified via genome-wide association studies (GWAS) and analyses of relevant single nucleotide polymorphisms (SNPs).

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Region Skane

Sponsor organisation

Region Skane

Address

Dockplatsen 26, Malmo S:t Petri Malmo S:t Petri

City

Malmo

Postcode

211 74

Country

Sweden

Scientific contact point

Organisation

Region Skane

Contact name

Tereza Planck

Public contact point

Organisation

Region Skane

Contact name

Tereza Planck

Locations

1 EU/EEA country · 9 investigational sites

By country

Investigational sites

Results and documents

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

Documents 12 files

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

Application history

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