GenSci098 for adults with Graves' disease

A Phase I Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single Ascending Subcutaneous Dose of GenSci098 in Patients With Graves' Disease

This phase 1 trial will test single increasing subcutaneous doses of GenSci098 to see if they are safe and well tolerated in adults with Graves' disease.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 24 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Changchun GeneScience Pharmaceutical Co., Ltd. (Industry-sponsored)
• Drugs / interventions: radiation
• Locations: 1 site (Shenyang, Liaoning)
• Trial ID: NCT07286656 on ClinicalTrials.gov

What this trial studies

This is a phase 1, single-ascending-dose study enrolling adults with confirmed diffuse toxic goiter (Graves' disease) to receive subcutaneous GenSci098. Participants will be screened for abnormal thyroid function and must have discontinued recent antithyroid or thyroid hormone treatments per protocol. Small cohorts will receive escalating single doses with close safety monitoring, laboratory testing, and follow-up visits to evaluate tolerability and adverse events. Key exclusions include prior radioactive iodine or thyroid surgery and active thyroid eye disease.

Who should consider this trial

Good fit: Adults (age ≥18) with confirmed diffuse toxic goiter, abnormal thyroid tests, no recent antithyroid or thyroid hormone use, and who meet contraception and follow-up requirements are ideal candidates.

Not a fit: Patients with non-diffuse causes of hyperthyroidism, prior radioactive iodine or thyroid surgery, active thyroid eye disease, recent thyroid hormone/antithyroid use, or who are pregnant are unlikely to benefit from this phase 1 trial.

Why it matters

Potential benefit: If successful, GenSci098 could become a new treatment option that safely reduces thyroid overactivity in patients with Graves' disease.

How similar studies have performed: Single-ascending-dose safety trials are a standard first step, but there is limited clinical evidence so far for subcutaneous biologics directly treating Graves' hyperthyroidism.

Eligibility criteria

Inclusion Criteria:

* Age ≥ 18 years.
* Voluntary signed informed consent.
* Confirmed diagnosis of diffuse toxic goiter (Graves' disease).
* Abnormal thyroid function tests (e.g., elevated T4, and suppressed TSH).
* No prior or recent use of antithyroid medications (discontinued for at least 4 weeks).
* Female participants must be postmenopausal, surgically sterile, or using a highly effective method of contraception.
* Male participants must agree to practice abstinence, use a highly effective method of contraception， or have undergone vasectomy.
* Ability to comply with the follow-up schedule and understand and adhere to the study requirements.

Exclusion Criteria:

* Non-diffuse toxic goiter-induced hyperthyroidism.
* Previous radioactive iodine treatment or thyroid surgery.
* History or risk of thyroid storm.
* Use of thyroid hormone medications within the past 6 weeks.
* accompanied by active thyroid eye disease.
* Thyroid eye disease treated with radiation/surgery,or need for urgent surgery surgical or medical intervention.
* Optic nerve lesions or corneal damage.
* Use of steroids or immunosuppressants within the past 3 months,or those who have used biologics within 6 months
* Inability to quit smoking during the study.
* Allergy to the study drug or monoclonal antibodies.
* Participation in another clinical trial within the past 3 months.
* Abnormal electrocardiogram.
* Significant hepatic or renal dysfunction.
* Pregnancy,breastfeeding,or positive pregnancy test.
* Positive for HIV,syphilis,hepatitis B,or hepatitis C.
* History of drug or substance abuse.
* Other autoimmune diseases requiring treatment.
* History of malignant tumors.
* Splenectomy or major surgery within the past 6 months.
* Severe cardiovascular,pulmonary,hepatic,renal,neurological,or hematological diseases.
* Other conditions deemed unsuitable by investigators.

Where this trial is running

Shenyang, Liaoning

• The First Affiliated Hospital of China Medical University — Shenyang, Liaoning, China (Recruiting)

Study contacts

• Principal investigator: Zhongyan Shan, PHD — First Hospital of China Medical University
• Study coordinator: Zhongyan Shan, PHD

Conditions Safety; Tolerability; GenSci098; Graves Disease