Clinical trial NCT05015127: A Study to Evaluate the Efficacy and Safety of HBM9161 on Moderate to Severe Thyroid Eye Disease
Official title: A Randomized, Double-blind, Placebo-controlled, Phase 2/3 Operational Seamless Design Clinical Study to Evaluate the Efficacy and Safety of HBM9161 Subcutaneous Injection in Chinese Patients With Active, Moderate to Severe Thyroid Eye Disease (TED)
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
PHASE3
Conditions: Thyroid Ophthalmopathy
Keywords: TED
Lead sponsor: Harbour BioMed (Guangzhou) Co. Ltd.
Sponsor class: INDUSTRY
First posted: 2021-08-20
Enrollment: 36
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Primary Objective: To preliminarily evaluate the efficacy of the treatment regimen of HBM9161 680 mg administered subcutaneously weekly and sequentially every other week for 12 weeks in Chinese patients with active moderate to severe TED.

Detailed description: This is a multicenter, randomized, double-blind and placebo-controlled study. The investigational drug is HBM9161 injection, with the indication of TED. Subjects will be randomized to treatment group I, treatment group II, or placebo group. Subjects in treatment group 1 will receive HBM9161 680 mg subcutaneously once weekly (qw) for 12 doses; Subjects in treatment group 2 will receive HBM9161 680 mg subcutaneously once weekly (qw) for 6 doses, followed by HBM9161 680 mg subcutaneously once every two weeks (q2w) for 3 doses; Subjects in placebo group will receive placebo subcutaneously once weekly (qw) for 12 doses. After the 12-week dosing period, there will be a 5-week follow-up period.

Eligibility criteria: Inclusion Criteria:

1. Signed written informed consent form.
2. Male or female between the ages of 18 and 70 (including boundary values).
3. Active TED associated with a clinical diagnosis of Graves' disease or Hashimoto's thyroiditis with CAS ≥ 3 at the screening visit and baseline (both on a 7-item scale) in the eye with the most severe proptosis.
4. The active period of TED starts within 9 months prior to screening visit
5. Positive anti-TSHR antibody at screening visit.
6. Subject is willing and able to receive treatment and complete corresponding assessments as required by the protocol.

Exclusion Criteria:

1. Other uncontrolled concurrent diseases
2. Serious TED requiring surgery or radiotherapy
3. Suffer from autoimmune diseases or ophthalmic conditions other than TED that, in the opinion of the investigator, would impact the assessment of the study drug
4. No significant laboratory abnormalities

Primary outcomes:
measure: Proptosis responder rates of the treatment groups and placebo group at Week 12
timeFrame: At week 12
Secondary outcomes:
measure: The percentage of subjects with a CAS value of 0 or 1 in the study eye
timeFrame: At week 6, week 12, week 18 and week 24
measure: The change from Baseline in proptosis measurement in the study eye
timeFrame: At week 6, week 12, week 18 and week 24
measure: Composite responder rate
timeFrame: At week 6, week 12, week 18 and week 24
measure: Diplopia response rate
timeFrame: At week 6, week 12, week 18 and week 24
measure: The change from baseline in the Graves' Ophthalmopathy Quality of Life (GO-QoL) Questionnaire visual function score and psychosocial function score
timeFrame: At week 6, week 12, week 18 and week 24
measure: The changes in proptosis responder rate over time
timeFrame: From week 1 to week 24
measure: The change from baseline in quantitative measurement of orbital structures by MRI, including exophthalmos values, fatty tissue thickness of inner side in eye balls, thickness of extraocular muscles. All the above parameters will be measured in millimeter
timeFrame: At week 12, week 24
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Shanghai Ninth People's Hospital, Shanghai Jiaotong University School of Medicine
state: Shanghai
Study officials:
affiliation: Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University
name: Huifang Zhou
role: PRINCIPAL_INVESTIGATOR
References: