Thyroid Eye DiseasePhase 1NCT05776121
Study of ZB001 in Chinese Patients With Thyroid Eye Disease
A Phase 1 study testing ZB001 for injection in people with Thyroid Eye Disease, run by Zenas BioPharma (USA), LLC at 9 sites in China.
Trial summary
The investigational drug, ZB001 is a humanized IgG1κ monoclonal antibody targeting human IGF-1R. The study is designed to evaluate the efficacy, safety, tolerability, and pharmacokinetics（PK）/pharmacodynamics (PD) profile of ZB001 in Chinese patients with Thyroid Eye Disease. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thyroid Eye Disease
- What is being tested?
- ZB001 for injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 9 sitesChina
- Study dates
- 10 May 2023to 17 Apr 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female adults, 18 years of age or older
- Clinical diagnosis of Graves' ophthalmopathy and CAS evaluation of eyes under study ≥ 4 points (7 points in total)
- Moderate-to-severe active thyroid-associated ophthalmopathy (i.e., severely affects daily life): exophthalmos extent is ≥18.6 mm, or progressive exophthalmos (≥3 mm greater than the previous exophthalmos record per the investigator); accompanied by at least one of the following symptoms: eyelid retraction ≥ 2 mm; moderate or severe soft tissue involvement (conjunctival congestion, edema, periorbital congestion or edema); non-persistent or persistent diplopia
- Before Screening, evidence of eye symptoms or signs related to thyroid-associated ophthalmopathy in medical records for ≤1 year
- Thyroid function normal, or only mild hyperthyroidism or hypothyroidism, defined as free thyroxine (FT4) ）and free triiodothyronine (FT3) levels within 0.5-1.5 times the normal range at Screening. Efforts have been made to correct any mild hypothyroidism or hyperthyroidism in a timely manner and try to maintain normal thyroid function throughout the study. Thyroidectomy is not an exclusion.
- If the patient is a female of childbearing potential (including a female with menopause < 1 year, amenorrhea < 1 year, or without surgical sterilization), the pregnancy test result will be negative at Screening. Such patients must agree to use the effective birth control methods described in the relevant protocol section (Section 9.2 Contraception and Pregnancy) at least one complete menstrual cycle before the first dose of the study drug, and continue to use contraception methods for 100 days after the last dose
- Male patients must be surgically sterilized for at least 6 weeks or agree to use the effective birth control methods described in the relevant protocol section (Section 9.2 Contraception and Pregnancy) before the first dose of the study drug and within 100 days after the last dose
Who cannot join
- In the past 6 months, due to optic neuropathy, new visual field defect or color defect secondary to optic nerve involvement, the best corrected visual acuity of the study eye decreased, defined as the result of standardized visual acuity chart decreased ≥ 0.2
- Corneal involvement of the study eye, and no improvement after medical interventions
- CAS decreased ≥ 2 points from Screening Assessment to Day -1
- The exophthalmos extent of the study eye ≥ 2 mm from Screening Assessment to Day -1
- The study eye previously received orbital radiotherapy or surgery due to thyroid-associated ophthalmopathy
- Known history of clinically significant ear disease, ear surgery or hearing loss
Show all 12Show fewer
- Inflammatory bowel disease (e.g., biopsy-proven or clinical evidence of inflammatory bowel disease)
- Cumulative use of glucocorticoid equivalent to ≥ 1g methylprednisolone as thyroid-associated ophthalmopathy treatment (A lower cumulative dose [<1g] of glucocorticoid used before Screening, or hormone eye drops withdrawn for ≥ 6 weeks before Screening, is allowed for inclusion)
- Received any doses of oral corticosteroids to treat diseases other than thyroid-associated ophthalmopathy within 4 weeks before Screening (local application is allowed for inclusion)
- Pregnant or lactating females
- Smokers (≥ 5 cigarettes/day) or former smokers (≥ 5 cigarettes/day) quit within 6 months before enrollment in the study
- Any vaccination planned during the study
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
China9 sites
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
Beijing Tongren Hospital, CMU
Beijing
Peking University Third Hospital
Beijing
Third Xiangya Hospital of Central South University
Changsha
Xiangya Hospital Central South University
Changsha
Chongqing Aier General Hospital
Chongqing
The Second Hosptial of Dalian Medical University
Dalian
The Second Hosptial of Anhui Medical University
Hefei
Affiliated Eye Hospital of Nanchang University
Nanchang
About this study
Official title: A Multiple Ascending Doses Study of ZB001 in Chinese Patients With Thyroid-Associated Ophthalmopathy
The investigational drug, ZB001 is a humanized IgG1κ monoclonal antibody targeting human IGF-1R. The study is designed to evaluate the efficacy, safety, tolerability, and pharmacokinetics（PK）/pharmacodynamics (PD) profile of ZB001 in Chinese patients with Thyroid Eye Disease.
- Conditions
- Thyroid Eye Disease
- Being tested
- ZB001 for injection
- Medical subject headings
- Graves Ophthalmopathy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ZB001 for injectionDrug
Dose Cohort1 (3 mg/kg) ZB001 four IV injections
ZB001 for injectionDrug
Dose Cohort2 (10 mg/kg) ZB001 four IV injections
| Study group | Role | What they receive |
|---|---|---|
| ZB001 for injection | Experimental | Treat different dose cohorts with four intravenous injections of ZB001 Receives: Drug: ZB001 for injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with Adverse Events, Serious Adverse Events, and Laboratory Evaluations as assessed by CTCAE v5.0
Measured: through study completion, up to 169 days
The exophthalmos response rates of study eye at Week 6 and Week 12 (defined as the proportion of patients whose exophthalmos measured by Hertel exophthalmos meter decreased ≥ 2mm from baseline)
Measured: At week6 and week 12
Secondary outcomes (24)
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Exophthalmos measured by MRI/CT scan
Measured: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Orbital fat volume measured by MRI/CT scan
Measured: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Extraocular eye muscle volume measured by MRI/CT scan
Measured: At week6, week 12 and week24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Facial fat volume measured by MRI/CT scan
Measured: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Digital and manual measurement of palpebral fissure height
Measured: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Exotropia deviation measurement
Measured: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Subjective diplopia score. The range of score is from 0-3. The lower score means the better outcome.
Measured: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Best corrected visual acuity
Measured: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Intraocular pressure
Measured: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Clinical Activity Score (CAS) . The range of score is from 0-7. The lower score means the better outcome
Measured: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Graves ophthalmopathy - Quality of Life (QoL). The range of score is from 0-100. The higher change of quality of life means the better outcome.
Measured: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Graves ophthalmopathy - Visual function QOL. The range of score is from 0-100. The higher change of quality of life means the better outcome.
Measured: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Graves ophthalmopathy - Social Function QoL. The range of score is from 0-100.The higher change of quality of life means the better outcome.
Measured: At week6, week 12 and week 24
Serum ZB001 antidrug antibody (ADA) titers
antidrug antibody (ADA) titers of ZB001
Measured: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
Serum IGF-1 concentrations in the blood over time
Measured: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
Maximum observed concentration (Cmax)
Pharmacokinetics
Measured: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
Time to measured peak concentration (Tmax)
Pharmacokinetics
Measured: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
Area under the concentration-time curve from time 0 to the last timepoint with measurable concentration (AUClast)
Pharmacokinetics
Measured: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
Area under the concentration-time curve extrapolated to infinity (AUCinf)
Pharmacokinetics
Measured: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
Area under the concentration-time curve over a dosing interval (AUCtau)
Pharmacokinetics
Measured: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
half life (t1/2)
Pharmacokinetics
Measured: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
Systemic clearance (CL)
Pharmacokinetics
Measured: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
Steady State Volume of Distribution (Vss)
Pharmacokinetics
Measured: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
The exophthalmos response rates of study eye at Week 24 (defined as the proportion of patients whose exophthalmos measured by Hertel exophthalmos meter decreased ≥ 2mm from baseline)
Measured: At week6 and week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Mar 2023
- Study start10 May 2023
- Primary completion17 Apr 2024
- Study completion17 Apr 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thyroid Eye DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Zenas BioPharma (USA), LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05776121
- Sponsor's protocol ID
- ZB001-01-002
- Status last verified
- Apr 2025
- First submitted
- 16 Feb 2023
- Last updated
- 11 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multiple Ascending Doses Study of ZB001 in Chinese Patients With Thyroid-Associated Ophthalmopathy
- ClinicalTrials.gov ID
- NCT05776121
- Acronym / sponsor ID
- ZB001-01-002
- Conditions
- Thyroid Eye Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Zenas BioPharma (USA), LLCIndustry (pharma, biotech, devices)
- Study start
- 10 May 2023 (actual)
- Primary completion
- 17 Apr 2024 (actual)
- Study completion
- 17 Apr 2024 (actual)
- First posted
- 20 Mar 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The investigational drug, ZB001 is a humanized IgG1κ monoclonal antibody targeting human IGF-1R. The study is designed to evaluate the efficacy, safety, tolerability, and pharmacokinetics（PK）/pharmacodynamics (PD) profile of ZB001 in Chinese patients with Thyroid Eye Disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ZB001 for injection | Experimental | Drug: ZB001 for injectionTreat different dose cohorts with four intravenous injections of ZB001 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Male or female adults, 18 years of age or older
- Clinical diagnosis of Graves' ophthalmopathy and CAS evaluation of eyes under study ≥ 4 points (7 points in total)
- Moderate-to-severe active thyroid-associated ophthalmopathy (i.e., severely affects daily life): exophthalmos extent is ≥18.6 mm, or progressive exophthalmos (≥3 mm greater than the previous exophthalmos record per the investigator); accompanied by at least one of the following symptoms: eyelid retraction ≥ 2 mm; moderate or severe soft tissue involvement (conjunctival congestion, edema, periorbital congestion or edema); non-persistent or persistent diplopia
- Before Screening, evidence of eye symptoms or signs related to thyroid-associated ophthalmopathy in medical records for ≤1 year
- Thyroid function normal, or only mild hyperthyroidism or hypothyroidism, defined as free thyroxine (FT4) ）and free triiodothyronine (FT3) levels within 0.5-1.5 times the normal range at Screening. Efforts have been made to correct any mild hypothyroidism or hyperthyroidism in a timely manner and try to maintain normal thyroid function throughout the study. Thyroidectomy is not an exclusion.
Key exclusion criteria (12 listed)
- In the past 6 months, due to optic neuropathy, new visual field defect or color defect secondary to optic nerve involvement, the best corrected visual acuity of the study eye decreased, defined as the result of standardized visual acuity chart decreased ≥ 0.2
- Corneal involvement of the study eye, and no improvement after medical interventions
- CAS decreased ≥ 2 points from Screening Assessment to Day -1
- The exophthalmos extent of the study eye ≥ 2 mm from Screening Assessment to Day -1
- The study eye previously received orbital radiotherapy or surgery due to thyroid-associated ophthalmopathy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ZB001 for injectionDose Cohort1 (3 mg/kg) ZB001 four IV injections | Drug | ZB001 for injection |
| ZB001 for injectionDose Cohort2 (10 mg/kg) ZB001 four IV injections | Drug | ZB001 for injection |
Primary outcome
Number of participants with Adverse Events, Serious Adverse Events, and Laboratory Evaluations as assessed by CTCAE v5.0
Time frame: through study completion, up to 169 days
The exophthalmos response rates of study eye at Week 6 and Week 12 (defined as the proportion of patients whose exophthalmos measured by Hertel exophthalmos meter decreased ≥ 2mm from baseline)
Time frame: At week6 and week 12
24 secondary outcomes
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Exophthalmos measured by MRI/CT scan
Time frame: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Orbital fat volume measured by MRI/CT scan
Time frame: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Extraocular eye muscle volume measured by MRI/CT scan
Time frame: At week6, week 12 and week24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Facial fat volume measured by MRI/CT scan
Time frame: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Digital and manual measurement of palpebral fissure height
Time frame: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Exotropia deviation measurement
Time frame: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Subjective diplopia score. The range of score is from 0-3. The lower score means the better outcome.
Time frame: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Best corrected visual acuity
Time frame: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Intraocular pressure
Time frame: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Clinical Activity Score (CAS) . The range of score is from 0-7. The lower score means the better outcome
Time frame: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Graves ophthalmopathy - Quality of Life (QoL). The range of score is from 0-100. The higher change of quality of life means the better outcome.
Time frame: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Graves ophthalmopathy - Visual function QOL. The range of score is from 0-100. The higher change of quality of life means the better outcome.
Time frame: At week6, week 12 and week 24
Change from baseline in the study eye at Week 6, Week 12 and Week 24 of Graves ophthalmopathy - Social Function QoL. The range of score is from 0-100.The higher change of quality of life means the better outcome.
Time frame: At week6, week 12 and week 24
Serum ZB001 antidrug antibody (ADA) titers
Time frame: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
Serum IGF-1 concentrations in the blood over time
Time frame: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
Maximum observed concentration (Cmax)
Time frame: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
Time to measured peak concentration (Tmax)
Time frame: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
Area under the concentration-time curve from time 0 to the last timepoint with measurable concentration (AUClast)
Time frame: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
Area under the concentration-time curve extrapolated to infinity (AUCinf)
Time frame: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
Area under the concentration-time curve over a dosing interval (AUCtau)
Time frame: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
half life (t1/2)
Time frame: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
Systemic clearance (CL)
Time frame: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
Steady State Volume of Distribution (Vss)
Time frame: Before and after drug administration, performed according to the protocol visit time specified in the plan, up to 169 days
The exophthalmos response rates of study eye at Week 24 (defined as the proportion of patients whose exophthalmos measured by Hertel exophthalmos meter decreased ≥ 2mm from baseline)
Time frame: At week6 and week 12
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Apr 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ZB001 for injection in people with Thyroid Eye Disease, run by Zenas BioPharma (USA), LLC at 9 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with Adverse Events, Serious Adverse Events, and Laboratory Evaluations as assessed by CTCAE v5.0 (time frame: through study completion, up to 169 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Apr 2025.