A study of efgartigimod PH20 SC given by prefilled syringe in adults with thyroid eye disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Argenx

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Eye Diseases [C11]

Trial duration

21 Oct 2024 → 15 Dec 2025

Decision date (initial)

2024-09-29

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

argenx, BV

External identifiers

EU CT number

2023-509198-22-00

ClinicalTrials.gov

NCT06307626

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Pharmacodynamic, Efficacy, Safety

To evaluate the efficacy of efgartigimod PH20 SC compared with placebo PH20 SC at Double- Blind treatment period (DBTP) week 24.

Secondary objectives 1

1. To evaluate the efficacy of efgartigimod PH20 SC compared with placebo PH20 SC

Conditions and MedDRA coding

Thyroid Eye Disease (TED)

Study design 3 periods

Regulatory references

Scientific advice from competent authorities

Center For Drug Evaluation, Pharmaceuticals And Medical Devices Agency, Food And Drug Administration

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. 1. The participant is at least 18 years of age
2. 2. The participant is capable of providing signed informed consent and following with protocol requirements
3. 3. The investigator determines active, moderate-to-severe thyroid eye disease (TED) associated with autoimmune thyroid conditions (Graves’ disease or Hashimoto’s thyroiditis) for the most severely affected eye
4. 4. The participant has first onset of active TED symptoms within 12 months before screening
5. 5. The participant must have normal thyroid function with the baseline disease under control or have mild hypo or hyperthyroidism at screening. Every effort should be made to correct the mild hypo or hyperthyroidism promptly and to maintain the normal thyroid function for the full duration of the study
6. 6. The participant agrees to use birth control consistent with local regulations and the people of child-bearing potential must have a negative blood pregnancy test at screening and a negative urine pregnancy test before receiving the study drug

Exclusion criteria 12

1. 1. Optic neuropathy (damage to optic nerve), defined as new visual field defect (blind spot), relative afferent pupillary defect (pupils respond differently to light), or color defect secondary to optic nerve involvement within the 6 months before screening
2. 2. Corneal decompensation (swelling of the cornea) unresponsive to medical management
3. 3. Previous orbital irradiation or surgery for TED
4. 4. Use of some medications before screening (more information is found in the protocol)
5. 5. Known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of TED or puts the participant at undue risk
6. 6. History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years. Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological findings of prostate cancer
7. 7. Clinically significant active infection that is not sufficiently resolved in the investigator’s opinion or positive serum test at screening for active infection with any of the following: Hepatitis B virus (HBV) , Hepatitis C virus (HCV) , HIV
8. 8. Current participation in another interventional clinical study or previous participation in an efgartigimod clinical study and at least 1 dose of study drug received or has received at least 1 dose of commercially available efgartigimod
9. 9. Known hypersensitivity to study drug or one of its excipients (inactive ingredients)
10. 10. History of or current alcohol, drug, or medication abuse within 12 months before screening as assessed by the investigator.
11. 11. Pregnant or lactating state or intention to become pregnant during the study
12. 12. Live or live-attenuated vaccine received <4 weeks before screening

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Percentage of participants who were proptosis responders at Double-Blind treatment period (DBTP) week 24.

Secondary endpoints 3

1. Change in proptosis measurement in the study eye from baseline to Double-Blind Treatment Period (DBTP) week 24
2. Change in the total GO-QoL score from baseline to Double-Blind Treatment Period (DBTP) week 24
3. Percentage of participants with a resolution of diplopia (responders) at Double-Blind Treatment Period (DBTP) week 24.

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Argenx

Sponsor organisation

Argenx

Address

Industriepark-Zwijnaarde 7

City

Gent

Postcode

9052

Country

Belgium

Scientific contact point

Organisation

Argenx

Contact name

Chief Scientific Officer

Public contact point

Organisation

Argenx

Contact name

Vice President Clinical Development

Third parties 14

Locations

10 EU/EEA countries · 37 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

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

Documents 134 files

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

Application history

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