An efficacy and safety study of cenegermin ophthalmic solution compared with vehicle in the treatment of PCED

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Dompe' Farmaceutici S.p.A.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Eye Diseases [C11]

Decision date (initial)

2026-05-05

Transition trial

No

Low-intervention

Yes

Rare-disease indication

No

Vulnerable population

No

Funding sources

Dompé farmaceutici S.p.A.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

To evaluate the efficacy of cenegermin ophthalmic solution compared to vehicle in inducing complete healing of the PCED after 4 weeks of treatment.

Secondary objectives 4

1. To evaluate the efficacy of cenegermin ophthalmic solution compared to vehicle in improving the PCED from baseline after 4 weeks of treatment
2. To evaluate the efficacy of cenegermin ophthalmic solution compared to vehicle in improving the PCED from baseline after 8 weeks of treatment
3. To evaluate the efficacy of cenegermin ophthalmic solution compared to vehicle in inducing complete healing of the PCED after 8 weeks of treatment
4. To evaluate the efficacy of cenegermin ophthalmic solution compared to vehicle in improving the PCED from baseline after 4 weeks of treatment

Conditions and MedDRA coding

Persistent Corneal Epithelial Defect (PCED)

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Men or women aged 18 years or above
2. Participants with PCED in the study eye with the following characteristics: a. PCED ≥ 1.0 mm in greatest diameter b. PCED of at least 14 days duration, refractory to 1 or more conventional nonsurgical treatments (ocular lubricants, discontinuation of preserved drops and medications, bandage contact lens) showing no clinical resolution.
3. Use of most ophthalmic medications (including glaucoma medications) indicated for ocular conditions other than PCED is permitted in the study eye, if the participant has been on a stable dose for at least 30 days and does not expect to have change in dosing regimen throughout the entire duration of the study. Please see exclusion criteria list for exceptions.
4. Use of prophylactic antibiotics in the study eye is permitted if the participant is already receiving them prior to enrollment.
5. Able to sign the inform consent as described in the protocol, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF).

Exclusion criteria 14

1. Contralateral eye with vision of no light perception or anatomic absence of contralateral eye.
2. Active ocular infection or inflammation in the study eye as follows: a. Bacterial, fungal, or protozoal infection at screening b. Active infectious stromal infiltrates or edema at screening c. Acute anterior uveitis of grade 2 or greater (SUN 2025) within 30 days of screening d. Acute intermediate uveitis or posterior uveitis within 30 days of screening e. Acute inflammation of the sclera or conjunctiva if it is not associated with the PCED
3. Corneal epithelial defect associated with stromal thinning greater than 30% (estimated on clinical slit lamp exam) or if associated with stromal infiltrate (corneal haze is acceptable), in the study eye.
4. Severe eyelid disease in the study eye, such as: a. Mechanical eyelid abnormalities that have direct contact with the PCED (e.g., trichiasis, severe entropion with lid margin keratinization, etc, if in direct contact with the PCED) b. Lagophthalmos greater than 2 mm as measured in the clinic c. Existing diagnosis of nocturnal lagophthalmos or Parkinson’s disease d. Inability to fully close eyelids despite voluntary eyelid closure e. Severe ectropion with abnormal eyelid-globe congruity (e.g., the lower eyelid does not come into contact with the globe due to severe ectropion)
5. Severe end-stage ocular surface disease in the study eye, including but not limited to: a. Severe limbal stem cell deficiency, defined as involvement of more than 270 cumulative degrees or more of limbal stem cell deficiency b. Keratinization of the bulbar conjunctiva or lid margin
6. Use of the following medications and devices within the indicated time window prior to randomization: a. Local medications in study eye: − Any prior use of cenegermin − Blood-derived (autologous serum) or other ocular surface re-epithelizing agents, anesthetic use by the participant outside of the clinical exam setting, insulin, or steroids (unless associated with post-operative treatment regimen) within 7 days − Botox (botulinum toxin) injections for pharmacologic tarsorrhaphy within 90 days b. Systemic medications: − High-dose systemic corticosteroids (greater than 0.5 mg/kg/day) within 30 days − Any changes in oral medication regimen intended for the treatment of the ocular surface (eg, oral doxycycline) within 30 days, or planned changes during the study period − Systemic opioid use within 30 days − Use of any systemic investigational product, ocular investigational product in the study eye, radiation of the head or neck, or systemic chemotherapy within 90 days, or planned to occur during the study period c. Devices: − Use of contact lens (including therapeutic contact lens) within 7 days, or planned use of contact lens during the study period, in the study eye − Anticipated need for punctal occlusion in the study eye during the study period. Participants with punctal occlusion or punctal plugs inserted prior to the study are eligible for enrolment provided that the punctual occlusion is maintained throughout the study.
7. Presence of acute severe systemic disease as follows: a. Any acute or active severe systemic inflammatory disease (eg, acute systemic Stevens-Johnson Syndrome, acute systemic GVHD, severe systemic Sjogren’s, mucous membrane pemphigoid) b. Presence of any systemic disease that may affect ability to participate in the clinical study according to the clinical judgment of the investigator
8. Recent surgery or amniotic membrane therapy as follows: a. Recent major surgical procedure for the treatment of PCED (eg, conjunctival flap, complete tarsorrhaphy, superficial keratectomy for epithelial defect revision, etc) within 14 days of randomization b. Presence of amniotic membrane from AMT for ocular surface indication if not dissolved within area of PCED within 14 days of randomization. Examples include: − Sutured AMT − Self-retaining AMT −Contact lens combined with AMT −Other AMT treatment for the ocular surface c. Partial tarsorrhaphy (temporary or permanent) placed within 14 days of randomization. If a participant is enrolled with partial tarsorrhaphy placed more than 14 days prior to randomization, then the tarsorrhaphy must not be removed for the entire duration of the study.
9. Contraception: a. Females of child-bearing age (defined as not surgically sterilized or post-menopausal for at least 1 year) are excluded if they meet any 1 of the following conditions: − Are known to be pregnant − Have a positive urine pregnancy test at baseline visit − Are planning to become pregnant during study period − Are breastfeeding − Are unwilling to use acceptable form of contraception until 30 days after the study treatment period is complete b. Male fertile participants (ie, not surgically sterilized by vasectomy) unwilling to use an acceptable form of contraception (male condom with spermicidal cream or jelly) until 30 days after the study treatment period is complete
10. Known active substance abuse or dependency, including but not limited to alcohol, illicit drugs, marijuana, or misuse of prescription medications within 30 days of randomization
11. Known or suspected ocular malignancy (e.g., ocular surface, intraocular, ocular adnexa), or presence of cancer or any other systemic disease that may affect the ability to participate in the clinical study in the opinion of the investigator including basal cell carcinoma of the head
12. Any ocular or systemic disorder that might hinder the efficacy of the study treatment or its evaluation or could be judged by the investigator to be incompatible with the study visit schedule or conduct
13. Concurrent participation in another investigational study
14. Hypersensitivity: a. Known or suspected allergy to any components of the cenegermin formulation b. Known hypersensitivity to 1 of the components of the study or procedural medications (eg, fluorescein)

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Achieving complete epithelial healing at week 4 and maintained at week 8.

Secondary endpoints 4

1. Percentage change from baseline in maximum diameter of PCED at week 4
2. Percentage change from baseline in maximum diameter of PCED at week 8
3. Achieving complete epithelial healing at week 8 and maintained at week 10
4. Linear change from baseline in maximum diameter (in mm) of the PCED at week 4

Investigational products

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

Test 1

Placebo 1

Auxiliary 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Dompe' Farmaceutici S.p.A.

Sponsor organisation

Dompe' Farmaceutici S.p.A.

Address

Via Santa Lucia 6

City

Milan

Postcode

20122

Country

Italy

Scientific contact point

Organisation

Dompe' Farmaceutici S.p.A.

Contact name

Dompé Medical Expert

Public contact point

Organisation

Dompe' Farmaceutici S.p.A.

Contact name

Dompé ServiceDesk

Third parties 5

Locations

8 EU/EEA countries · 36 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 71 files

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

Application history

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