Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Ongoing, recruitment ended
Participants planned
100
Countries
1
Sites
11
Retinopathy of Prematurity
The primary objective in this study is to examine whether preventative treatment with topical Dexamethasone treatment may reduce the proportion of infants who need conventional ROP treatment in comparison to Placebo arm.
Key facts
- Sponsor
- Vaestra Goetalandsregionen
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Eye Diseases [C11]
- Trial duration
- 22 Sep 2022 → ongoing
- Decision date (initial)
- 2023-08-30
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Vetenskapsrådet
External identifiers
- EU CT number
- 2023-505318-97-00
- EudraCT number
- 2020-004933-19
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy
The primary objective in this study is to examine whether preventative treatment with topical Dexamethasone treatment may reduce the proportion of infants who need conventional ROP treatment in comparison to Placebo arm.
Secondary objectives 7
- 1) To study time from detection of proliferative ROP to Type 1 ROP in Dexamethasone treatment and Placebo arm and will be evaluated by following secondary outcomes: a. Time from randomization to Type 1 ROP or end of treatment phase study (all infants) b. Time from randomization to Type 1 ROP (infants without regression
- 2) To study recurrences after laser/Anti-VEGF treatment in Dexamethasone treatment and Placebo arm and will be evaluated by following secondary outcome: a. The proportion of infants with any recurrences after laser/Anti-VEGF treatment (all infants) b. The number of recurrences per infant per follow-up time after laser/Anti-VEGF treatment (all infants) c. The proportion of infants with any recurrences after laser/Anti-VEGF treatment (infants progressed to Type 1 ROP) d. The number of recurrences per infant per follow-up time after laser/Anti-VEGF treatment (infants progressed to Type 1 ROP)
- 3) To study retinal morphology at 40 weeks PMA and at 2.5 and 6.5 years with optical coherence tomography (OCT) in Dexamethasone treatment and Placebo arm and will be evaluated by: a. Optic disc, cup and rim area at 40 weeks PMA and 2.5 and 6.5 years of age b. Retinal nerve fiber thickness at 40 weeks PMA and 2.5 and 6.5 years of age c. Macular thickness at 40 weeks PMA and 2.5 and 6.5 years of age
- 4) To study measurements of intraocular pressure before treatment at 1 and 2 weeks after start of treatment and after completed treatment and at 2.5 and 6.5 years will be evaluated by: a. Change in IOP (mmHg) from before treatment, 1-2 weeks after treatment, and after end of treatment
- 5) To study visual outcome and refractive errors at 2.5 and 6.5 years of age in Dexamethasone treatment and Placebo arm and will be evaluated by: a. Visual acuity at 2.5 (Fix and follow) and 6.5 years (KM or HVOT) of age b. Refraction at 2.5 and 6.5 years of age c. Orthoptic status including motility, stereovision, presence of nystagmus and strabismus at 2.5 and 6.5 years of age
- 6) To evaluate safety and tolerability of the treatment through adverse events, clinical chemistry, retinal exam, intraocular pressure, physical examination, MRI of the brain, body composition with PeaPod (Lund and Gothenburg) and vital signs.
- 7) To analyse leftovers of blood from clinical blood sampling with regard to selected biomarkers and their relation to ROP development and treatment effect.
Conditions and MedDRA coding
Retinopathy of Prematurity
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.1 | LLT | 10036858 | Proliferative retinopathy | 10015919 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- 1) Gestational age <30 weeks
- 2) Zone I ROP without plus disease with either a. ROP Stage 1, or ROP Stage 2 or Zone II ROP without plus disease with either a. Posterior ROP stage 2 or b. ROP stage 3 with or without notch in zone I
- 3) ROP stage documented using RetCam photography and confirmed by two ophthalmologists
- 4) Parent’s informed (oral and written information) consent.
Exclusion criteria 2
- 1) Ongoing ocular infection
- 2) If the patient is judged unsuitable for the study by treating neonatologist/paediatrician or ophthalmologist for any other reason
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- The proportion of infants with ROP progression to Type 1 ROP needing conventional treatment in each study arm.
Secondary endpoints 6
- 1. Time from detection of proliferative ROP to Type 1 ROP
- 2. Recurrences after laser/Anti-VEGF treatment in dexamethasone treatment and placebo arm
- 3. Retinal morphology at 40 weeks PMA and at 2.5 and 6.5 years with optical coherence tomography (OCT)
- 4. Measurements of intraocular pressure before treatment at 1 and 2 weeks after start of treatment and after completed treatment and at 2.5 and 6.5 years of age
- 5. Visual outcome and refractive errors at 2.5 and 6.5 years of age
- 6. To analyse leftovers of blood from the clinical blood sampling with regard to selected biomarkers and their relation to ROP development and treatment effect
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
DEXAFREE 1 mg/ml, collyre en solution en récipient unidose
PRD317019 · Product
- Active substance
- Dexamethasone Phosphate
- Substance synonyms
- DEXAMETHASONE 21-(DIHYDROGEN PHOSPHATE)
- Pharmaceutical form
- EYE DROPS, SOLUTION
- Route of administration
- EYE/EAR/NOSE DROPS
- Max daily dose
- 0.05 mg milligram(s)
- Max total dose
- 4.2 mg milligram(s)
- Max treatment duration
- 12 Week(s)
- Authorisation status
- Authorised
- ATC code
- S01BA01 — DEXAMETHASONE
- Marketing authorisation
- 34009 374 774 5 1
- MA holder
- LABORATOIRES THEA
- MA country
- France
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Placebo 1
"DROP-it" ögondroppar 5 ml Physiologic saline 0,9 % natriumklorid i 100 ml renat vatten
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Vaestra Goetalandsregionen
- Sponsor organisation
- Vaestra Goetalandsregionen
- Address
- Regionens Hus
- City
- Vänersborg
- Postcode
- 462 80
- Country
- Sweden
Scientific contact point
- Organisation
- Vaestra Goetalandsregionen
- Contact name
- Ann Hellström
Public contact point
- Organisation
- Vaestra Goetalandsregionen
- Contact name
- Ann Hellström
Locations
1 EU/EEA country · 11 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Sweden | Ongoing, recruitment ended | 100 | 11 |
| Rest of world | — | 0 | — |
Investigational sites
Queen Silvia Childrens Hospital - Sahlgrenska University Hospital - Vastra Gotalandsregionen
Sahlgrenska University Hospital, Department of Ophtalmology, Diagnosvägen 11, 416 85 Göteborg, Behandlingsvagen 7, Harlanda, Gothenburg
Region Skane Skanes Universitetssjukhus
Department of Ophtalmology, Entrégatan 7, Lund, Entregatan 7, Lunds Allhelgonafors, Lund
Skaraborg Hospital-Vastra Gotalandsregionen
Department of Ophtalmology, Lövängsvägen 1, 541 42 Skövde, Lovangsvagen 1, 541 42, Skovde
Uppsala University Hospital
Department of Surgical Sciences, Ophtalmic Biophsics. Akademiska sjukhuset, 75185 Uppsala, Akademiska Sjukhuset, 751 85, Uppsala
Region Joenkoepings Laen
Department of Ophtalmology, Lanssjukhuset Ryhov, Sjukhusgatan, Jonkoping
Region Vaesterbotten
Department of Ophtalmology, Norrlands universitetssjukhus, Daniel Naezéns väg, 907 37 Umeå, Koksvagen 11, Alidhem, Umea
Linkoping University Hospital Region Ostergotland
Department of Ophtalmology, Universitetssjukhuset I Linkoping, 581 85, Linkoping
Region Vaestmanland
Department of Ophtalmology, Västmanlands sjukhus Västerås, Sigtunagatan, 721 89 Västerås, Sigtunagatan, 721 89, Vasteras
Region Gaevleborg
Gävle Hospital, Department of Ophatlomolgy. Lasarettsvägen 1, 803 24 Gävle, Lasarettsvagen 1, 803 24, Gavle
St Erik Eye Hospital
Department of Ophtalmology, Eugeniavägen 12, 171 64 Stockholm, Eugeniavagen 12, 171 64, Solna
Region Vaermland
Department of Ophtalmology, Rosenborgsgatan, Centralsjukhuset, 652 30 Karlstad, Rosenborgsgatan 50, 652 33, Karlstad
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Sweden | 2022-09-22 | 2022-10-03 | 2025-09-17 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 3 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol_2023-505318-97-00 | 5.0 |
| Summary of Product Characteristics (SmPC) (for publication) | G2_SmPC Dexafree | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol_synopsis_2023-505318-97-00 | 1 |
Application history
2 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-08-17 | Sweden | Acceptable 2023-08-29
|
2023-08-30 |
| 2 | SUBSTANTIAL MODIFICATION | SM-4 | 2025-10-21 | Sweden | Acceptable 2025-12-08
|
2025-12-11 |