European Union · Phase II and III, including I/II · Stargardt disease
EU Phase II and III Stargardt disease trials: sites, PIs and sample sizes
Ghent University Hospital appears in five of six Stargardt studies, followed by Quinze-Vingts with four. The studies list 29 EU sites and 32 principal investigators across six countries, spanning oral treatments, subretinal gene therapy and long-term follow-up.
: Expanded to include Phase II and combined Phase I/II studies alongside Phase III.
three III, two I/II, one II
across six countries
across all six studies
Top 10 sites · All six studies
Ghent spans five studies across oral and gene therapies
Quinze-Vingts follows with four studies, while Bonn and Radboud each appear in three. These are distinct study appearances, including planned and historical sites, not measures of recruitment performance.
Results by trial setting:
Phase IIIThree oral-treatment studiesGhent and Quinze-Vingts each appear in all three. NORTHSTAR lists 27 EU sites, DRAGON four and the tinlarebant extension three.
Phase I/IICELESTE and ASTRAThese gene-therapy studies share Ghent and together list six EU sites. CELESTE adds the Rotterdam Eye Hospital to the wider Stargardt network.
Long-term follow-upSAR422459The Phase II-coded follow-up lists Quinze-Vingts as its sole EU site. It follows previously treated participants.
NORTHSTAR’s six French sites are listed in a country record marked “Not authorised”. They remain in these all-status participation counts and should not be treated as authorised or open sites.
Top 10 principal investigators · All six studies
Bart Leroy appears in five studies
Isabelle Audo and Philipp Herrmann each appear in three. Carel Hoyng and Francesca Simonelli each appear in two.
Results by trial setting:
Phase IIIThree studiesBart Leroy and Isabelle Audo each appear in three, followed by Carel Hoyng and Philipp Herrmann with two. The tinlarebant extension adds a study appearance for returning trial teams.
Phase I/IISubretinal gene therapyBart Leroy appears in both CELESTE and ASTRA. CELESTE also names Francesca Simonelli, L. I. van de Born and Suzanne Yzer. ASTRA names Katharina Stingl and Philipp Herrmann.
Long-term follow-upSAR422459The French follow-up names José-Alain Sahel. Quinze-Vingts therefore has four study appearances across two named principal investigators.
All six participating EU countries
Belgium appears in five studies, France in four
Germany and the Netherlands each appear in three studies. Italy appears in two and Spain in one.
Results by trial setting:
Phase IIISix-country networkBelgium and France appear in all three studies, Germany and the Netherlands in two, and Italy and Spain in NORTHSTAR. Spain has seven NORTHSTAR sites, the largest national site count in that trial.
Phase I/IIFour EU countriesCELESTE lists Belgium, Italy and the Netherlands. ASTRA lists Belgium and Germany. Belgium is their shared EU country. CELESTE also has a Norwegian site, outside the EU counts.
Long-term follow-upFranceSAR422459 adds one French study appearance. The separate tinlarebant extension lists Belgium, France and the Netherlands.
A multinational trial can appear in several countries. The French NORTHSTAR entry is not authorised, as noted above.
Planned sample sizes · Worldwide participants
NORTHSTAR has the largest planned enrollment at 230
The two randomized Phase III trials have a mean original planned size of 160 participants. The Phase I/II gene-therapy studies plan 75 and 86 participants, averaging 80.5.
Results by trial setting:
Phase IIIPlanned versus actualNORTHSTAR plans 230 and DRAGON originally targeted 90. DRAGON ultimately randomized 104. The tinlarebant extension plans 70 returning participants, giving a planned mean of 130 across the three Phase III studies.
Phase I/IICELESTE and ASTRACELESTE’s protocol plans 75, approximately 15 in Part A and 60 in Part B. ASTRA’s CTIS targets total 86, comprising 13 in the EU and 73 elsewhere. Its older April 2025 ClinicalTrials.gov record estimates 57.
Long-term follow-upSAR422459The registry reports 27 actually enrolled. That figure is kept separate from planned-enrollment comparisons.
Targets cover worldwide participants. Extension and follow-up studies include previously treated people, so study enrollment figures must not be added as a count of unique patients.
The six EU Stargardt studies
Phase I/II studies are included because they contain a Phase II component. SAR422459 is coded Phase II in CTIS and titled a Phase I/II follow-up study.
| Study / EU CT number | Phase | Treatment and setting | EU sites |
|---|---|---|---|
| NORTHSTAR 2026-525296-60-00 | III | Gildeuretinol, randomized placebo-controlled | 27 listed six French sites not authorised |
| DRAGON 2024-513483-26-00 | III | Tinlarebant, randomized placebo-controlled, ended in EU countries | 4 |
| Tinlarebant extension 2025-522460-33-00 | III | Open-label extension for parent-study completers | 3 |
| CELESTE 2025-522207-15-01 | I/II | AAVB-039, subretinal gene therapy | 4 |
| ASTRA 2024-519535-42-00 | I/II | SB-007, subretinal gene therapy | 3 |
| SAR422459 follow-up 2024-513501-31-00 | II in CTIS | Long-term follow-up after gene therapy | 1 |
Sources: CTIS trial records and protocols and the linked ClinicalTrials.gov records, reviewed 17 September 2026. DRAGON’s original enrollment target was 90. Site and investigator rankings count distinct protocols, including extensions and follow-up. Ties are ordered alphabetically, including at the tenth position.