Phase 3 Study of Tinlarebant in the Treatment in Adolescent Stargardt Subjects

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Belite Bio Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Eye Diseases [C11]

Trial duration

28 Nov 2022 → 11 Sep 2025

Decision date (initial)

2024-05-17

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Belite Bio, Inc

External identifiers

EU CT number

2024-513483-26-00

EudraCT number

2021-003253-36

ClinicalTrials.gov

NCT05244304

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Pharmacodynamic, Efficacy

To assess the efficacy of Tinlarebant in slowing the growth of atrophic lesion(s) in overall adolescent STGD1 subjects

Secondary objectives 3

1. To assess the efficacy of Tinlarebant in slowing the rate of growth of total area of atrophic lesion(s) (i.e. DAF lesions), and change in photoreceptor morphology and BCVA in adolescent STGD1 subjects
2. To evaluate the pharmacodynamics (PD) of Tinlarebant in adolescent STGD1 subjects
3. To assess systemic and ocular safety and tolerability of Tinlarebant

Conditions and MedDRA coding

Stargardt Disease

Study design 3 periods

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-003225-PIP01-22

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Male or female subjects 12 to 20 years old, inclusive.
2. Subject must have clinically diagnosed STGD1 with at least 1 mutation identified in the ABCA4 gene.
3. Subject must have a defined aggregate atrophic lesion size within 3 disc areas (7.62 mm2), as imaged by FAF in the study eye. Subjects must have a BCVA of 20/200 or better for the study eye based on ETDRS letter score
4. Subject and their parent(s) or legal guardian are willing to provide their consent on an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)/Human Research Ethics Committee (HREC)- approved informed consent form (ICF) prior to participating in any study-related procedures. For the complete list, please refer to protocol.

Exclusion criteria 5

1. Any ocular disease other than STGD1 at screening that, in the opinion of the investigator, would complicate assessment of a treatment effect.
2. History of ocular surgery in the study eye in the last 3 months.
3. Investigational drug use of any kind in the last 3 months or within 5 half-lives of the investigational drug, whichever is shorter.
4. Any prior gene therapy.
5. Vitamin A deficiency as defined based upon plasma values less than 20 μg/dL (=0.7 μmol/L). For the complete list, please refer to protocol.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary efficacy endpoint is the annualized rate of lesion growth in the aggregate area of atrophy (definitely decreased autofluorescence [DDAF]) from baseline as assessed by FAF photography. The primary assessment of efficacy will be performed at Month 25.

Secondary endpoints 6

1. · Annualized rate of lesion growth in total area of decreased autofluorescence (DAF; the sum of DDAF and questionably decreased autofluorescence [QDAF]) from baseline by FAF photography
2. · Change in photoreceptor morphology (EZ defect area) from baseline to post baseline assessments (assessed by SD-OCT)
3. · Change in BCVA measured by the ETDRS method under standard luminance from baseline to post baseline assessments
4. · Change in RBP4 levels from baseline to Month 25
5. · Correlation between the change in RBP4 level and the change of aggregate lesion size from baseline to Month 25
6. Physical examination, vital signs measurement, ECG, ophthalmic examination, clinical laboratory tests (including serum chemistry and hematology panels, urinalysis, and pregnancy tests on all female subjects), retinol chemistries (plasma retinol, plasma RBP4), visual function questionnaire, measurement of intraocular pressure (IOP), dark adaptation test, dilated funduscopy, contrast sensitivity assessment of AEs, and monitoring of concomitant medications.

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Belite Bio Inc.

Sponsor organisation

Belite Bio Inc.

Address

Ugland House, P. O. Box 309 P. O. Box 309

City

Grand Cayman

Postcode

KY1-1104

Country

Cayman Islands

Scientific contact point

Organisation

Belite Bio Inc.

Contact name

Nathan Mata

Public contact point

Organisation

Belite Bio Inc.

Contact name

Clinical Project Manager

Third parties 4

Locations

4 EU/EEA countries · 4 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 42 files

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

Application history

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