DRAGON

24 evidence topics · 11 sources

Objective

Primary study objective

“assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1” (opens the source at this quote in a new tab)

Location and study date

Original UK protocol: ethics opinion

Field	Quoted record
Ethics opinion date	“25 Nov 2021”
Ethics committee	“Wales REC 5”

Completion of the final study visit

“In September 2025, we completed the last subject visit in the Phase 3 DRAGON trial.” (opens the source at this quote in a new tab)

Study design

DRAGON pivotal phase 3 trial: trial design

“Completed, 24-month, 104 subjects, aged 12 to 20 years old, randomized (2:1, active: placebo), double-masked, placebo-controlled, global, multi-center, pivotal Phase 3 trial” (opens the source at this quote in a new tab)

Eligibility criteria

DRAGON pivotal phase 3 trial: enrollment criteria

“All enrolled patients had an aggregate atrophic lesion size of 0.05–7.62 mm² and best-corrected visual acuity of ≥ 20/200.” (opens the source at this quote in a new tab)

Treatment

DRAGON pivotal phase 3 trial: treatment groups

“Tinlarebant”	“placebo”
“n=69”	“n=35”

Study outcomes

Rationale for using DDAF lesion growth as the primary endpoint

Primary endpoint and reported regulatory agreement

“Primary Endpoint: Reduction in DDAF lesion growth rate, assessed by FAF imaging (as agreed with the FDA)” (opens the source at this quote in a new tab)

Planned patient-reported assessments

“quality of life questionnaires.” (opens the source at this quote in a new tab)

Visual-acuity endpoint: natural-history rate of change

“Overall, BCVA declined at a rate of 0.011 logMAR per year (−0.55 letters/y)” (opens the source at this quote in a new tab)

Statistical analysis description

Number of participants (Planned and analyzed)

Original UK protocol: planned enrollment

Population	Planned participants
Global	“60”
United Kingdom	“6”

DRAGON: randomized and analyzed populations

Population	Quoted record
Safety set	“N = 104”
Modified full analysis set	“N = 96”

Description of analysis sets

Modified full analysis set: baseline and post-baseline requirements

“mFAS subjects must have a defined DDAF lesion meeting the eligibility criteria at baseline and have at least one post baseline assessment.” (opens the source at this quote in a new tab)

Primary statistical model

“an unstructured covariance matrix under the Mixed Model for Repeated Measures (MMRM).” (opens the source at this quote in a new tab)

Results

Participant disposition

DRAGON pivotal phase 3 trial: treatment-related discontinuations

“four treatment-related discontinuations” (opens the source at this quote in a new tab)

Interim participant disposition

“By the time of the interim analysis, 87 (84%) of subjects had completed Month 16.” (opens the source at this quote in a new tab)

Baseline characteristics

Efficacy results

DRAGON: primary-endpoint results

Endpoint, population, treatment, or analysis	Source quotation
“DDAF”	“Study Eye”
“mFAS”	“N = 96”
“Tinlarebant 5 mg”	“0.38 mm²/year”
“Placebo”	“0.59 mm²/year”
“treatment effect size”	“35.7%”
“unstructured covariance matrix”	“P = 0.0033”

DRAGON pivotal phase 3 trial: visual acuity

“the overall change in visual acuity was minimal over the period of 24 months in both study groups” (opens the source at this quote in a new tab)

DRAGON quantitative autofluorescence secondary endpoint: tinlarebant

“qAF values remained stable to slightly decreased from baseline (approximately 2%)” (opens the source at this quote in a new tab)

DRAGON quantitative autofluorescence secondary endpoint: placebo

“placebo-treated subjects showed an approximate 20% increase in qAF from baseline.” (opens the source at this quote in a new tab)

Health-related quality of life and patient-reported outcomes

No evidence found.

Safety results

DRAGON pivotal phase 3 trial: adverse events

“Xanthopsia and delayed dark adaptation were the most common drug related ocular adverse events.” (opens the source at this quote in a new tab)
“Headaches were the most commonly reported treatment-related non-ocular adverse events.” (opens the source at this quote in a new tab)

DRAGON pivotal phase 3 trial: serious non-ocular treatment-emergent adverse events

“6 serious non-ocular TEAEs” (opens the source at this quote in a new tab)
“4 assessed as unrelated and 2 assessed as unlikely related to the study treatment” (opens the source at this quote in a new tab)

Non-ocular adverse events: safety population

Category	Tinlarebant 5 mg, 69 participants	Placebo, 35 participants
Any non-ocular TEAE	“59 (85.5%)”	“27 (77.1%)”
Serious non-ocular TEAE	“2 (2.9%)”	“4 (11.4%)”
Drug-related non-ocular TEAE	“14 (20.3%)”	“4 (11.4%)”
Non-ocular TEAE resulting in study-drug discontinuation	“0 (0.0%)”	“0 (0.0%)”

Ocular adverse events: safety population

Category	Tinlarebant 5 mg, 69 participants	Placebo, 35 participants
Any ocular TEAE	“53 (76.8%)”	“8 (22.9%)”
Drug-related ocular TEAE	“49 (71.0%)”	“8 (22.9%)”
Serious ocular TEAE	“0 (0.0%)”	“0 (0.0%)”
Ocular TEAE resulting in study-drug discontinuation	“4 (5.8%)”	“0 (0.0%)”
Ocular TEAE resulting in study discontinuation	“2 (2.9%)”	“0 (0.0%)”

Study limitations

Summary

DRAGON enrolled 104 participants aged 12–20 years with defined atrophic lesions and visual acuity of 20/200 or better. Its modified full analysis set included 96 participants, rather than all randomized participants. The primary outcome was anatomical lesion growth; minimal visual-acuity change in both groups limits conclusions about functional benefit during the 24-month treatment period. These eligibility restrictions also limit direct applicability to younger children and participants outside the studied lesion-size and visual-acuity ranges.