A Study to Investigate the Safety, Tolerability, and Exposure of Single Doses of the study medicine STK-002, in Patients with Autosomal Dominant Optic Atrophy (ADOA)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Stoke Therapeutics Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

8 Dec 2025 → ongoing

Decision date (initial)

2024-05-06

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Stoke Therapeutics, Inc.

External identifiers

EU CT number

2023-506290-35-00

WHO UTN

U1111-1295-3741

ISRCTN

ISRCTN41725621

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Others

• To evaluate the safety and tolerability of single ascending doses of STK 002 in patients with ADOA
• To determine the exposure in serum following single intravitreal (IVT) doses of STK-002

Secondary objectives 2

1. • To evaluate changes in visual function and ocular structure following single doses of STK-002
2. • To evaluate the effect of single doses of STK-002 on quality of life in patients with ADOA

Conditions and MedDRA coding

Autosomal Dominant Optic Atrophy

Regulatory references

Scientific advice from competent authorities

Medicines And Healthcare Products Regulatory Agency

Plan to share IPD

No

IPD plan description

Currently there are no plans to directly release study data to others. However, results of the study may be shared as: Publication in peer reviewed scientific journals and conference presentations, Publication of summary results on public registries, Submission to regulatory authorities as applicable.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. 1. Patient must be ≥18 to <55 years to participate in Part A and ≥6 to <18 years to participate in Part B
2. 2. Patient must have a clinical diagnosis of ADOA and have a heterozygous OPA1 gene variant confirmed at Screening by central lab genotyping
3. 3. Patient must have a BCVA EDTRS letter score of ≥35 and ≤70 with each eye individually, with the exception of the first two patients in Cohort 1 of Part A who must have a BCVA ETDRS letter score ≥5 and ≤35 in each eye

Exclusion criteria 5

1. Patient has a gain-of-function variant, or compound heterozygous or homozygous pathogenic or likely pathogenic variant in the OPA1 gene
2. Patient has extraocular phenotypic manifestations of (syndromic) ADOA (ADOA-plus) or have Behr syndrome
3. Patient has, or has a history of, any ocular condition in either eye that, in the opinion of the Investigator, could affect study parameters
4. Patient is considered to be at risk for uveitis or ocular infection during the study period
5. Patient is taking, or has taken at any time, any medication or treatment that can or might cause an optic neuropathy

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Safety variables for analysis
2. The exposure of STK-002 in serum will be determined using Pharmacokinetic (PK) parameters

Secondary endpoints 7

1. Peripapillary retinal nerve fiber layer (RNFL) and macular ganglion cell layer (GCL, or ganglion cell complex) thicknesses as assessed by optical coherence tomography (OCT)
2. Best-corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) optotypes
3. Contrast sensitivity as assessed by low contrast BCVA (at 25%, 5% and 2.5% contrast levels)
4. Visual field (as assessed by automated, static perimetry [10-2 and 24-2 Swedish Interactive Threshold Algorithm (SITA) FAST])
5. Electrical activity of the retina as assessed by phototopic negative response (PhNR) ERG (if available)
6. Continuous text reading acuity as assessed by MNREAD Acuity Charts
7. Quality of life as measured by scores on the National Eye Institute Visual Function Questionnaire-25 (NEI-VFQ-25), Impact of Vision Impairment for Children (IVI-C), and the European Quality of Life-5 Dimensions (EQ-5D)/EQ-5D-Y questionnaire

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Stoke Therapeutics Inc.

Sponsor organisation

Stoke Therapeutics Inc.

Address

45 Wiggins Avenue

City

Bedford

Postcode

01730-2314

Country

United States

Scientific contact point

Organisation

Stoke Therapeutics Inc.

Contact name

Dr Steven Gross, M.D.

Public contact point

Organisation

Stoke Therapeutics Inc.

Contact name

Dr Steven Gross, M.D.

Third parties 6

Locations

4 EU/EEA countries · 5 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Application history

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