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CTx001

Phase 1

Geographic Atrophy Secondary to Age-related Macular Degeneration | Gene therapy | Ophthalmology |cbdMD, Inc.|Last Updated: Jun 22, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment75

FDA Designations

No designations recorded

Clinical trial landscape

CTx001 · 1 trial · 1 indication

Phase 1 1
NCT07392255An Optimised GA Interventional Trial (Opti-GAIN) to Test if Treatment With CTx001 is Safe and Works for People With Geographic Atrophy (GA)Geographic Atrophy Secondary to Age-related Macular Degeneration
RECRUITING75 Analytics
PHASE1RECRUITING
An Optimised GA Interventional Trial (Opti-GAIN) to Test if Treatment With CTx001 is Safe and Works for People With Geographic Atrophy (GA)
Geographic Atrophy Secondary to Age-related Macular DegenerationUnlock trial analytics

Study Endpoints

Primary Endpoints

To monitor the safety and tolerability of a single administration of CTx001 at 3 dose levels
From dosage to Week 52

Incidence and severity of ocular and non-ocular adverse events (AE)s and serious AEs (SAEs) up to Week 52 (Year 1)

Secondary Endpoints

To evaluate the preliminary efficacy of treatment with CTx001 through changes in the structural and functional parameters of geographic atrophy (GA)
From dosage to Year 1
To monitor the long-term safety and tolerability of a single administration of CTx001 at 3 dose levels
From dosage up to Week 260 (Year 5)
To assess immunogenicity to adeno-associated virus Serotype 2 (AAV2)-vector and mini-CR1 transgene product
From dosage up to Week 260 (Year 5)
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort 1EXPERIMENTALLow Dose
Cohort 2EXPERIMENTALMedium Dose
Cohort 3EXPERIMENTALHigh Dose
Cohort 4EXPERIMENTALExpansion of a dose selected from Cohort 1-3
Cohort 5EXPERIMENTALExpansion of a second dose selected from Cohort 1-3

Interventions

NameTypeDescription
CTx001GENETICSubretinal administration of CTx001
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Eligibility Criteria

Age Range55 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites5

Inclusion Criteria: Meet protocol-defined age eligibility Have bilateral geographic atrophy secondary to AMD, confirmed by the Reading Center Meet baseline lesion size requirements, as assessed by fundus autofluorescence imaging Meet best-corrected visual acuity and low-luminance visual acuity c...

Countries:United StatesUnited Kingdom
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Frequently asked questions about CTx001

What is CTx001 used for in Geographic Atrophy Secondary to Age-related Macular Degeneration?

CTx001 is an investigational gene therapy being developed for the treatment of Geographic Atrophy Secondary to Age-related Macular Degeneration. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The therapy is being studied in a controlled clinical trial to evaluate its safety and efficacy.

Who makes CTx001?

CTx001 is being developed by cbdMD, Inc., a company traded on the stock exchange under the ticker symbol YCBD. The company is conducting a Phase 1 clinical trial of CTx001 for Geographic Atrophy Secondary to Age-related Macular Degeneration.

What phase is CTx001 in?

CTx001 is currently in Phase 1 clinical development. It is an investigational gene therapy and has not yet been approved by the FDA or other regulatory agencies. The ongoing Phase 1 trial is actively recruiting participants to assess the safety and potential efficacy of the treatment.

What clinical trials is CTx001 in?

CTx001 is being evaluated in a Phase 1 clinical trial with the identifier NCT07392255, titled "An Optimised GA Interventional Trial (Opti-GAIN) to Test if Treatment With CTx001 is Safe and Works for People With Geographic Atrophy (GA)." The trial is recruiting 75 participants in the United States and United Kingdom.

Is CTx001 a gene therapy?

Yes, CTx001 is a gene therapy being developed for Geographic Atrophy Secondary to Age-related Macular Degeneration. It is currently in Phase 1 clinical trials, where it is being tested in a controlled study to determine if the treatment is safe and effective for patients with this condition.