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Virus Therapy

AAV2-hCHM for Choroideremia

Phase 1 & 2
Waitlist Available
Research Sponsored by Spark Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights

Study Summary

This trial will check if a new gene therapy for choroideremia is safe and works well.

Eligible Conditions
  • Choroideremia

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Secondary outcome measures
Number of Participants With Anti-AAV2 Viral Capsid Antibody Titers That Rose Above Baseline At Least Once After Dosing
Number of Participants With Cellular Immune Response to AAV2 Through Interferon Gamma Enzyme-linked Immunosorbent Spot (ELISpot) Assay
Number of Participants With Cellular Immune Response to Rab Escore Protein-1 (REP-1) Through Interferon Gamma ELISPOT Assay

Side effects data

From 2022 Phase 1 & 2 trial • 15 Patients • NCT02341807
40%
Cataract
40%
Sinusitis
20%
Diplopia
20%
Diarrhoea
20%
Insomnia
20%
Vitamin D deficiency
20%
Headache
20%
Cataract subcapsular
20%
Neuroma
20%
Upper respiratory tract infection
20%
Vomiting
20%
Oral herpes
20%
Cataract nuclear
20%
Dental restoration failure
20%
Pyrexia
20%
Abscess limb
20%
Visual acuity reduced
20%
Visual impairment
20%
Ear pain
20%
Eye irritation
20%
Gingival pain
20%
Synovial cyst
20%
Ocular hyperaemia
20%
Eye pain
20%
Optic nerve sheath haemorrhage
20%
Ligament rupture
20%
Chest pain
20%
Bronchitis
20%
Ophthalmic migraine
20%
Obsessive-compulsive disorder
20%
Intraocular pressure increased
20%
Syncope
20%
Dermatitis contact
100%
80%
60%
40%
20%
0%
Study treatment Arm
Cohort 2: AAV2-hCHM Dose 2
Cohort 1: AAV2-hCHM Dose 1
Cohort 3 (Expansion Cohort): AAV2-hCHM Dose 2

Trial Design

3Treatment groups
Experimental Treatment
Group I: Cohort 3 (Expansion Cohort): AAV2-hCHM Dose 2Experimental Treatment1 Intervention
Single, unilateral subretinal administration of a single high dose range of AAV2-hCHM.
Group II: Cohort 2: AAV2-hCHM Dose 2Experimental Treatment1 Intervention
Single, unilateral subretinal administration of a single high dose range of AAV2-hCHM.
Group III: Cohort 1: AAV2-hCHM Dose 1Experimental Treatment1 Intervention
Single, unilateral subretinal administration of a single low dose range of AAV2-hCHM.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AAV2-hCHM
2015
Completed Phase 2
~20

Find a Location

Who is running the clinical trial?

Spark Therapeutics, Inc.Lead Sponsor
14 Previous Clinical Trials
424 Total Patients Enrolled
Spark TherapeuticsLead Sponsor
13 Previous Clinical Trials
339 Total Patients Enrolled
Clinical DirectorStudy DirectorSpark Therapeutics
17 Previous Clinical Trials
4,480 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations
2015. "Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02341807.
~1 spots leftby Apr 2025
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