Prospective clinical trial to evaluate the efficacy of acetazolamide for the treatment of cystoid macular edema in inherited retinal dystrophies: the CAR trial

Phase 2

Recruiting

Conditions: 10047060
Inherited retinal dystrophies
Inhertited retinal diseases

Registration Number: NL-OMON51635

Lead Sponsor: Amsterdam UMC

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 18

Inclusion Criteria

Eligible patients received a clinical diagnosis of IRD, and underwent at least
one clinical examination, in combination with one of the following
prerequisites:
• IRD associated with causal genetic variant(s) (in e.g., USH2A, CRB1, RHO,
RP1, RP2, RPGR, PRPF31, or RS1 gene)
• CME involving the fovea confirmed on spectral-domain optical coherence
tomography (OCT)

Exclusion Criteria

A potential subject who meets any of the following criteria will be excluded
from participation in this study:
• Eyes will be excluded when the visual dysfunction is also significantly
associated with other ocular diseases besides the IRDs (e.g., glaucoma,
perforating trauma).
• Patients treated with loop diuretics
• Severe hepatic impairment
• Severe renal insufficiency
• Sodium and Potassium Depletion
• Addison's disease
• Hyperchloremic Acidosis
• Cor pulmonale
• Chronic non-congestive angle-closure glaucoma
• The use of Acetazolamide
• • Patients treated with interacting medication such as:
o Folic acid antagonists: methotrexate, trimethoprim and pyrimethamine
o Hypoglycaemics
o Oral anti-coagulants
o Asprin
o Cardiac glycosides: digoxine
o diuretics, such as thiazides and loop diuretics:
o anticonvulsants such as: phenytoin, primidone and carbamazepine
o Carbonic Anhydrase Inhibitors
o Procaine
o Sodium hydrogen carbonate
o Cyclosporine
o Ephedrine, methadone, amphetamine, quinidine and lithium

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The main endpoint is the to determine the effective dosage, the outcome of the<br /><br>treatment, and the effect on the visual acuity. As such, these findings will<br /><br>have immediate impact on translational scientific progress, by applying<br /><br>cutting-edge multidisciplinary technology to facilitate patient identification<br /><br>and selection for novel treatments. </p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Secondary study endpoints include the following compared within the treated<br /><br>group and compared to control group:<br /><br>1) To determine the optimal acetazolamide dose for maximum effect on CME and<br /><br>minimal side effects<br /><br>2) To determine the intra- and inter individual variability in such treatment-<br /><br>and side effects<br /><br>3) To determine the proportion of IRD patients with CME in which acetazolamide<br /><br>treatment is able to completely resolve CME for a spectrum of different<br /><br>IRD-associated genes</p><br>