Our study objective is to evaluate the efficacy of acetazolamide for the treatment of cystoid macula edema in inherited retinal dystrophies in anticipation of future clinical trials.
ID
Source
Brief title
Condition
- Retina, choroid and vitreous haemorrhages and vascular disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The main endpoint is the to determine the effective dosage, the outcome of the
treatment, and the effect on the visual acuity. As such, these findings will
have immediate impact on translational scientific progress, by applying
cutting-edge multidisciplinary technology to facilitate patient identification
and selection for novel treatments.
Secondary outcome
Secondary study endpoints include the following compared within the treated
group and compared to control group:
1) To determine the optimal acetazolamide dose for maximum effect on CME and
minimal side effects
2) To determine the intra- and inter individual variability in such treatment-
and side effects
3) To determine the proportion of IRD patients with CME in which acetazolamide
treatment is able to completely resolve CME for a spectrum of different
IRD-associated genes
Background summary
Inherited retinal dystrophies (IRDs) encompass a spectrum of severe eye
diseases, characterized by progressive loss of retinal structure and visual
function. Besides marked vision loss, IRDs may be complicated by cystoid
macular edema (CME) in the macula (the center of the retina, responsible for
detailed vision, color and contrast vision). The presence of CME has been
reported to have a possible negative effect on central vision and disease
progression. Moreover, IRD patients with CME may not be eligible for upcoming
innovative treatments such as gene therapy. Therefore, it is of the utmost
importance to try to effectively treat CME with existing potential treatment
options, not only to reduce the risk of vision loss, but also to increase
chances for patients to be eligible for future (gene) therapy.
Study objective
Our study objective is to evaluate the efficacy of acetazolamide for the
treatment of cystoid macula edema in inherited retinal dystrophies in
anticipation of future clinical trials.
Study design
Investigator-initiated, single-center, prospective, experimental study
consisting of seven visits at 2, 4, 8, 12, 16, and 32 weeks after baseline
evaluation visit. During each visit participants will perform several
ophthalmological measurements
Intervention
The used intervention in this study is acetazolamide, which belongs to a class
of drugs known as carbonic anhydrase inhibitors and has been used with other
medications to treat high pressure inside the eye due to certain types of
glaucoma.
Study burden and risks
Acetazolamide will be used during this study and there is possible risk of
uncommon (infrequent) to very rare adverse events as mentioned in the Summary
of Product Characteristics. Also, participants (especially young children and
elderly) may experience a minimal physical burden of visiting the hospital
seven times in 32 weeks. Additionally, patients may feel confronted with their
visual impairments.
Meibergdreef 9
Amsterdam 1105 AZ
NL
Meibergdreef 9
Amsterdam 1105 AZ
NL
Listed location countries
Age
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
Design
Recruitment
Medical products/devices used
Kamer G4-214
Postbus 22660
1100 DD Amsterdam
020 566 7389
mecamc@amsterdamumc.nl
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2002-000237-17-NL |
| CCMO | NL80249.018.22 |