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NCT04770415
Ophthalmic Manifestations of Patients With Mucopolysaccharidosis
trial in Ophthalmic Manifestations in 28 participants. Status unknown.
1 April 2023
Quick facts
| Lead sponsor | Shrouk Shaban Bakr |
|---|---|
| Status | Status unknown |
| Study type | OBSERVATIONAL |
| Enrollment | 28 |
| Start date | 1 April 2021 |
| Primary completion | 1 April 2023 |
| Estimated completion | 1 April 2023 |
| Sites | 1 location across Egypt |
Conditions studied
- Ophthalmic Manifestations — all drugs for Ophthalmic Manifestations →
Sponsor
Shrouk Shaban Bakr
Who can join
Under 18, any sex, with Ophthalmic Manifestations. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
Mucopolysaccharidosis (MPSs) are a group of disorders caused by inherited defects in lysosomal enzymes resulting in widespread intra- and extra-cellular accumulation of glycosaminoglycan(1,2). They have been subdivided according to enzyme defect and systemic manifestations and include MPS IH (Hurler)(3) , MPS IS (Scheie), MPS IH/S (Hurler/Sheie), MPS II(4,5) (Hunter), MPS III (Sanfilippo)(6) , MPS IV (Morquio)(7,8), MPS VI (Maroteaux-Lamy)(9), MPS VII (Sly)(10,11) and MPS IX (Natowicz)(12). Mucopolysaccharidosis have a spectrum of systemic manifestations, including airway and respiratory compromise, skeletal deformities, intellectual and neurological impairment, cardiac abnormalities, gastrointestinal problems and ocular manifestations(13). Ocular manifestation are common in the mucopolysaccharidosis and may result in significant visual impairment(14). Corneal opacification of varying severity is frequently seen, as well as retinopathy, optic nerve swelling and atrophy, ocular hypertension, and glaucoma(14). New treatment modalities for the systemic manifestations of the mucopolysaccharidosis include bone marrow transplant and enzyme replacement therapy have resulted in an improved prognosis in many cases(15).
Publications & conference data
No peer-reviewed publications indexed yet for this trial.
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Related trials
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT04770415 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Shrouk Shaban Bakr
- Last refreshed: 25 February 2021
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04770415.
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