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NCT06622577

The Effect of Dietary Management and Cysteine Supplementation on Growth Parameters and Biochemical Control for Pediatric Qatari Patients Affected with Classical B6 Non-responsive Homocystinuria.

Not yet recruiting NA Last updated 2 October 2024
What this trial tests

NA trial testing Cysteine in Classical Homocystinuria in 50 participants. Not yet recruiting.

Timeline
1 December 2024
Primary endpoint
1 July 2025
31 August 2025

Quick facts

Lead sponsorHamad Medical Corporation
PhaseNA
StatusNot yet recruiting
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment50
Start date1 December 2024
Primary completion1 July 2025
Estimated completion31 August 2025
Sites1 location across Qatar

Drugs / interventions tested

Conditions studied

Sponsor

Hamad Medical Corporation — full company profile →

Who can join

Adults 1 Day to 18, any sex, with Classical Homocystinuria. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Classical homocystinuria (HCU) is an autosomal recessive disorder caused by the deficiency of an enzyme cystathionine β-synthase (CβS) that affects the catabolic pathway of the amino acid methionine (Met) which leads to an accumulation of high levels of methionine and Homocysteine causing complications in the multi system. Therefore, a strict dietary management is crucial to maintain good biochemical control, growth parameters and avoid complications. The main objective would be to analyze the impact of the Met restricted diet on growth parameters, biochemical markers and long-term complications in patients up to 18 years. In addition, the efficacy of dietary management with additional cysteine (Cys) supplementation for the patients up to 18 years would also be examined. The participants of the study would be recruited from the metabolic and genetics clinic in Hamad General Hospital (HGH), Qatar. All Qatari participants with confirmed diagnosis of HCU \<18 years of age will be included in the study. A mixed method study design would be used which include a cross sectional study design to assess the impact of methionine restricted diet on outcome variables and a single arm interventional study to analyze the effect of additional cysteine supplementation in patients from birth to 18 years. For the retrospective study, all the required data would be retrieved from electronic record from the Cerner of HMC and would be stored in a local drive with password protection. Further, all the eligible participants would be prospectively followed to supplement additional cysteine for the period of 6 months. The collected data will be statistically analyzed using the "SPSS windows version 22.0 software. The study would improve better understanding of dietary management through the identified outcomes. The outcome of Cys supplementation will improve the protein tolerance, biochemical parameters, growth parameters and may standardize the Cys supplementation.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.

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Other trials of Cysteine

Trials testing the same drug.

Other Hamad Medical Corporation trials

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Data sources for this page

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