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Liver consequences of cysteine, glutathione and taurine deficiency in classical homocystinuria

Functional consequences and therapeutic intervention in hampered production of cysteine, glutathione and taurine in classical homocystinuria

Status
Recruiting
Phases
Unknown
Study type
Interventional
Source
REBEC
Registry ID
RBR-9kk7p2
Enrollment
Unknown
Registered
2019-09-16
Start date
2019-03-01
Completion date
Unknown
Last updated
2025-10-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Classical Homocystinuria (CBS deficiency)

Interventions

Patients (n=5) and controls (n=5) that accepted participate of the study will be oriented to attend at the study site after breakfast, being totally free of alcohol or tobacco in the last 48 hours. Th
Drug

Sponsors

Universidade Federal do Rio Grande do Sul
Lead Sponsor
Fundação de Amparo à Pesquisa do Estado do Rio Grande do Sul
Collaborator
Hospital de Clínicas de Porto Alegre
Collaborator

Eligibility

Age
18 Years to 60 Years

Inclusion criteria

Inclusion criteria: Age over 18 years; for patients: molecular diagnosis of CBS deficiency

Exclusion criteria

Exclusion criteria: Patients with gastric, hepatic or kidney disease; smoking; illicit drug users;Acetaminophen or N-acetylcysteine hypersensitivity.

Design outcomes

Primary

MeasureTime frame
Increase in at least 30% in hepatic transaminases after Acetaminophen administration. ;Prevention of increase in liver damage biomarkers after N-acetylcysteine administration.

Secondary

MeasureTime frame
Increase in at least 30% in oxidative stress markers.;Increase of red blood cells glutathione in at least 30%.

Countries

Brazil

Contacts

Public ContactIda;Soraia Doederlein Schwartz;Poloni

Universidade Federal do Rio Grande do Sul;Universidade Federal do Rio Grande do Sul

idadschwartz@gmail.com;soraiapoloni@yahoo.com.br+555133598011;+555133598849

Outcome results

None listed

Source: REBEC (via WHO ICTRP)