MovDisordV3, Main, Exploration, bibRecord, 003551

CYTE‐I‐HD: Phase I dose finding and tolerability study of cysteamine (Cystagon) in Huntington's disease

Identifieur interne : 003551 ( Main/Exploration ); précédent : 003550; suivant : 003552

CYTE‐I‐HD: Phase I dose finding and tolerability study of cysteamine (Cystagon) in Huntington's disease

Auteurs : Richard Dubinsky [États-Unis] ; Carolyn Gray [États-Unis]

Source :

Movement Disorders [ 0885-3185 ] ; 2006-04.

RBID : ISTEX:DDFB9FF238EFD7F0B575C910D08765B81163D2F1

Descripteurs français

Pascal (Inist)
- Chorée Huntington, Essai clinique phase I, Mercaptamine, Protein-glutamine γ-glutamyltransferase, Système nerveux pathologie.

English descriptors

KwdEn :
- Adult, Cysteamine (therapeutic use), Drug Administration Schedule, Drug Tolerance, Female, Humans, Huntington Disease (drug therapy), Huntington disease, Huntington's disease, Male, Mercaptamine, Middle Aged, Nervous system diseases, Phase I trial, Protein-glutamine γ-glutamyltransferase, Radiation-Protective Agents (therapeutic use), Severity of Illness Index, cysteamine, transglutaminases inhibitors.
MESH :
- chemical , therapeutic use : Cysteamine, Radiation-Protective Agents.
- drug therapy : Huntington Disease.
- Adult, Drug Administration Schedule, Drug Tolerance, Female, Humans, Male, Middle Aged, Severity of Illness Index.

Abstract

Cystamine, an inhibitor of transglutaminases, slows progression of Huntington's disease in the murine model by approximately 20%. Cysteamine, the dimer of cystamine, is an orphan drug approved for the treatment of nephropathic cystinosis and has a similar benefit in the murine model but with a narrower therapeutic window. In a single‐center open‐label study, we determined the maximum tolerable dose (MTD) and side effects of cysteamine in people with Huntington's disease. Cysteamine was started at a dose of 10 mg/kg per day, divided into four doses, and increased by 10 mg/kg per day weekly until the development of intolerable side effects or a maximum dose of 70 mg/kg per day. Of the 9 subjects, 1 had an MTD of 10 mg/kg per day, 1 had an MTD of 20 mg/kg per day, the maximum dose was 30 mg/kg per day for 2, 40 mg/kg per day for 2, and 50 mg/kg per day for 3. Dose‐limiting side effects were motoric impairment in 5 and nausea in 4. The dose found tolerable by 8 of the subjects was 20 mg/kg per day. All had a noticeable hydrogen sulfide odor at doses of 40 mg/kg per day or higher. We conclude that, at a dose of 20 mg/kg per day, cysteamine was tolerable in people with Huntington's disease. Nausea and motoric impairment were the dose‐limiting side effects. © 2005 Movement Disorder Society

Url:

https://api.istex.fr/document/DDFB9FF238EFD7F0B575C910D08765B81163D2F1/fulltext/pdf

DOI: 10.1002/mds.20756

Affiliations:

Le document en format XML

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<front><div type="abstract" xml:lang="en">Cystamine, an inhibitor of transglutaminases, slows progression of Huntington's disease in the murine model by approximately 20%. Cysteamine, the dimer of cystamine, is an orphan drug approved for the treatment of nephropathic cystinosis and has a similar benefit in the murine model but with a narrower therapeutic window. In a single‐center open‐label study, we determined the maximum tolerable dose (MTD) and side effects of cysteamine in people with Huntington's disease. Cysteamine was started at a dose of 10 mg/kg per day, divided into four doses, and increased by 10 mg/kg per day weekly until the development of intolerable side effects or a maximum dose of 70 mg/kg per day. Of the 9 subjects, 1 had an MTD of 10 mg/kg per day, 1 had an MTD of 20 mg/kg per day, the maximum dose was 30 mg/kg per day for 2, 40 mg/kg per day for 2, and 50 mg/kg per day for 3. Dose‐limiting side effects were motoric impairment in 5 and nausea in 4. The dose found tolerable by 8 of the subjects was 20 mg/kg per day. All had a noticeable hydrogen sulfide odor at doses of 40 mg/kg per day or higher. We conclude that, at a dose of 20 mg/kg per day, cysteamine was tolerable in people with Huntington's disease. Nausea and motoric impairment were the dose‐limiting side effects. © 2005 Movement Disorder Society</div>
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Movement Disorders (revue)

CYTE‐I‐HD: Phase I dose finding and tolerability study of cysteamine (Cystagon) in Huntington's disease

CYTE‐I‐HD: Phase I dose finding and tolerability study of cysteamine (Cystagon) in Huntington's disease

Source :

Descripteurs français

English descriptors

Abstract

Links toward previous steps (curation, corpus...)

Le document en format XML

Pour manipuler ce document sous Unix (Dilib)

Pour mettre un lien sur cette page dans le réseau Wicri

	Movement Disorders (revue)
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