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Viernes 2 oct 2026SEC · NASDAQ biomédico

Calendario›AMGN›RP103

RP103

Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis

Ensayo de AMGEN INC en Cystinosis (del registro, en inglés).

Fase
Fase 3
Estado
Completado
Participantes
41
previstos
Centros
14
Fin del objetivo primario
feb 2015
fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, de un solo grupo, abierto. Comenzó en ene 2013.

Qué mide

Average Difference Between Morning and Non-Morning Log White Blood Cell (WBC) Cystine Values (While taking Cystagon® (Months 1, 2, 3): within 15 minutes pre-morning (AM) and pre-non AM dose. During 3 months of RP103 (Months 5, 6, 7): 30 minutes post-AM and post-evening (PM) dose.) (del registro, en inglés)

Qué se prueba

Se compara una nueva forma de bitartrato de cisteamina llamada RP103 con el fármaco ya aprobado Cystagon en pacientes con cistinosis, midiendo la reducción de cistina y los efectos secundarios.

Lo que dice el documentogather information about the effectiveness (how well it works to treat cystinosis) and safety of a new form of cysteamine bitartrate called RP103, compared to the already-approved drug cystinosis patients are taking called Cystagon®.

Traducción automática (claude-haiku-4-5-20251001). Lo comprobado es la cita: está literal en el documento y sus cifras cuadran con las de la frase. Todavía no la ha leído una persona.

Cómo lo describe el promotor

En el documentoEn inglés, del registroThe purpose of this study is to gather information about the effectiveness (how well it works to treat cystinosis) and safety of a new form of cysteamine bitartrate called RP103, compared to the already-approved drug cystinosis patients are taking called Cystagon®. In cystinosis, the body builds up cystine. When taken regularly, the active ingredient of Cystagon® (cysteamine bitartrate) reduces cystine in the body. RP103 has the same active ingredient as Cystagon® and is designed to reduce cystine in a similar way that Cystagon® does. To decide if RP103 is better than Cystagon®, the study will look at two types of blood tests. One test is pharmacodynamics (PD), which measures the amount of white blood cell (WBC) cystine after taking study drug. WBC cystine is a laboratory test used to find out if cysteamine bitartrate is reducing cystine levels in the body. The second test is pharmacokinetics (PK), which measures the amount of cysteamine in the blood after taking the drug. RP103 is different from Cystagon®: Instead of the cysteamine bitartrate being absorbed from the stomach, RP103 is designed to be absorbed from the small intestine. This may make the effects of the drug last longer, so that it can be taken twice a day instead of four times a day like Cystagon®. Some cystinosis patients have bad breath (halitosis) when they take Cystagon®. Study participants who experience bad breath with Cystagon® will be asked if they would like to participate in an optional "halitosis substudy" to investigate this issue by collecting some extra PK blood samples.

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Ficha completa en ClinicalTrials.gov (NCT01733316), actualizada en dic 2024.