Description Module
The Description Module contains narrative descriptions of the clinical trial, including a brief summary and detailed description. These descriptions provide important information about the study's purpose, methodology, and key details in language accessible to both researchers and the general public.
Description Module path is as follows:
Study -> Protocol Section -> Description Module
Description Module
Ignite Creation Date:
2025-12-24 @ 2:05 PM
Ignite Modification Date:
2025-12-24 @ 2:05 PM
NCT ID:
NCT01096095
Brief Summary:
DESIGN: Pilot, Phase II, double-blind, placebo-controlled study
JUSTIFICATION: In the literature one does not find a pharmacological treatment that changes the natural history of Spinocerebellar ataxtia type 3 (SCA3). Patients with this disease invariably become dependent.
OBJECTIVES I. To determine safety and tolerability of phenylbutyrate in patients with SCA3.
II. To provide early subsidies on the efficacy of phenylbutyrate in SCA3.
DURATION: 12 months of a double-blind study.
PLACE OF REALIZATION: Hospital de Clínicas de Porto Alegre, Brazil.
NUMBER OF PATIENTS: 20 patients.
CONCOMITANT MEDICATIONS: There are no concomitant medications that are prohibited unless they affect safety parameters of this study (hemogram and platelets; fasting serum glucose, AST, ALT, Gamma-GT, Bilirubins, Prothrombin time, Creatinine, Urea, Na, K, chlorides and arterial gasometry; electrocardiogram and echocardiogram).
MEDICATIONS UNDER INVESTIGATION: Powdered sodium phenylbutyrate in sachets containing each 3g. At the start of the study, the dose will be 15g/day (five sachets) and may be reduced in case of mild adverse events.
OUTCOMES Primary safety outcome: The number of adverse events, interruptions and dose reductions in the two groups (cases and controls).
Efficacy outcomes: Efficacy outcomes are the following scores in both groups: NESSCA, SARA, Barthel, BDI, and WHOQol.
Study:
NCT01096095
Study Brief:
Protocol Section:
NCT01096095