Trehalose 100g powder form to be dissolved in water, to be consumed by mouth, every day for 3 months.

Malthose 30g powder form to be dissolved in water, to be consumed by mouth, every day for 3 months.

Spinocerebellar Ataxia Type 3

A disaccharide sugar usually used as food preservative, but for this study it is given at a higher dose

Trehalose

Maltose

UKM Medical Centre

Efficacy Of Oral Trehalose In Spinocerebellar Ataxia 3

A Randomised Controlled Trial on the Clinical Efficacy of Oral Trehalose in Patients With Spinocerebellar Ataxia 3: Clinical & FMRI Correlation

An 8-item scale to quantify the severity of ataxia with a scoring of 0 (no ataxia) to 40 (most severe ataxia). Total time taken for test administration is estimated at 10 minutes.

A validated multi-modal assessment tool that is composed of: a) timed 8 metre walk (8MW); b) the 9-hole peg test (9HPT); and c) the rate of "PATA" repetition over 10 seconds (PATA) to rate speech performance. Total time taken for test administration is estimated at 10 minutes.

A validated scale assessing non-cerebellar signs with a scoring of 0 (no non-ataxia sign) to 16 (all assessed systems affected). Total time taken for test administration is estimated at 10 minutes.

CCAS Scale is a brief cognitive screening tool to help identify CCAS in patients with cerebellar impairment. It derives a total score of 120 as a continuous measure; as well as an ordinal measure in accordance to the number of failed tests: 1) Possible CCAS = 1 failed test; 2) Probable CCAS = 2 failed tests; Definite CCAS = 3 or more failed tests. Four different forms were available to minimize the practice effect. Total time taken for test administration is estimated at 10 minutes.

WAIS - 4 is an IQ test designed to measure intelligence and cognitive ability in adults and older adolescents. The selected subtests are Matrix Reasoning, Digit Span, and Coding. These subtests are used to measure abstract reasoning, WM, and processing speed respectively. These index and subtests have good reliability

D - KEFS is a neuropsychological battery designed to measure various subdomains of executive function from 8 - 89 years old. The selected subtests are: Tower Test, Trail Making Test (TMT), Colour-Word Interference Test (CWIT). These tests measure planning, set-shifting, and inhibition abilities respectively.

RBANS Update is a neuropsychological battery designed to measure 5 neuropsychological domains from 12:0 - 89:11 years old. The selected subtests are: Figure Copy and Figure Recall. These tests measure visual construction, visual memory, and language abilities respectively.

The level of measurement of oxy-Hb (activation level) is continuous; greater oxy-Hb (mM.mm) indicates greater activation.

The level of measurement of N-Acetyl Aspartate (NAA; metabolism) is continuous; greater NAA (ppm) indicates greater metabolism.

The level of measurement of grey matter density (structural) is continuous; greater grey matter density indicates greater structural volume.

SIMS comprises 16 items on four subscales, Intrinsic motivation (e.g., "I think that this activity is interesting"), Identified regulation (e.g., "I am doing it for my own good"), External regulation (e.g., "I am supposed to do it") and Amotivation (e.g., "I don't see what this activity brings me"). It contains 4 items per subscale scored on a scale from 1 to 7 providing a score between 4 and 28 for each subscale. It has an internal reliability of α = .74 - .83 for the four subscales. Total time taken for test administration is estimated at 5 minutes.

EQ-5D-3L consists of 2 parts: the EQ-5D descriptive system and the EQ visual analogue scale (EQ VAS). EQ-5D has 5 dimensions of assessment (mobility, self-care, usual activities, pain/discomfort and anxiety/depression). Each dimension has 3 levels (ordinal): no problem, some problem, and extreme problem. It has an internal reliability of α = .83 and valid among clinically and demographically heterogeneous patients. Total time taken for test administration is estimated at 5 minutes.

Adverse event will be recorded in terms of severity, study intervention relationship, action taken regarding study intervention, outcome of adverse event, expected, and serious adverse event.

Dr Norlinah Mohamed Ibrahim

Professor

National University of Malaysia

Radboud University

This study will employ a double-blind randomised controlled trial (RCT) with two arms. Patients with SCA3 will be randomly allocated to oral trehalose group and a maltose placebo group. A total of 20 participants will be allocated to each group. There is a possibility of including other types of SCA at later stage of the study.

Patients with SCA 3 will be randomised into the active treatment group and placebo group to which they are blinded from their group allocation. Likewise, the outcome assessor will be blinded from the patient's group status.

Ranibizumab patients will receive three monthly Ranibizumab 0.5 mg/0.05 mL injections, followed by PRN dosing, as treatment for acute pseudophakic cystoid macular edema.

Triamcinolone acetonide patients will receive PRN Triamcinolone acetonide 4 mg/0.1 mL injections, every 3 months, as treatment for acute pseudophakic cystoid macular edema.

CYSTOID MACULAR EDEMA

Intravitreal injection of Ranibizumab as treatment for acute pseudophakic cystoid macular edema. Ranibizumab patients will receive three monthly Ranibizumab 0.5 mg/0.05 mL injections, followed by PRN dosing, as treatment for acute pseudophakic cystoid macular edema.

Ranibizumab,

Intravitreal injection of Triamcinolone acetonide as treatment for acute pseudophakic cystoid macular edema. Triamcinolone acetonide patients will receive PRN Triamcinolone acetonide 4 mg/0.1 mL injections, every 3 months, as treatment for acute pseudophakic cystoid macular edema.

Triamcinolone acetonide

Soll Eye

Acute Pseudophakic Cystoid Macular Edema Treatment Trial: Intravitreal Ranibizumab Versus Triamcinolone Acetonide

To assess the safety of intravitreal R and T for the treatment of acute pseudophakic cystoid macular edema by evaluating:
Incidence and severity of ocular adverse events, as identified by eye examination
Incidence and severity of other adverse events, as identified by physical examination, subject reporting, and changes in vital signs

Mean change in spectral domain OCT CMT from baseline (microns)

Mean change in ETDRS letters from baseline (letters)

Proportion of patients with 3 line or better ETDRS letter improvement (%)

Time to 3 line ETDRS letter improvement (days)

trehalose/атрофия

Efficacy Of Oral Trehalose In Spinocerebellar Ataxia 3

Acute Pseudophakic Cystoid Macular Edema Treatment Trial: Intravitreal Ranibizumab Versus Triamcinolone Acetonide

Самая полная база данных о лекарственных травах, подтвержденная наукой