TREAT-SC: Early, Short Course Oral Dexamethasone for the Treatment of Sydenham Chorea in Children

Official title

TREAT-SC: A Randomised, Double-Blinded, Placebo-Controlled Trial of Early, Short Course Oral Dexamethasone for the Treatment of Sydenham Chorea in Children

Overview

The purpose of this study is to find out whether an early three-day course of an oral steroid medication (dexamethasone) can improve the physical and mental recovery and wellbeing for children with Sydenham's chorea. Sydenham's chorea is a condition that impacts approximately 12% of children with acute rheumatic fever. It is caused by inflammation in the brain following an abnormal immune response to Group A streptococcus bacterial infection. Sydenham's chorea is a movement disorder that causes children's faces, hands, and feet to move quickly and uncontrollably, and can also affect mood and concentration. The physical recovery from Sydenham's chorea can take two to six months but the mental recovery (e.g. mood and concentration) can take longer to resolve. Sydenham's chorea remains endemic in Māori, Pacific Islander, Aboriginal and Torres Strait Islander children in New Zealand and Australia. There is limited evidence to direct treatment of Sydenham's chorea, and clinical practice differs widely around the world. Dexamethasone is an oral steroid which targets the abnormal immune response and successfully treats other immune-mediated brain disorders, with good tolerability. TREAT-SC is a randomized, double-blinded, placebo-controlled trial which will investigate whether a three day course of oral dexamethasone safely and effectively treats the movement disorder and psychiatric symptoms of Sydenham's chorea. The trial will recruit 80 participants from study sites in Australia and New Zealand.

Detailed description

Aim: The aim of the trial is to determine whether an early, short course of oral dexamethasone is a safe and effective treatment to reduce morbidity associated with Sydenham's chorea in New Zealand and Australian children.

Objectives:

* The primary objective is to determine whether oral dexamethasone is an effective treatment to reduce Sydenham's chorea severity in New Zealand and Australian children at one month * The secondary objectives are to determine whether oral dexamethasone is an effective treatment to reduce Sydenham's chorea severity at different time points, relapse and recurrence rates at three and 12 months, total hospital length of stay at three months, treatment failure at 14 days, use of adjunctive chorea treatments, Universidade Federal de Minas Gerais (UFMG) Sydenham's Chorea Rating Scale (USCRS) subscores, and psychiatric symptoms scored by the Strengths and Difficulties Questionnaire at one, three and 12 months. The safety of oral dexamethasone use will be assessed at study visits on days three and seven and at one month to monitor for any adverse events which may relate to dexamethasone use.

Research Design: TREAT-SC is a pragmatic parallel-group, double-blinded, placebo-controlled, 1:1 randomized efficacy trial comparing a three-day course of oral dexamethasone with placebo to treat Sydenham's chorea in New Zealand and Australian children. TREAT-SC will be a multi-site trial with participants recruited from hospitals in New Zealand and Australia.

Interventions

• Drug Dexamethasone Oral
  Dexamethasone suspension (1mg/ml) 20mg/m2/day (maximum dose 24mg/day) given in three divided doses
• Drug Placebo
  Matching capsules taken orally three times daily for three days

Primary outcome measures

• Chorea severity at 1 month [Time frame: 1 month]

Secondary outcome measures (7)

• Chorea severity [Time frame: 7 days, 3 months, 12 months]
• Total hospital length of stay [Time frame: 3 months]
• Treatment failure at 14 days [Time frame: 14 days]
• Use of adjunctive chorea treatments [Time frame: 1 month]
• Chorea relapse and recurrence [Time frame: 3 and 12 months]
• Universidade Federal de Minas Gerais Sydenham's Chorea Rating Scale Subscores. [Time frame: 7 days, 1 month, 3 months, 12 months]
• Psychiatric symptoms [Time frame: 1 month, 3 months and 12 months]

Eligibility criteria

Inclusion criteria

• Sydenham's chorea of any severity diagnosed by a paediatrician or neurologist based on national ARF guidelines
• Child or adolescents aged 4 years to <18 years of age

Exclusion criteria

• Administered steroids or intravenous immunoglobulin since onset of the current Sydenham's chorea episode
• Evidence of concomitant severe, acute infection
• History of hypersensitivity to dexamethasone or its excipients
• Pregnancy
• Confirmed exposure of an unimmunised child to measles, mumps, rubella or chickenpox within the previous four weeks
• Receipt of a live vaccine within the previous four weeks
• Medical condition or treatment with medication which in the opinion of the trial investigators would make the child unsuitable for the trial

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Quadruple blind

Primary purpose

Treatment

Study locations

New Zealand · 16 centers

• KidzFirst Hospital — Auckland
• Starship Child Health, Te Toka Tumai, Auckland — Auckland
• Waitākere Hospital — Auckland
• Gisborne Hospital — Gisborne
• Waikato Hospital — Hamilton
• Hawkes Bay Fallen Soldiers' Memorial Hospital — Hastings
• Hutt Hospital — Lower Hutt
• Wairarapa Hospital — Masterton
• … and 8 more centers

Australia · 1 center

• Royal Darwin Hospital — Darwin

Identifiers

NCT: NCT06259006 · StarshipChild

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗