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

Rheumatic Fever Clinical Trial

— TREAT-SC  

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06259006
• Other study ID #: StarshipChild
• Status: Not yet recruiting
• Phase: Phase 3
• Start date: May 2024
• Est. completion date: May 2028

Study information

• Verified date: April 2024
• Source: Starship Child Health, Te Toka Tumai Auckland
• Contact: Hannah F Jones, MBChB PhD
• Phone: +64 9 307 4949
• Email: hannahj1[@]adhb.govt.nz
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 80
• Est. completion date: May 2028
• Est. primary completion date: May 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 17 Years

Eligibility

Inclusion Criteria:

1. Sydenham's chorea of any severity diagnosed by a paediatrician or neurologist based on national ARF guidelines

2. Child or adolescents aged 4 years to <18 years of age

Exclusion Criteria:

1. Administered steroids or intravenous immunoglobulin since onset of the current Sydenham's chorea episode

2. Evidence of concomitant severe, acute infection

3. History of hypersensitivity to dexamethasone or its excipients

4. Pregnancy

5. Confirmed exposure of an unimmunised child to measles, mumps, rubella or chickenpox within the previous four weeks

6. Receipt of a live vaccine within the previous four weeks

7. Medical condition or treatment with medication which in the opinion of the trial investigators would make the child unsuitable for the trial

Related Conditions & MeSH terms

• Chorea
• Rheumatic Fever
• Sydenham Chorea

Intervention

Drug:
• Dexamethasone Oral
4mg capsules taken orally in three divided doses to a total daily dose of 20mg/m2 (maximum dose 24mg/day) for three days
• Placebo
Matching capsules taken orally three times daily for three days

Locations

Country	Name	City	State
Australia	Royal Darwin Hospital	Darwin	Northern Territories
New Zealand	Starship Child Health, Te Toka Tumai Auckland, Te Whatu Ora Health New Zealand	Auckland

Sponsors (3)

Lead Sponsor	Collaborator
Starship Child Health, Te Toka Tumai Auckland	Health Research Council, New Zealand, Menzies School of Health Research

Countries where clinical trial is conducted

Australia,  New Zealand, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Chorea severity at 1 month	Chorea severity measured using the Universidade Federal de Minas Gerais Sydenham's Chorea Rating Scale. Scores range from 0 to 108. A higher score correlates with a worse outcome.	1 month
Secondary	Adverse events	Frequency of adverse events	3 days, 7 days, 1 month
Secondary	Chorea severity	Chorea severity measured using the Universidade Federal de Minas Gerais Sydenham's Chorea Rating Scale. Scores range from 0 to 108. A higher score correlates with a worse outcome.	7 days, 3 months, 12 months
Secondary	Total hospital length of stay	Total hospital length of stay for chorea at three months	3 months
Secondary	Treatment failure at 14 days	Number of participants with treatment failure at 14 days	14 days
Secondary	Use of adjunctive chorea treatments	Number of participants treated with adjunctive chorea treatments	1 month
Secondary	Chorea relapse and recurrence	Number of participants with chorea relapse and recurrence at 3 and 12 months	3 and 12 months
Secondary	Universidade Federal de Minas Gerais Sydenham's Chorea Rating Scale Subscores.	Behaviour (0 to 24 points), Activities of Daily Living (0 to 28 points) and Motor Assessment (0-56 points). A higher score correlates with a worse outcome.	7 days, 1 month, 3 months, 12 months
Secondary	Psychiatric symptoms	Strength and Difficulties Questionnaire Total Difficulties Score. The resultant score ranges from 0 to 40. An abnormally high total difficulties score correlates with worse outcome.	1 month, 3 months and 12 months