Antithyroid Drug Treatment of Thyrotoxicosis in Young People

NCT ID: NCT01436994

Last Updated: 2016-09-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

81 participants

Study Classification

INTERVENTIONAL

Study Start Date

2004-07-31

Study Completion Date

2015-11-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The investigators aim to establish whether biochemical control during anti-thyroid drug therapy in young people with thyrotoxicosis varies depending upon whether a 'block and replace' or 'dose titration' regimen is used. The investigators will also assess remission rates and the frequency of side-effects in the two treatment groups.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Thyrotoxicosis is an uncommon disorder in childhood and adolescence with a UK incidence around 1 per 100,000 (0-15 years). Most patients with thyrotoxicosis have Graves' disease which develops because of thyrotropin (TSH) receptor stimulation by autoantibodies. Patients with Hashimoto's thyroiditis can also be thyrotoxic in the early phase of the disease and occasionally thyrotoxicosis develops because of activating mutations of the TSH receptor. Many general paediatricians have experience of managing patients with thyrotoxicosis but national guidelines to assist in patient care have not been produced to date.

There is no ideal therapy for thyrotoxicosis in children and adolescents. The three treatment modalities for thyrotoxicosis - anti-thyroid drugs (ATD), surgery and radioiodine all have significant disadvantages. Particular considerations when managing young people include:

1. Low remission rates following a course of ATD.
2. Concerns about the morbidity associated with thyroidectomy.
3. Inadequate data regarding the long term safety of radioiodine.

Children and adolescents presenting with autoimmune thyrotoxicosis in the UK are usually treated with ATD from diagnosis for 1 - 4 years. Treatment is then stopped and patients who relapse return to ATD or are offered more definitive treatment with surgery or radioiodine. Life-long thyroid hormone replacement will be required if the thyroid gland is removed by surgery or ablated by radioiodine.

Excess thyroid hormone can have a major detrimental impact on cognitive function as well as cardiovascular and skeletal health. The maintenance of a clinically and biochemically euthyroid state is therefore highly desirable. There are two possible approaches when treating patients with ATD.

* 'Block and replace' (combined) therapy - where thyroid hormone production is prevented by ATD and thyroxine is then added in a replacement dose.
* 'Dose titration' (adaptive) therapy - where the dose of ATD is adjusted so that hormone production is normalised.

Both strategies are used by adult endocrinologists but it is unclear which of these approaches is the most appropriate in the young person.

Potential advantages of the 'block and replace' regimen include:

* Improved stability with fewer episodes of hyper or hypothyroidism.
* A reduced number of venepunctures and visits to hospital.
* Improved remission rates following a larger anti-thyroid drug dose.

Potential advantages of the dose titration approach include:

* Fewer side effects with a lower anti-thyroid drug dose
* Improved compliance on one rather than two medications. A meta-analysis conducted primarily in adult patients concluded that 'dose titration' was the most appropriate way to manage thyrotoxicosis because of fewer ATD-related side-effects although a group of authors subsequently highlighted significant limitations of this study.

This study is a prospective, multi-centre trial which aims to establish which regimen - block and replace or dose titration - is the most appropriate medical therapy for thyrotoxicosis during childhood and adolescence.

* Primary completion date changed from January 2019 to November 2014
* Study completion date changed from January 2019 to November 2015

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Paediatric Thyrotoxicosis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Block and Replace

Carbimazole is commenced in a dose of 0.75 mg/kg/day. The intention is to completely prevent endogenous thyroxine production. Thyroxine is then added in a replacement dose as the thyroid hormone levels fall into the lower half of the laboratory normal range. The principal measure of control during the first 6 months will be thyroid hormone levels rather than TSH.

Group Type ACTIVE_COMPARATOR

Block and Replace

Intervention Type PROCEDURE

The primary objective of treatment is to maintain a euthyroid state with TSH and thyroid hormone levels in the local laboratory normal range. Carbimazole is commenced in a dose of 0.75 mg/kg/day (propylthiouracil - for dose see below) with the aim being to completely preventing endogenous thyroxine production. Thyroxine is then added in a low replacement dose as the thyroid hormone levels fall into the lower half of the laboratory normal range. The principle measure of control during the first 6 months will be thyroid hormone levels rather than TSH. Carbimazole is the preferred treatment because of the increased risk of hepatotoxicity with propylthiouracil but patients who are treated with propylthiouracil can also be recruited and randomised. 1mg of carbimazole is approximately equivalent to 10 mg of propylthiouracil.

carbimazole

Intervention Type DRUG

Carbimazole 5mg and 20 mg tablets Administered as a once or twice daily regimen with total daily dose adjusted according to prevailing biochemistry

propylthiouracil

Intervention Type DRUG

50 mg tablets administered once daily with the dose adjusted according to the prevailing biochemistry

thyroxine

Intervention Type DRUG

25mcg, 50mcg and 100mcg tabletes administered once daily with the dose adjusted according to the prevailing biochemistry

Dose Titration

Carbimazole is commenced in a dose of 0.75 mg/kg/day until thyroid hormone levels fall into the local laboratory normal range. The dose is then reduced to 0.25 mg/kg/day with the intention of maintaining the euthyroid state. The principal measure of control during the first 6 months will be thyroid hormone levels rather than TSH.

Carbimazole is the preferred treatment because of the increased risk of hepatotoxicity with propylthiouracil but patients who are treated with propylthiouracil can also be recruited and randomised. 1mg of carbimazole is approximately equivalent to 10 mg of propylthiouracil.

Drug: Carbimazole 5mg and 20 mg tablets. Administered as a once or twice daily regimen with total daily dose adjusted according to prevailing biochemistry Drug: propylthiouracil 50 mg tablets administered once daily with the dose adjusted according to the prevailing biochemistry.

Group Type ACTIVE_COMPARATOR

Dose Titration

Intervention Type PROCEDURE

The primary objective of treatment is to maintain a euthyroid state with TSH and thyroid hormone levels in the local laboratory normal range.

Carbimazole is commenced in a dose of 0.75 mg/kg/day until thyroid hormone levels fall into the local laboratory normal range. The dose is then reduced to 0.25 mg/kg/day with the intention of maintaining a euthyroid state as reflected by a free thyroxine and TSH within the normal range.

Most paediatricians in the UK commence thyrotoxic children on carbimazole rather than propylthiouracil. Carbimazole is the preferred treatment because of the increased risk of hepatotoxicity with propylthiouracil but patients who are treated with propylthiouracil can also be recruited and randomised. The guidelines detailed above can be used in the knowledge that 1mg of carbimazole is approximately equivalent to 10 mg of propylthiouracil.

carbimazole

Intervention Type DRUG

Carbimazole 5mg and 20 mg tablets Administered as a once or twice daily regimen with total daily dose adjusted according to prevailing biochemistry

propylthiouracil

Intervention Type DRUG

50 mg tablets administered once daily with the dose adjusted according to the prevailing biochemistry

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Block and Replace

The primary objective of treatment is to maintain a euthyroid state with TSH and thyroid hormone levels in the local laboratory normal range. Carbimazole is commenced in a dose of 0.75 mg/kg/day (propylthiouracil - for dose see below) with the aim being to completely preventing endogenous thyroxine production. Thyroxine is then added in a low replacement dose as the thyroid hormone levels fall into the lower half of the laboratory normal range. The principle measure of control during the first 6 months will be thyroid hormone levels rather than TSH. Carbimazole is the preferred treatment because of the increased risk of hepatotoxicity with propylthiouracil but patients who are treated with propylthiouracil can also be recruited and randomised. 1mg of carbimazole is approximately equivalent to 10 mg of propylthiouracil.

Intervention Type PROCEDURE

Dose Titration

The primary objective of treatment is to maintain a euthyroid state with TSH and thyroid hormone levels in the local laboratory normal range.

Carbimazole is commenced in a dose of 0.75 mg/kg/day until thyroid hormone levels fall into the local laboratory normal range. The dose is then reduced to 0.25 mg/kg/day with the intention of maintaining a euthyroid state as reflected by a free thyroxine and TSH within the normal range.

Most paediatricians in the UK commence thyrotoxic children on carbimazole rather than propylthiouracil. Carbimazole is the preferred treatment because of the increased risk of hepatotoxicity with propylthiouracil but patients who are treated with propylthiouracil can also be recruited and randomised. The guidelines detailed above can be used in the knowledge that 1mg of carbimazole is approximately equivalent to 10 mg of propylthiouracil.

Intervention Type PROCEDURE

carbimazole

Carbimazole 5mg and 20 mg tablets Administered as a once or twice daily regimen with total daily dose adjusted according to prevailing biochemistry

Intervention Type DRUG

propylthiouracil

50 mg tablets administered once daily with the dose adjusted according to the prevailing biochemistry

Intervention Type DRUG

thyroxine

25mcg, 50mcg and 100mcg tabletes administered once daily with the dose adjusted according to the prevailing biochemistry

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Carbimazole propylthiouracil thyroxine carbimazole propylthiouracil

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. All patients with thyrotoxicosis aged between 2 and 16 years at the time of diagnosis. Thyrotoxicosis will be diagnosed by the paediatrician on the basis of the clinical picture and the biochemistry (suppressed TSH with high thyroid hormone levels).
2. Child has consented/assented or consent via parent/guardian has been gained prior to any study specific procedures

Exclusion Criteria

1. Known toxic adenoma / toxic hyperplasia (germline activating TSHR mutation).
2. McCune Albright Syndrome.
3. Previous episodes of Thyrotoxicosis..
4. Known allergic response to any of the study medication or ingredients as per SmPC.
5. Previous participation in this study.
Minimum Eligible Age

2 Years

Maximum Eligible Age

16 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Newcastle-upon-Tyne Hospitals NHS Trust

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Tim Cheetham

Dr Tim Cheetham

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Tim Cheetham

Role: PRINCIPAL_INVESTIGATOR

Newcastle upon Tyne Hospiatls NHS Foundation Trust

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Royal Aberdeen Children's Hospital

Aberdeen, , United Kingdom

Site Status

Birmingham Children's Hospital

Birmingham, , United Kingdom

Site Status

Addebrookes Hospital

Cambridge, , United Kingdom

Site Status

Wales College of Medicine

Cardiff, , United Kingdom

Site Status

University Hospital

Coventry, , United Kingdom

Site Status

Ninewells Hospital

Dundee, , United Kingdom

Site Status

Royal Hospital for Sick Children

Edinburgh, , United Kingdom

Site Status

Royal Hospital for Sick Children

Glasgow, , United Kingdom

Site Status

Hereford Hospital

Hereford, , United Kingdom

Site Status

Crosshouse Hospital

Kilmarnock, , United Kingdom

Site Status

Alder Hey Children's Hospital

Liverpool, , United Kingdom

Site Status

St Bart's Hospital

London, , United Kingdom

Site Status

St George's Hospital

London, , United Kingdom

Site Status

Royal Manchester Children's Hospital

Manchester, , United Kingdom

Site Status

Royal Victoria Infirmary

Newcastle upon Tyne, , United Kingdom

Site Status

Norfolk & Norwich University Hospitals

Norwich, , United Kingdom

Site Status

Oxford Radcliffe Hospitals

Oxford, , United Kingdom

Site Status

Sheffield Children's Hospital

Sheffield, , United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United Kingdom

Related Links

Access external resources that provide additional context or updates about the study.

http://www.bsped.org.uk/

British Society for Paediatric Endocrinology and Diabetes supports this study

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

NUTH 2759

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Prospective Evaluation of OptiThyDose
NCT06864351 RECRUITING NA
Thyroxine Replacement in Organ Donors
NCT00238030 COMPLETED PHASE1/PHASE2
Thyroid Hormone for Thyroid Cancer
NCT06647602 NOT_YET_RECRUITING
Study of NTBC for Tyrosinemia I
NCT00004443 COMPLETED NA