The purpose of this 5-year trial is to determine if the surgical procedure, extended transsternal thymectomy (ETTX), combined with prednisone therapy is more beneficial in treating individuals with non-thymomatous MG than prednisone therapy alone.
ID
Source
Brief title
Condition
- Autoimmune disorders
- Muscle disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary objective is to determine in the non-thymomatous MG patient
population studied wether ETTX combined with prednisone therapy should be
preferred to prednisone therapy alone.
Secondary outcome
The secondary objectives are (i) to determine the efficacy of the therapies by
documenting their effects on myasthenic weakness and (ii) to determine their
safety by documenting total exposure to prednisone and by reecording the Trial
Specific Adverse Events (TSAEs) and Adverse Symptoms (TSASs). Importantly, we
shall also investigate wether outcomes vary between different subgroups by the
following planned sub-group analyses:
-use of corticosteroids vs. none prior to entering the study
-male versus female
-age < 40 years and age > 40 years at disease onset
Background summary
Myasthenia gravis (MG) is an autoimmune disease involving the thymus in which
85 percent of patients have antibodies to muscle acetylcholine receptors
(AChR-Ab) that interfere with neuromuscular transmission. MG frequently causes
severe disability that can be life-threatening. Thymectomy*a surgical procedure
that removes thymus gland tissue from the chest cavity*has been an established
therapy for non-thymomatous MG, or MG without thymoma, for more than 60 years
(based on retrospective, non-randomized studies). Corticosteroids are now being
used increasingly either as the sole treatment or in combination with
thymectomy. Both therapies have associated adverse effects and indications for
their use based on randomized trial data are lacking.
Study objective
The purpose of this 5-year trial is to determine if the surgical procedure,
extended transsternal thymectomy (ETTX), combined with prednisone therapy is
more beneficial in treating individuals with non-thymomatous MG than prednisone
therapy alone.
Study design
A Multi-Center, Single-Blind, Randomized Study Comparing Thymectomy to No
Thymectomy in Non-Thymomatous Myasthenia Gravis (MG) Patients Receiving
Prednisone
Intervention
Study participants will be randomized either to undergo the surgical procedure
ETTX and receive prednisone treatment, or to receive prednisone treatment alone
without surgery. Participants will be followed for at least 3 years.
Study burden and risks
There is no increased risk for the patients. Thymectomy and immunosuppressive
treatment are now routinely used in the treatment of myasthenia gravis.
P.O. Box 5801
Bethesda, MD 20824
USA
P.O. Box 5801
Bethesda, MD 20824
USA
Listed location countries
Age
Inclusion criteria
Male and female MG patients aged 18 to 60 years inclusive (thymectomy is not regularly performed in non-thymomatous MG patients > 60 years of age).
Onset of generalized MG within the last 3 years.
Positive serum anti-acetylcholine receptor binding antibodies (AChR Ab <=/> 1.0 nmol/L).
MGFA class II-IV at entry, using the MG Foundation of America (MGFA) classification, while receiving optimal anti-cholinesterase treatment with or without oral prednisone.
Exclusion criteria
Ocular MG without generalized weakness (MGFA Class I) or minimal weakness that would not require the use of corticosteroids.
Myasthenic weakness requiring intubation (MGFA class V) in the prior month.
Immunosuppressive therapy other than corticosteroids in the preceding year.
Medically unfit for thymectomy.
Chest computertomogreaphy evidence for thymoma.
Pregnancy or lactation; contraindications to the use of corticosteroids; unwillingness to practice effective contraception.
A serious concurrent medical, neurological or psychiatric condition.
Current daily dose of prednisone of more than 50 mg.
Participation in another experimental clinical trial.
History of alcohol or drug abuse.
Unwillingness or inability to comply with the requirements of the protocol.
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| ISRCTN | ISRCTN78813854enNCT00294658 |
| CCMO | NL15086.058.07 |