WHO Trial Registration Data Set
| Data category | Information |
|---|---|
| Primary registry and trial identifying number | EUCTR2014-001355-23-NL |
| Date of registration in primary registry | 27 March 2014 |
| Secondary identifiers | 40960 |
| Source(s) of monetary or material support | The Netherlands Organisation for Health Research and Development (ZonMW), grant number 1520020301 |
| Primary sponsor | Leiden University Medical Center |
| Secondary sponsor(s) | None |
| Contact for public queries | Ephedrine study contact Leiden University Medical Center Albinusdreef 2 2333 ZA Leiden The Netherlands Tel: (+31) 17 15 262 118 Email: myasthenie@lumc.nl |
| Contact for scientific queries | Ephedrine study contact Leiden University Medical Center Albinusdreef 2 2333 ZA Leiden The Netherlands Tel: (+31) 17 15 262 118 Email: myasthenie@lumc.nl |
| Public title | Ephedrine as add-on therapy for patients with MG |
| Scientific title | Ephedrine as add-on therapy for patients with MG |
| Country of recruitment | Netherlands |
| Condition studied | MG |
| Intervention(s) | Active comparator: Ephedrine hydrochloride. Placebo comparator |
| Key inclusion and exclusion criteria | Ages eligible for study: ≥18 years Sexes eligible for study: both Inclusion criteria: Adult patient with generalised MG for whom treatment with pyridostigmine, either alone or in combination with low-dose prednisone or other immunosuppressive drugs, does not adequately improve symptoms Exclusion criteria: purely ocular myasthenia, ephedrine is contraindicated or was not tolerated in the past, reliance on medium-to-high dose prednisone, intravenous immunoglobulin, or plasma exchange therapy, myasthenic crisis in past 3 months, thymectomy in past 6 months or scheduled, inability to comply with study procedures or give informed consent |
| Study type | Interventional Controlled: yes, placebo Allocation: randomised Intervention model: multiple crossover assignment Masking: double blind Followed by optional open-label extension phase |
| Date of first enrolment | 7 October 2014 |
| Target sample size | 4 |
| Recruitment status | Complete as of 29 December 2014 |
| Primary outcome(s) | Add-on effect of ephedrine on muscle strength/endurance as measured by the QMG score for all patients enrolled |
| Key secondary outcomes | Add-on effect of ephedrine for individual patients; feasibility of a larger series of n-of-1 trials, including patients’ and physicians’ experiences with the current trial; changes in secondary outcomes measures including the MG-Composite and MG-ADL; long-term effects of ephedrine (open-label extension study only); adverse effects of ephedrine |
MG, myasthenia gravis; MG-ADL, MG-Activities of Daily Living; QMG, Quantitative Myasthenia Gravis.