Symmetric Lipomatoses, Multiple Clinical Trial
— N-of-1 SLNOfficial title:
Hydrocortisone Treatment for Symmetric Lipomatosis Associated With Neuropathy (SLN): an N-of-1 Study
A young women with Symmetric Lipomatosis associated with Neuropathy (SLN) was seen at the department of Internal Medicine - Endocrinology and at the department of Neurology at the Erasmus MC, Rotterdam, the Netherlands. The patient presented with balance problems due to neuropathy and prominent cervical and genital lipomas. In the past, the patient had been treated with steroids for a short period. The patient noticed that, as a 'side effect', during this treatment period the lipomas shrank and that the balance problems nearly disappeared. The complaints reappeared after withdrawal of the steroids. In the current study, the usefulness of steroid treatment in this single patient will be investigated in an N-of-1 trial. Primary objective: to determine the efficacy of hydrocortisone treatment for neuropathy on an individual level in a patient with SLN as assessed with the Rasch-built Overall Disability Scale (RODS).
| Status | Not yet recruiting |
| Enrollment | 1 |
| Est. completion date | September 1, 2022 |
| Est. primary completion date | September 1, 2022 |
| Accepts healthy volunteers | No |
| Gender | Female |
| Age group | 31 Years and older |
| Eligibility | The study is especially designed for a specific female patient. The patient is 31 years old at the moment. Since the study is especially designed for this patient, there are no formal inclusion and exclusion criteria. However, the study will be stopped when the patient gets pregnant as this can influence the primary and secondary outcome measures. Inclusion Criteria: - N/A Exclusion Criteria: - Pregnancy |
| Country | Name | City | State |
|---|---|---|---|
| Netherlands | Erasmus MC | Rotterdam | Zuid-Holland |
| Lead Sponsor | Collaborator |
|---|---|
| Erasmus Medical Center |
Netherlands,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Rasch-built Overall Disability Scale (RODS) | The RODS is a validated 24-items questionnaire to measure the activity and social participation limitations in patients with neuropathy. The questionnaire has a score range of 0 to 100. A higher score means a better outcome (less limitations of the neuropathy) | Baseline to 414 days | |
| Secondary | Size of lipoma | The size of the lipoma will be measured with a centimeter by the researcher during hospital visits. The researcher will also take a picture of the lipoma in front of graph paper in a standardized manner. In between hospital visits, the participant will take a picture of the lipoma in front of graph paper. | Baseline to 414 days | |
| Secondary | Medical Research Council (MRC) sumscore | The MRC sumscore is a validated tool to measure muscle strength and must be carried out by a physician, nurse, physiotherapist or other professional. The score has a range of 1 to 5. A higher score means a better outcome. | Baseline to 414 days | |
| Secondary | Goal Attainment Scaling (GAS) | GAS is an individualized outcome measure in which several personal goals and the corresponding scaling are defined in consultation with the patient. The scaling is standardized, which makes it possible to reliably measure the change in the situation of the patient. The levels range from -3 to +2. A higher level means a better outcome. The predefined personal goals are a measure of the effectivity of the treatment. If possible, three goals will be set. | Baseline to 414 days |