- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04821583
Hydrocortisone Treatment for Symmetric Lipomatosis Associated With Neuropathy (SLN): an N-of-1 Study Hydrocortisone Treatment for Symmetric Lipomatosis Associated With Neuropathy (SLN): an N-of-1 Study (N-of-1 SLN)
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).
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
A young women with Symmetric Lipomatosis associated with Neuropathy (SLN) was seen at the department of Internal Medicine-Endocrinology and the department of Neurology at the Erasmus MC, Rotterdam, the Netherlands. SLN is a rare disorder characterized by symmetric lipomas and neuropathy due to a homozygous mutation of c.2119C>T p.R707W in the MFN2 gene. Only seven cases of SLN are known in the literature. 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 (prednisolone) for a short period, because of a suspicion of adrenal insufficiency. The patient noticed that, as a 'side effect', during this treatment period the lipomas shrank and that the balance problems nearly disappeared. However, as a synacthen test showed that adrenal function was normal, steroid treatment was tapered and the complaints reappeared. In a follow-up visit at our outpatient clinic, the patient requested to be treated again with steroids for the complaints. As there is no literature on the effects of treatment with steroids on balance problems and on the size of lipomas in patients with SLN, the usefulness of steroid treatment in this single patient is investigated in an N-of-1 trial in order to improve the clinical care for this patient.
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).
Secondary objectives: to determine the effect of hydrocortisone treatment on the size of a lipoma in the neck, on the Medical Research Council (MRC) sum score and on specific personalized goals that are important to the patient and the patients' environment (Goal Attainment Scaling (GAS)).
Study Type
Enrollment (Anticipated)
Phase
- Phase 3
Contacts and Locations
Study Locations
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-
Zuid-Holland
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Rotterdam, Zuid-Holland, Netherlands, 3015GD
- Erasmus MC
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: RANDOMIZED
- Interventional Model: CROSSOVER
- Masking: QUADRUPLE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: Hydrocortisone
Hydrocortisone (20 mg/day) in a 10-5-5 schedule: 10 mg at 0700h, 5 mg at 1200h, and 5 mg at 1700h.
The hydrocortisone periods last 10 weeks per period.
In total, there are two hydrocortisone periods.
|
Hydrocortisone is orally administered.
|
PLACEBO_COMPARATOR: Placebo
Placebo (20 mg/day) in a 10-5-5 schedule: 10 mg at 0700h, 5 mg at 1200h, and 5 mg at 1700h.
The placebo periods last 10 weeks per period.
In total, there are two placebo periods.
|
Placebo is orally administered.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Rasch-built Overall Disability Scale (RODS)
Time Frame: Baseline to 414 days
|
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 Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Size of lipoma
Time Frame: Baseline to 414 days
|
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
|
Medical Research Council (MRC) sumscore
Time Frame: Baseline to 414 days
|
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
|
Goal Attainment Scaling (GAS)
Time Frame: Baseline to 414 days
|
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
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (ANTICIPATED)
Primary Completion (ANTICIPATED)
Study Completion (ANTICIPATED)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ACTUAL)
Study Record Updates
Last Update Posted (ACTUAL)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- N-of-1 SLN
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
IPD Sharing Time Frame
IPD Sharing Access Criteria
IPD Sharing Supporting Information Type
- STUDY_PROTOCOL
- SAP
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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