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

NCT04821583 | Unknown Phase 3

• 1 other identifier: N-of-1 SLN
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

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).

Conditions

Symmetric Lipomatoses, Multiple

Keywords

Symmetric Lipomatosis associated with Neuropathy; Hydrocortisone; Lipoma; Neuropathy

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Size of lipoma

  Baseline to 414 days

• Medical Research Council (MRC) sumscore

  Baseline to 414 days

• Goal Attainment Scaling (GAS)

  Baseline to 414 days

Study Arms (2)

Hydrocortisone

EXPERIMENTAL

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.

Drug: Hydrocortisone

Placebo

PLACEBO COMPARATOR

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.

Drug: Placebo

Interventions

Hydrocortisone DRUG

Hydrocortisone is orally administered.

Hydrocortisone

Placebo DRUG

Placebo is orally administered.

Placebo

Eligibility Criteria

• Age: 31 Years+
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may not qualify if:

• N/A
• Pregnancy

Sponsors & Collaborators

• Erasmus Medical Center: lead

Study Sites (1)

Erasmus MC

Rotterdam, South Holland, 3015GD, Netherlands

Location

MeSH Terms

Conditions

Lipomatosis, Multiple Symmetrical; Lipoma

Interventions

Hydrocortisone

Condition Hierarchy (Ancestors)

Lipomatosis; Skin Diseases; Skin and Connective Tissue Diseases; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Neoplasms, Adipose Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms

Intervention Hierarchy (Ancestors)

Pregnenediones; Pregnenes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; 11-Hydroxycorticosteroids; Hydroxycorticosteroids; Adrenal Cortex Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; 17-Hydroxycorticosteroids

Study Officials

• Laura de Graaff, MD, PhD

  Department of Internal Medicine - Endocrinology, Erasmus MC, Rotterdam, the Netherlands

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Laura de Graaff, MD, PhD

CONTACT

+31618843010; l.degraaff@erasmusmc.nl

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Both the participant and the investigator are masked. The investigator is also the care provider and outcomes assessor. The statistician and clinical pharmacist are unblinded.
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal investigator

Model Details: An N-of-1 study is considered as a reliable and suitable design to assess the efficacy of a treatment at an individual level. The study will consist of two cycles with each two periods of active treatment or placebo, a run-in period, tapering period and a wash-out period. Within each cycle, the sequence of the hydrocortisone and placebo period is randomized.

Study Record Dates

• First Submitted: March 16, 2021
• First Posted: March 29, 2021
• Study Start: June 1, 2021
• Primary Completion: September 1, 2022
• Study Completion: September 1, 2022
• Last Updated: March 29, 2021

Record last verified: 2021-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: The data will become available after completion of the study and only upon reasonable request and with permission of the participant.
• Access Criteria: A motivated request.

Locations

NL (1)