Clinical Research Directory

Subcutaneous Immunoglobulin Therapy Effectiveness Monitoring in CIDP Patients Using Smart Devices

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare autoimmune disease that affects the peripheral nerves. It causes progressive weakness and sensory loss in the arms and legs, which can severely limit daily activities. Many patients need long-term treatment with immunoglobulins, either through intravenous infusions (IVIG) or subcutaneous injections (fSCIG). The S.T.E.P.S. study aims to explore how digital health technologies-specifically smartwatches-can help monitor the disease course and treatment effects in CIDP patients who use fSCIG at home. Current clinical tests are useful but sometimes miss small changes in strength or function. Wearables may provide a more detailed and continuous picture of patients' health between clinic visits. Study Goals: The main goal is to find out whether smartwatch data (such as step count and physical activity) reflect disease severity and treatment response when compared to standard clinical scores like the Inflammatory Neuropathy Cause and Treatment (INCAT) scale and the Inflammatory Rasch-built Overall Disability Scale (I-RODS). Secondary goals include: Assessing how smartwatch data relate to patients' quality of life and sleep patterns. Comparing smartwatch results with other clinical scores such as muscle strength (MRC sum score) and grip strength. Evaluating how well patients can use the smartwatch over the long term during home treatment. An additional exploratory goal is to see whether smartwatch data can detect early signs of worsening disease before symptoms appear. Study Design: This is a 12-month observational study with five main clinic visits (at 0, 3, 6, 9, and 12 months). After enrolling, participants will begin or continue subcutaneous immunoglobulin therapy as decided by their treating physician. Each visit includes standard clinical assessments and questionnaires. Participants will receive a smartwatch at the start of the study to continuously track their activity and sleep patterns. A follow-up phone call one week later will check that the device is working properly. Duration: Recruitment will last about 6 months, and each participant will be followed for 12 months. Why This Matters: By combining established clinical measures with continuous digital monitoring, the S.T.E.P.S. study may help improve understanding of disease activity and treatment response in CIDP. This could lead to more personalized therapy schedules and better long-term care for patients.

A Study to Assess Efficacy and Safety of Empasiprubart Versus IVIg in Adults With CIDP

The main purpose of this study is to compare empasiprubart and IVIg for treating people with CIDP. This study consists of a Part A where participants will either receive empasiprubart and a placebo resembling IVIg, or IVIg and a placebo resembling empasiprubart for 24 weeks (6 months). Following Part A, participants will enter Part B in which all participants will receive empasiprubart for 96 weeks (24 months). More information can be found here: https://clinicaltrials.argenx.com/emvigorate

A Worldwide Pregnancy Safety Study to Assess Maternal, Fetal, and Infant Outcomes Following Exposure to Efgartigimod During Pregnancy and/or Breastfeeding.

This is a multi-country, prospective safety study of pregnant women exposed to efgartigimod or efgartigimod PH20 SC any time within 25 days prior to conception or any time during pregnancy. Women exposed to efgartigimod or efgartigimod PH20 SC only during breastfeeding will also be eligible to enroll. Background rates of major congenital malformations (MCMs) will be obtained from populations within the same countries/regions as the countries/regions in which the efgartigimod or efgartigimod PH20 SC exposed pregnancies were reported.

Multispectral Optoacoustic Imaging for the Detection of Inflammation and Damage of Peripheral Nerves in Guillain-Barré Syndrome and Chronic Inflammatory Demyelinating Polyneuropathy

The aim of this study is to assess disease activity in patients with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) and Guillain-Barré Syndrome (GBS) using multispectral optoacoustic tomography (MSOT). The currently available diagnostic procedures for CIDP and GBS do not allow for a clear distinction between remission and active disease and show limitations in sensitivity and specificity during acute diagnostics. This can lead to delayed diagnosis, which is crucial for timely initiation of therapy and, consequently, for a better prognosis. Long-term therapy management is also challenging, as objective parameters for assessing therapeutic success are largely lacking. MSOT can detect inflammation through the measurement of hemoglobin, a method that our research group has already successfully demonstrated in patients with chronic inflammatory bowel diseases (Knieling, NEJM 2017). The use of MSOT could therefore also be applied to nerve inflammation, allowing for earlier detection of inflammation and nerve damage and contributing to timely treatment of patients. For nerve imaging, a CE-certified MSOT device from iThera Medical is available at the Pediatric Clinic in Erlangen. In addition, a non-CE-certified device with lower laser power is available, which can be used for bedside examinations. The arm nerves will be examined in three cohorts, which will be compared with each other and with standard diagnostics (blood tests, electrophysiology, conventional ultrasound). Each cohort will include ten healthy control subjects, ten patients with CIDP, and ten patients with GBS.

Subcutaneous Immunoglobulin in De-novo CIDP (SIDEC)

SIDEC - (Subcutaneous Immunoglobulin in De-novo CIDP) ia a study designed as a randomized, parallel study with an open-label extension phase. The aims are to compare the effect of SCIG and IVIG in 60 treatment-naïve CIDP patients, and to detect the lowest effective dosage for maintenance treatment.