sarcoidosis (sarcoidose) neuropathy (neuropathie) pain (pijn) eye exams (oogonderzoek)
Conditions
Interventions
ARA 290 (1, 4 or 8 mg) or placebo injected subcutaneously daily for 28 consecutive days
Sponsors
ARAIM Pharmaceuticals Inc
Eligibility
Inclusion criteria
Inclusion criteria: The subjects will have to present the following criteria: Established diagnosis of sarcoidosis with any of the following two criteria: 1) Score of 4 or greater on Brief Pain Inventory “pain now” or “average pain” questions (BPI; 0 (least discomfort)-10 (worst discomfort)) 2) Discomfort defined as distal pain/discomfort plus one of the following: 1) dysesthesia, 2) burning/painful feet worsening at night, or 3) intolerance of sheets or clothes touching the legs or feetAND either of the following two criteria 1) Corneal nerve fiber density reduced compared to normal (i.e., greater than 1 standard deviation less than the mean of a normative population) 2) A previous skin biopsy (obtained within the prior 2 years) showing a reduced intraepidermal nerve fiber density ((i.e., greater than 1 standard deviation less than the mean of a normal age and gender relevant population) In addition, subjects must:  Be able to read and understand the written consent form, complete studyrelated procedures, and communicate with the study staff  Be willing to comply with study restrictions  Be willing to check in with the study center via the telephone  Between 18 and 70 years of age (inclusive)  Body Mass Index (BMI) < 35 kg/m2 (inclusive)  If female of childbearing potential, a negative urine pregnancy test at screening and acceptable contraception will be maintained during the screening and dosing period and 1 month beyond. Acceptable contraception consists of hormonal methods such as oral, implantable, injectable, or transdermal contraceptives for a minimum of 1 full cycle (based on the patient’s usual menstrual cycle period) before study entry, intrauterine device (IUD), or double-barrier method (condoms, sponge, diaphragm, or vaginal ring with spermicidal jellies or cream).  Able to complete self-administered questionnaires (RAND-36, SFNSL, BPI, COMPASS-31, FAS, NPSI)  Refrigerator at home for storage of study medication.
Exclusion criteria
Exclusion criteria: The subjects should not present any of the following criteria:  Clinically relevant abnormal history of physical and mental health other than conditions related to sarcoidosis, as determined by medical history taking (as judged by the investigator)  Clinically relevant abnormal laboratory results, vital signs, or physical findings other than conditions related to sarcoidosis (as judged by the investigator) or could interfere with conduct of 6-minute walk assessment  Known clinically relevant abnormalities in ECG (as judged by the investigator)  Illicit drug abuse or excessive alcohol consumption (as judged by the investigator)  History of serious malignancy within the last 5 years other than a basal cell or squamous cell carcinoma that has been removed  History of fainting (as judged by the investigator)  History of severe allergies, or has had an anaphylactic reaction or significant intolerability to prescription or non-prescription drugs or food (as judged by the investigator)  Anti-TNF therapy or other biological anti-inflammatory agents administered within the 6 months prior to screening.  Use of erythropoiesis stimulating agents within the two months prior to screening or during the trial  Participation in an investigational drug trial in the 3 months prior to administration of the initial dose of study drug or more than 4 times in the calendar year preceding study enrollment  Inadequate venous accessibility as judged by clinicians (physician or nurse)  Inability or unwillingness to self-administer ARA 290 via subcutaneous injections (or not have access to home health care for assistance in administration)  If female, pregnant or breast-feeding  Any other condition that in the opinion of the investigator would complicate or compromise the study, or the well-being of the patient
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The study primary endpoint is:  change in corneal nerve fiber density at day 28 versus baseline | — |
Secondary
| Measure | Time frame |
|---|---|
| The study secondary endpoints are:  change in IENFD of an ankle biopsy at 28 days versus baseline  change in the scores of the Small Fiber Neuropathy Screening List, BPI, NPSI, RAND-36, FAS, and COMPASS-31 at days 35 and 56 compared to baseline  change in quantitative sensory testing (QST) at day 28 versus baseline  change in the 6 minute walk test at day 28 versus baseline  change in cardiac autonomic function (heart rate variability; R-R and QT intervals) at day 28 versus baseline  change in response to anergy skin panel at day 28 versus baseline  change in the scores of the Small Fiber Neuropathy Screening List, BPI, NPSI, RAND-36, FAS, and COMPASS-31 at days 70, 84, 98 and 112 compared to days 28, 56 and baseline (persistence of effect)  frequency of adverse events, serious adverse events, and laboratory parameters | — |
Contacts
Public ContactM. Velzen, van
LUMC, Anesthesiology, P5 Albinusdreef 2
Outcome results
None listed