title: Safety and efficacy of ARA 290 in sarcoidosis patients with symptoms of small fiber neuropathy: a randomized, double-blind pilot study.
abstract: ARA 290 (a peptide designed to activate the innate repair receptor that arrests injury and initiates cytoprotection, antiinflammation and healing) reduces allodynia in preclinical neuropathy models. We studied the safety and efficacy of ARA 290 to reduce symptoms of small fiber neuropathy (SFN) in patients with sarcoidosis. A total of 22 patients diagnosed with sarcoidosis and symptoms of SFN were enrolled in a double-blind, placebo-controlled exploratory trial consisting of three times weekly intravenous dosing of ARA 290 (2 mg; n = 12) or placebo (n = 10) for 4 wks. Inclusion criteria were a diagnosis of neuropathy and a spontaneous pain score of ≥5 (Brief Pain Inventory [BPI]). Endpoints assessed were changes in pain intensity and the small fiber neuropathy screening list (SFNSL) score, quality of life (SF-36), depressive symptoms (Inventory of Depressive Symptomatology [IDS]) and fatigue (Fatigue Assessment Scale [FAS]). No safety concerns were raised by clinical or laboratory assessments. The ARA 290 group showed significant (p < 0.05) improvement at wk 4 in SFNSL score compared with placebo (Δ -11.5 ± 3.04 versus Δ -2.9 ± 3.34 [standard error of the mean]). Additionally, the ARA 290 group showed a significant change from baseline in the pain and physical functioning dimensions of the SF-36 (Δ -23.4 ± 5.5 and Δ -14.6 ± 3.9, respectively). The mean BPI and FAS scores improved significantly but equivalently in both patient groups. No change was observed in the IDS. ARA 290 appears to be safe in patients with sarcoidosis and can reduce neuropathic symptoms.
authors.affiliation: Department of Anesthesiology, Leiden University Medical Center, Leiden, the Netherlands.
authors.forename: Lara
authors.initials: L
authors.lastname: Heij
authors.forename: Marieke
authors.initials: M
authors.lastname: Niesters
authors.forename: Maarten
authors.initials: M
authors.lastname: Swartjes
authors.forename: Elske
authors.initials: E
authors.lastname: Hoitsma
authors.forename: Marjolein
authors.initials: M
authors.lastname: Drent
authors.forename: Ann
authors.initials: A
authors.lastname: Dunne
authors.forename: Jan C
authors.initials: JC
authors.lastname: Grutters
authors.forename: Oscar
authors.initials: O
authors.lastname: Vogels
authors.forename: Michael
authors.initials: M
authors.lastname: Brines
authors.forename: Anthony
authors.initials: A
authors.lastname: Cerami
authors.forename: Albert
authors.initials: A
authors.lastname: Dahan
mesh_terms: D004311:Double-Blind Method; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009422:Nervous System Diseases / Q000150:complications* / Q000188:drug therapy* / Q000503:physiopathology; D010455:Peptides / Q000008:administration & dosage / Q000009:adverse effects* / Q000494:pharmacology / Q000627:therapeutic use*; D010865:Pilot Projects; D010919:Placebos; D012507:Sarcoidosis / Q000150:complications* / Q000188:drug therapy* / Q000503:physiopathology; D012677:Sensation / Q000187:drug effects; D011795:Surveys and Questionnaires; D016896:Treatment Outcome
chemical_list: D010455:Peptides; D010919:Placebos