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Molecular Medicine

, Volume 18, Issue 11, pp 1430–1436 | Cite as

Safety and Efficacy of ARA 290 in Sarcoidosis Patients with Symptoms of Small Fiber Neuropathy: A Randomized, Double-Blind Pilot Study

  • Lara Heij
  • Marieke Niesters
  • Maarten Swartjes
  • Elske Hoitsma
  • Marjolein Drent
  • Ann Dunne
  • Jan C. Grutters
  • Oscar Vogels
  • Michael Brines
  • Anthony Cerami
  • Albert Dahan
Research Article

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.

Notes

Acknowledgments

The authors thank patients, and their families as well as F Breedveld, A Rabelink, L Aarts, E Lansink and E Sarton for support and assistance in making this study possible. This was an investigator-initiated study. ARA 290 was supplied by Araim Pharmaceuticals.

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Authors and Affiliations

  • Lara Heij
    • 1
  • Marieke Niesters
    • 1
  • Maarten Swartjes
    • 1
  • Elske Hoitsma
    • 2
  • Marjolein Drent
    • 3
    • 8
  • Ann Dunne
    • 4
  • Jan C. Grutters
    • 5
    • 6
  • Oscar Vogels
    • 7
  • Michael Brines
    • 4
  • Anthony Cerami
    • 1
    • 4
  • Albert Dahan
    • 1
  1. 1.Department of AnesthesiologyLeiden University Medical CenterLeidenThe Netherlands
  2. 2.Department of NeurologyDiaconessenhuisLeidenThe Netherlands
  3. 3.Faculty of Health, Medicine and Life ScienceUniversity of MaastrichtMaastrichtThe Netherlands
  4. 4.Araim PharmaceuticalsOssiningUSA
  5. 5.Department of PulmonologySt. Antonius Hospital NieuwegeinNieuwegeinThe Netherlands
  6. 6.Division of Heart and LungsUniversity Medical Center UtrechtUtrechtThe Netherlands
  7. 7.Department of NeurologySt. Antonius HospitalNieuwegeinThe Netherlands
  8. 8.Department of Interstitial Lung DiseasesHospital Gelderse ValleiEdeThe Netherlands

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