Nanoencapsulated Sirolimus Plus Pegadricase (NASP) Reduces Gout Clinical Manifestations in Patients with Uncontrolled Gout (UG) and Stage 3 CKD
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Le résumé fourni par la source
Background: Patients (pts) with UG often have chronic kidney disease (CKD). CKD limits urate-lowering therapy efficacy in UG, amplifying the unmet needs for pts with CKD. NASP (formerly SEL-212) is an investigational, novel, every 4-wk (Q4W), sequential 2-component infusion therapy consisting of targeted nanoencapsulated sirolimus (NAS) and pegadricase (P) that reduces serum uric acid (sUA). NASP reduced sUA levels in pts with stage 3 CKD (CKD3) in the phase 3 DISSOLVE I and II (DI/II) studies (NCT04513366, NCT04596540; Khanna et al, ASN 2024). Here, we report additional efficacy and safety outcomes. Methods: In DI/II, pts with UG were randomized 1:1:1 to receive NASP (0.15 mg/kg [high-dose; HD] or 0.1 mg/kg [low-dose; LD] NAS plus 0.2 mg/kg P) or placebo (PBO) Q4W for six treatment periods. This post hoc analysis included pts with CKD3 (eGFR 30–59 mL/min/1.73 m2). Results: Of 265 pts in the intent-to-treat (ITT) populations, 61 had CKD3 (HD NASP: n=20; LD NASP: n=18; PBO: n=23); baseline disease burden was high (Table). A larger proportion of NASP- vs PBO-treated pts had a complete resolution of tophi by wk 24 (HD NASP: 39%; LD NASP: 53%; PBO: 2%; Table). Compared to PBO, the least-square mean difference (standard error) in the change in number of tender/swollen joints from baseline to wk 24 was −3.1/−0.9 (1.52/0.98) for HD NASP, −3.8/−0.4 (1.53/0.98) for LD NASP. Gout flares (GFs) were similar in NASP- and PBO-treated pts during wk 1–20; NASP-treated pts had fewer GFs in wk 21–24. Safety was similar to the ITT population (Table), with only one case of proteinuria in each NASP arm (not considered drug related). Conclusion: NASP was well tolerated and reduced disease burden in pts with UG and CKD. Funding: Commercial Support - The DISSOLVE I and II (NCT04513366 and NCT04596540) studies were jointly funded by Sobi and Selecta Biosciences, Inc. Sobi funded medical writing and editorial assistance.
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Le contrôle bibliographique ouvert
DOI retrouvé dans Crossref DOI retrouvé ; titre concordant.
- Titre Crossref
- Nanoencapsulated Sirolimus Plus Pegadricase (NASP) Reduces Gout Clinical Manifestations in Patients with Uncontrolled Gout (UG) and Stage 3 CKD
- Date Crossref
- 01/10/2025
- Éditeur
- Ovid Technologies (Wolters Kluwer Health)
- Type
- journal-article
Ce recoupement confirme des métadonnées liées au DOI. Il ne confirme ni la méthode ni les conclusions de l’étude, et il ne compte pas comme une seconde source scientifique indépendante.
Où se fait cette recherche
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University Hospitals Geauga Medical Center pays non établi dans la noticeÉtablissement de santé
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Case Western Reserve University pays non établi dans la noticeUniversité ou école supérieure
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University of Michigan pays non établi dans la noticeUniversité ou école supérieure
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Michigan Medicine pays non établi dans la noticeÉtablissement de santé
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Swedish Orphan Biovitrum (Sweden) pays non établi dans la noticeEntreprise
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University of Southern California pays non établi dans la noticeUniversité ou école supérieure
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UH Geauga Medical Center pays non établi dans la noticeÉtablissement de santé
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Sobi Inc pays non établi dans la noticeEntreprise
University Hospitals Geauga Medical Center, Case Western Reserve University et University of Michigan, avec 5 autres affiliations.
Une affiliation ne permet pas de déduire la nationalité d’un auteur.