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A Phase 2, Single Center, Open-Label, Multiple Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Daily Subcutaneously Administered ELX-02 in Patients With Nephropathic Cystinosis Bearing One or More CTNS Gene (Cystinosin) Nonsense Mutations

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Trial Profile

A Phase 2, Single Center, Open-Label, Multiple Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Daily Subcutaneously Administered ELX-02 in Patients With Nephropathic Cystinosis Bearing One or More CTNS Gene (Cystinosin) Nonsense Mutations

Status: Discontinued
Phase of Trial: Phase II

Latest Information Update: 05 Nov 2021

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At a glance

  • Drugs Exaluren (Primary)
  • Indications Nephropathic cystinosis
  • Focus Adverse reactions; Pharmacokinetics; Proof of concept
  • Sponsors Eloxx Pharmaceuticals

    • Most Recent Events

    • 22 May 2020 Status changed from recruiting to discontinued.
    • 14 Jan 2020 According to an Eloxx Pharmaceuticals Media Release, company is reviewing cohort 1 data with a panel of cystinosis scientific and clinical experts to determine if protocol modifications would be appropriate before initiating cohort 2 of this study.
    • 14 Jan 2020 Data fro from the first cohort of this study presented in an Eloxx Pharmaceuticals Media Release.
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