Scientific Publications

2025

Trappsol® Cyclo™ (HPβCD) for the Long-Term Treatment of Niemann-Pick Type C1: Efficacy and Safety Data from 4 Clinical Studies and the Ongoing Expanded Access Program

21st Annual WORLDSymposium™, February 3-7, 2025, San Diego, CA

Authors: Caroline Hastings, Fatih Ezgu, Roberto Giugliani, Beata Kieć-Wilk, Lukasz Pawlinski, Eugen Mengel, Elena Martin-Hernandez, Sema Kalkan Ucar, Reena Sharma, Nicholas Smith, Yin-Hsiu Chien1, Mark Walterfang, Moeen AlSayed, Ozlem Goker- Alpan, Leonardo Oliveira Mendonça, Julian Raiman, Ronen Spiegel, Rita Barone, Alberto Burlina, Cristian Calandra, Jordi Gascón, Heidi Peters, Orna Staretz Chacham, Bryan Murray, Andreas Brecht, and Joseph Mejia

Trappsol® Cyclo™ and NPC: Efficacy Shown Across Individual 5D Domains and Utilization of Future Assessment Tools to Demonstrate Clinically Relevant Outcomes

21st Annual WORLDSymposium™, February 3-7, 2025, San Diego, CA

Authors: Caroline Hastings, Fatih Ezgu, Roberto Giugliani, Beata Kieć-Wilk, Lukasz Pawlinski, Eugen Mengel, Patricia Perez Mohand, Sema Kalkan Ucar, Reena Sharma, Nicholas Smith, Yin-Hsiu Chien, Mark Walterfang, Moeen AlSayed, Ozlem Goker-Alpan, Leonardo Oliveira Mendonça, Julian Raiman, Ronen Spiegel, Rita Barone, Alberto Burlina, Cristian Calandra, Jordi Gascón, Heidi Peters, Orna Staretz Chacham, Bryan Murray, Andreas Brecht, and Joseph Mejia

Trappsol® Cyclo™: Open Label Treatment in the TransportNPC™ Sub-Study in Patients Under the Age of 3 Diagnosed with Niemann-Pick Disease Type C1

21st Annual WORLDSymposium™, February 3-7, 2025, San Diego, CA

Authors: Orna Staretz Chacham, Fatih Ezgu, Roberto Giugliani, Eugen Mengel, Sema Kalkan Ucar, Ronen Spiegel, Caroline Hastings, Bryan Hurst, Bryan Murray, Andreas Brecht, and Joseph Mejia

2024

TRANSPORTNPC™: Phase 3 global trial of intravenous hydroxypropyl-beta-cyclodextrin (HPβCD) in patients three years of age or older with Niemann-Pick disease type C1 (NPC1)

SSIEM Annual Symposium, September 3-6, 2024, Porto, Portugal.

Authors: Caroline Hastings, Roberto Giugliani, Fatih Ezgu, Beata Kieć-Wilk, Lukasz Pawlinski, Eugen Mengel, Elena Martin-Hernandez, Thorsten Marquardt, Reena Sharma, Nicholas Smith, Yin-Hsiu Chien, Sema Kalkan Ucar, Mark Walterfang, Dariusz Rokicki, Moeen Al-Sayed, Victor Fung, Ozlem Goker-Alpan1, Norberto Guelbert, Leonardo Oliveira Mendonça, Julian Raiman, Anna Ardissone, Rita Barone, Alberto Burlina, Cristian Calandra, Jordi Gascon-Bayarri, Loren Pena, Heidi Peters, Ronen Spiegel27, Orna Staretz Chacham, Bryan Hurst, Bryan Murray, Andreas Brecht, and Joseph Meji

TRANSPORTNPC™: Phase 3 global trial of intravenous hydroxypropyl-beta-cyclodextrin (HPβCD) in patients with Niemann-Pick disease type C1 (NPC1) – open label sub-study in patients below 3 years of age

SSIEM Annual Symposium, September 3-6, 2024, Porto, Portugal.

Authors:  Orna Staretz Chacham, Fatih Ezgu, Eugen Mengel, Sema Kalkan Ucar, Ronen Spiegel, Dariusz Rokicki, Moeen Al-Sayed, Caroline Hastings, Roberto Giugliani, Bryan Hurst1, Bryan Murray1, Andreas Brecht1, and Joseph Mejia

2023

Cyclo Therapeutics Scientific Presentation

          December, 2023

Intravenous Trappsol® Cyclo ™ in patients with Niemann Pick Disease type C1: Updates on the Results from Phase I and Phase I/II studies and the international Phase III pivotal Transport NPC trial

          NP Association of China, Patient Group Meeting, July 16, 2023    
          Authors: Andreas Brecht, PhD – Director Medical Affairs Europe Cyclo Therapeutics

 

Long-term administration of intravenous Trappsol® Cyclo™ (HP-β-CD) results in clinical benefits and stabilization or slowing of disease progression in patients with Niemann-Pick disease type C1: Results of an international 48-week Phase I/II trial

Authors: Reena Sharmaa / Caroline Hastings / Orna Staretz-Chachamc / Julian Raimand / Martin Paucar / Ronen Spiegel /
Bryan Murray / Bryan Hurst / Benny Liu / Lise Kjems / Sharon Hrynkow

 

TransportNPC: A Phase 3 Global Trial of Trappsol® Cyclo™ Administered Intravenously to Patients with Niemann-Pick Disease Type C1 (NPC1)

          Authors: Joseph Mejia, MD / Lise Kjems, PhD, MD / Rebecca Fine / Caroline Hastings, MD

 

2022

 Intravenous 2-hydroxypropyl-β-cyclodextrin (Trappsol® Cyclo™) demonstrates biological activity and impacts cholesterol metabolism in the central nervous system and peripheral tissues in adult subjects with Niemann-Pick Disease Type C1: Results of a phase 1 trial

          Authors: Caroline Hastings, MD / Benny Liu, MD / Bryan Hurst / Gerald F. Cox, MD, PhD / Sharon Hrynkow, PhD

 

Intravenous Trappsol® Cyclo™in patients with Niemann Pick Disease type C1: Updates on the Results from Phase I and Phase I/II studies and launch of the international Phase III pivotal Transport NPC trial

      • NNPDF/INPDA Annual Meeting, Orlando, FL
      • Caroline Hastings, MD / Lise Kjems, MD, PhD
      • July 30, 2022

From Bench to Bedside – A Review of the Trappsol® Cyclo™ Program for the Treatment of Niemann Pick Disease Type C

      • World Orphan Drug Congress USA 2022
      • Lise Kjems, MD, PhD
      • July 13, 2022

    2021

    Topline Results from Phase 1/2 Study to Evaluate Safety and Efficacy of Intravenous Trappsol® Cyclo™ in Patients with Niemann-Pick Type C1 Disease

        • Australian NPC Disease Foundation Annual Conference
        • Julian Raiman, MD
        • June 25, 2021
      Results from Phase 1 study to evaluate the single- and multiple-dose pharmacokinetics of Intravenous Trappsol® Cyclo ™ in patients with Niemann Pick Disease type C1 and the Effects of Dosing upon Biomarkers of NPC Disease

          • Australian NPC Disease Foundation Annual Conference
          • Caroline Hastings, MD
          • June 25, 2021

        Phase 3 Study to Evaluate Intravenous Trappsol® Cyclo™ Versus Placebo in Pediatric and Adult Patients With Niemann-Pick Disease Type C1

            • Australian NPC Disease Foundation Annual Conference
            • Gerald Cox, MD, PhD
            • June 25, 2021
          A phase I/II Study to Evaluate the Safety and Pharmacokinetics of Intravenous Trappsol® Cyclo™ Versus Placebo in Patients With Niemann-Pick Disease Type C (NPC1) and the Pharmacodynamic Effects of Treatment upon Markers of Cholesterol Metabolism and Clinical Outcomes (NCT02912793)

              • WORLDSymposium presentation
              • Julian Raiman, MD
              • Feb 9, 2021

            Update on Phase 1 study to evaluate the single- and multiple-dose pharmacokinetics of intravenous hydroxypropyl betacyclodextrin (HPβCD) in patients with Niemann-Pick disease type C (NPC-1) and the effects of dosing upon biomarkers of NPC disease (NCT02939547)

              • WORLDSymposium presentation
              • Caroline Hasting, MD
              • Feb 9, 2021

            2020

            Interim Analysis of Phase I/II Trial with Trappsol® Cyclo™ Delivered Intravenously in NPC Patients

              • NNPDF Presentation
              • Julian Raiman, MD
              • Jul 9, 2020

            Top Line Results of Phase I Intravenous Trial for NPC

              • NNPDF Presentation
              • Caroline Hasting, MD
              • Jul 9, 2020
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