Evaluate the Safety, Tolerability, PK & PD of INZ-701

• Research type

  Research Study

• Full title

  A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Adults with ENPP1 Deficiency

• IRAS ID

  290016

• Contact name

  Peter Selby

• Contact email

  peter.selby@cmft.nhs.uk

• Sponsor organisation

  Inozyme Pharma, Inc.

• Eudract number

  2020-003716-27

• Clinicaltrials.gov Identifier

  NCT04686175

• Clinicaltrials.gov Identifier

  n/a, n/a

• Duration of Study in the UK

  1 years, 2 months, 0 days

• Research summary

  Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency is a rare, genetic disorder caused by inactivating mutations in the Enpp1 gene that encodes the ENPP1 enzyme.
  Study INZ701-101 is a Phase 1/2, open-label, first-in-human (FIH), first-in-patient (FIP), multiple ascending dose (MAD), dose-finding study in adult subjects (18 to 64 years of age) with ENPP1 Deficiency. This is a multicenter, multinational trial to be conducted at 1 site in the United States, 1 site in Germany and 1 site in United Kingdom. Approximately 9 adult subjects will be included.
  This study will explore the safety and tolerability, immunogenicity (ability to cause an immune response), and pharmacokinetics (abbreviated PK; means the effects of drugs and how they interact in the body) of multiple subcutaneous (abbreviated SC; means injected by a short needle under the skin) doses of INZ-701. This study will also explore the changes in a chemical called pyrophosphate (abbreviated PPi) and associated biomarkers of ENPP1 Deficiency, which are called pharmacodynamic (PD) changes. This study is different from the regular medical care. The purpose of regular medical care is to improve or manage health; however, the purpose of research is to gather information to advance science and medicine and possibly develop a treatment for ENPP1 Deficiency, and does not replace the regular medical care.

• REC name

  London - Chelsea Research Ethics Committee

• REC reference

  20/LO/1305

• Date of REC Opinion

  20 Jan 2021

• REC opinion

  Further Information Favourable Opinion