Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency
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|ClinicalTrials.gov Identifier: NCT04686175|
Recruitment Status : Not yet recruiting
First Posted : December 28, 2020
Last Update Posted : January 5, 2021
|Condition or disease||Intervention/treatment||Phase|
|Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency ENPP1||Drug: INZ-701||Phase 1 Phase 2|
This study is a Phase 1/2, multi-center, assessor blind, open-label, first in human study conducted in adults with ENPP1 Deficiency designed to assess the safety, tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of multiple ascending doses of INZ-701. The goal of the study is to identify a dose that restores Plasma Inorganic Pyrophosphate (PPi) to normal levels in ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) Deficiency to be used in further clinical development. No placebo/control arm or reference therapies will be used in the study, but all measurements will be assessor blinded.
The study design is a multiple ascending dose 3x3 schedule (3 dose cohorts with 3 subjects per cohort). Based on nonclinical pharmacology modeling, the planned doses will be 0.2 mg/kg, 0.6 mg/kg, and 1.8 mg/kg.
The Principal Investigator (PI) or an appropriately qualified delegate will administer INZ-701 at the prescribed dose and dosing interval for each given cohort.
The Final PK and Safety Assessment Period includes PK sample collection and safety assessments after the last dose of INZ-701 on Days 30 to 35 and the Final Safety Visit. After completion of the Final Safety Visit, where approved by regulatory agencies and ethics committees, subjects may continue dosing at their assigned dose level as part of a separate, independent long-term extension study.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||9 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Study INZ701-101 is a Phase 1/2, multi-center, assessor blind, open-label, First in human (FIH), First in patient (FIP), and Multiple Ascending Dose (MAD) study conducted in adults with ENPP1 Deficiency. The study design is a MAD 3×3 (3 dose cohorts with 3 subjects per cohort).|
|Masking:||None (Open Label)|
|Official 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|
|Estimated Study Start Date :||January 2021|
|Estimated Primary Completion Date :||January 2022|
|Estimated Study Completion Date :||April 2022|
The study design is a MAD 3×3 (3 dose cohorts with 3 subjects per cohort). The planned doses will be 0.2 mg/kg, 0.6 mg/kg, and 1.8 mg/kg.
Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody (rhENPP1-Fc)
- Dose Toxicity [ Time Frame: 5 weeks ]Occurrence of dose limiting toxicity (DLT) during first 5 weeks of therapy
- Determination of Immunogenicity [ Time Frame: 5 weeks ]The presence of anti-drug antibodies will be assayed and, if present, further evaluation will confirm positivity for antidrug antibodies and determine specificity, neutralizing ability, cell-mediated immune response and correlation with clinical responses.
- Measurement of Plasma Inorganic Pyrophosphate (PPi) in Plasma [ Time Frame: 5 weeks ]For each subject, blood plasma will be assayed for Plasma Inorganic Pyrophosphate (PPi), comparing the subjects baseline value over time
- Measurement of Fibroblast Growth Factor 23 (FGF23) in plasma [ Time Frame: 5 weeks ]For each subject, blood plasma will be assayed for FGF23, comparing the subjects baseline value over time
- Area under the plasma concentration versus time curve (AUC) [ Time Frame: 5 weeks ]For each subject, variation of concentration of INZ-701 in the plasma will be measured
- Peak Plasma Concentration (Cmax) [ Time Frame: 5 weeks ]For each subject, the maximum concentration of INZ-701 in the plasma will be measured
- Systemic clearance [ Time Frame: 5 weeks ]For each subject, clearance of INZ-701 from the body will be measured
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04686175
|Contact: Inozyme Clinical Trial Information||+1 857 330 firstname.lastname@example.org|