The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 Deficiency.
INZ-701 is an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy in development for the treatment of the ultra-rare genetic disorder, ENPP1 Deficiency or with ABCC6 Deficiency. Study INZ701-104 (the ENERGY study) is a Phase 1b, open-label study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of INZ-701 in infant study participants with ENPP1 Deficiency or ABCC6 Deficiency. The study will consist of up to a 60-day Screening Period, a 52-week Treatment Period during which study participants will receive INZ-701, an Extension Period during which participants may continue to receive INZ-701 until it is commercially available in the country where the participant resides, or until an alternative study of INZ-701 is available, and an End of Treatment (EOT) visit 30 days after the last dose of INZ-701. Upon treatment discontinuation, participants will continue to be followed for their ongoing disposition for survival outcome at least quarterly through the end of the study.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
16
Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.
Rady Children's Hospital
San Diego, California, United States
RECRUITINGBoston Children's Hospital
Boston, Massachusetts, United States
RECRUITINGNationwide Children's Hospital
Columbus, Ohio, United States
RECRUITINGThe Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
RECRUITINGThe University of Utah
Salt Lake City, Utah, United States
RECRUITINGHospital Sant Joan de Déu
Barcelona, Spain
NOT_YET_RECRUITINGRoyal Manchester Children's Hospital
Manchester, United Kingdom
RECRUITINGNumber of Treatment Emergent Adverse Events (TEAEs)
Treatment-emergent AEs are defined as any AE occurring from the first dose of INZ-701 through 30 days after the last dose of INZ-701.
Time frame: 52 weeks (Treatment Period)
Incidence of Anti-Drug Antibodies (ADA)
For each participant, the presence of ADAs will be assessed and, if present, further evaluation will determine specificity and subtypes.
Time frame: 52 weeks (Treatment Period)
Left Ventricular Ejection Fraction
For each participant, an echocardiogram will be collected, and used to assess heart function. (Including measurement of left ventricular ejection fraction), and to identify any other abnormalities, for example, calcification of heart valves.
Time frame: 52 weeks (Treatment Period)
Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels
For each participant, plasma PPi will be measured via a series of blood samples obtained throughout the study, comparing the participant's baseline value over time.
Time frame: 52 weeks (Treatment Period)
Area under the Plasma Concentration versus Time Curve (AUC) of INZ-701
For each participant, variation of concentration of INZ-701 in the plasma will be measured via a series of blood samples obtained throughout the study, comparing the participant's baseline value over time.
Time frame: 52 weeks (Treatment Period)
Maximum Plasma Concentration (Cmax) of INZ-701
For each participant, the maximum concentration of INZ-701 in the plasma will be measured via a series of blood samples obtained throughout the study, comparing the participant's baseline value over time.
Time frame: 52 weeks (Treatment Period)
ENPP1 Activity
For each participant, the activity of INZ-701 in the serum will be measured via a series of blood samples obtained throughout the study, comparing the participant's baseline value over time.
Time frame: 52 weeks (Treatment Period)
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