The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency

ConditionsEctonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency Autosomal Recessive Hypophosphatemic Rickets Generalized Arterial Calcification of Infancy ATP-Binding Cassette Subfamily C Member 6 Deficiency Pseudoxanthoma Elasticum

Trial statusRecruiting

Trial phasePhase 1

Trial typeInterventional

Biological sexAll

AgeUp to 1

SponsorInozyme Pharma

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Infant aged ≤ 1 year at the time of enrollment

Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency

Study participants must have clinical manifestations of generalized arterial calcification of infancy (GACI) or GACI-2, which must include at least one of the following: ectopic calcification, heart failure, respiratory distress, edema, cyanosis, hypertension, and cardiomegaly.

Study participant must weigh ≥0.5 kg at the time of the first dose of INZ-701 in this study

Disqualifiers

In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality that precludes study participation or may confound interpretation of study result

Receiving end of life or hospice care

Known malignancy

Concurrent participation in another non-Inozyme interventional study

Trial design

Design model

Single group

Treatments tested in this trial

INZ-701
Drug
Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.

Treatment groups

16 Participants

are divided into 1 treatment group

Group A: INZ-701Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

Number 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)

Secondary outcomes

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)