RecruitingPhase 1

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

United States16 participantsStarted 2023-06-25

Plain-language summary

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.

Who can participate

Age range1 Year

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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
✓. Written informed consent provided by a parent or legal guardian

Exclusion criteria

✕. 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
✕. Treatment with any non-Inozyme product or investigational device during study participation

What they're measuring

Number of Treatment Emergent Adverse Events (TEAEs)

Timeframe: 52 weeks (Treatment Period)

Incidence of Anti-Drug Antibodies (ADA)

Timeframe: 52 weeks (Treatment Period)

Left Ventricular Ejection Fraction

Timeframe: 52 weeks (Treatment Period)

Trial details

NCT IDNCT05734196

SponsorInozyme Pharma

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-11-11

Contact for this trial

Trial Specialist

+1 800-983-4587 medinfo@bmrn.com

View on ClinicalTrials.gov More Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency trials

Who can participate

Age range1 Year

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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
✓. Written informed consent provided by a parent or legal guardian

Exclusion criteria

✕. 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
✕. Treatment with any non-Inozyme product or investigational device during study participation