Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency

3

Review clinical trials related to Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

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

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.

Participants needed: 16
Trial details
Phase: Phase 1Age: Up to 1Biological sex: AllType: InterventionalSponsor: Inozyme PharmaUpdated: Mar 24, 2026Locations: 7
Eligibility criteria

Infant aged ≤ 1 year at the time of enrollment [+4]

In the opinion of the Investigator, presence of any clinically significant disea... [+4]

Status: Recruiting

ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency

The primary purpose of ENERGY 2 (Study INZ701-105) is to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.

Participants needed: 12
Trial details
Phase: Phase 3Age: 0-1Biological sex: AllType: InterventionalSponsor: Inozyme PharmaUpdated: Mar 18, 2026Locations: 8
Eligibility criteria

Infant aged ≤ 1 year at the time of enrollment. [+4]

Receiving end-of-life or hospice care. [+3]

Status: Recruiting

ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency

The purpose of this study (Study INZ701-304 \[ADAPT\]) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition.

Participants needed: 200
Trial details
Phase: Phase 2Age: 1+Biological sex: AllType: InterventionalSponsor: Inozyme PharmaUpdated: Nov 29, 2024Locations: 5
Eligibility criteria

Provide written or electronic informed consent after the nature of the study has... [+6]

In the opinion of the Investigator, presence of any clinically significant disea... [+4]