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

Conditions

• Arterial Calcification
• Autosomal Recessive Hypophosphatemic Rickets Type 2
• Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
• Gene Mutations
• Pseudoxanthoma Elasticum

Eligibility Criteria

Sex

ALL

Age

1 Year - N/A

Healthy Volunteers

Not accepted

Interventions

• INZ-701 — DRUG
  INZ-701 is a 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).

Study Details

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.

Key Dates

Start date

Jun 19, 2024

Status verified

Nov 2024

Primary completion

Nov 30, 2030

Completion

Dec 31, 2030

Study Design

Enrollment

200 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: INZ-701
  INZ-701 will be administered by subcutaneous injection on a once-weekly basis as follows: * Study participants from 1 year to \<13 years of age will receive a dose of 2.4 mg/kg * Study participants ≥13 years will either receive a 1.8 mg/kg dose or a 150 mg flat dose of INZ-701

Primary Outcome Measure

Number of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: 6 years (long term safety assessment) ]

Central Contacts

• Inozyme Clinical Trial Information
  +1 857 330 4340
  [email protected]

Locations (2)

Find similar trials in Rochester, MN

Related Studies

• The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
  PHASE1 · Recruiting · Inozyme Pharma · San Diego, California