Trials / Withdrawn
WithdrawnNCT06739980
The ENABLE Study: Safety and Efficacy Study of INZ-701 in Patients With ENPP1 Deficiency
The ENABLE Study: An Open-Label, Long-Term Safety and Efficacy Study of INZ-701 in Patients With Ectonucleotide Pyrophosphatase/ Phosphodiesterase 1 (ENPP1) Deficiency
- Status
- Withdrawn
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 0 (actual)
- Sponsor
- Inozyme Pharma · Industry
- Sex
- All
- Age
- 1 Year
- Healthy volunteers
- Not accepted
Summary
The purpose of Study INZ701-108 (ENABLE) is to assess the safety and efficacy of INZ-701 in patients 1 year of age and older with ENPP1 Deficiency who have not previously received INZ-701 and are not eligible for existing Inozyme-sponsored clinical studies that are still open to enrollment.
Detailed description
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. Study INZ701-108 (ENABLE) is an open-label study to assess the long-term safety and efficacy of INZ-701 in patients 1 year of age and older with ENPP1 Deficiency who have not previously received INZ-701 and are not eligible for an Inozyme-sponsored clinical study that is open to recruitment. The study will consist of a 30-day Screening Period, followed by an open-label Treatment Period during which all participants will receive once-weekly subcutaneous (SC) doses of INZ-701 and continue treatment through 104 weeks. The dose will be determined by the participant's age and weight.
Conditions
- Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
- Autosomal Recessive Hypophosphatemic Rickets
- Generalized Arterial Calcification of Infancy 1
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | INZ-701 | Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody. |
Timeline
- Start date
- 2025-01-31
- Primary completion
- 2027-03-31
- Completion
- 2028-03-31
- First posted
- 2024-12-18
- Last updated
- 2025-05-01
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06739980. Inclusion in this directory is not an endorsement.