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| Name | Class |
|---|---|
| BioMarin Pharmaceutical | INDUSTRY |
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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.
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 or with ABCC6 Deficiency.
Study INZ701-104 (the ENERGY study) is a Phase 1b, open-label study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of INZ-701 in infant study participants with ENPP1 Deficiency or ABCC6 Deficiency.
The study will consist of up to a 60-day Screening Period, a 52-week Treatment Period during which study participants will receive INZ-701, an Extension Period during which participants may continue to receive INZ-701 until it is commercially available in the country where the participant resides, or until an alternative study of INZ-701 is available, and an End of Treatment (EOT) visit 30 days after the last dose of INZ-701. Upon treatment discontinuation, participants will continue to be followed for their ongoing disposition for survival outcome at least quarterly through the end of the study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| INZ-701 | Experimental | The first 2 study participants will receive 1 dose of 0.2 mg/kg on Day 1 and start at Dose Level A (0.2 mg/kg twice weekly) on Day 8. The Data Review Committee (DRC) comprised of representatives of the Sponsor, the study Investigators, and a physician who is a subject matter expert not affiliated with the study or Sponsor, will review the safety data of the first study participant through Day 8. Contingent upon this review, the Sponsor will decide if additional study participants can begin receiving INZ-701. After the second study participant completes Day 32, the DRC will perform a cumulative review of safety and PK/PD data and will make dosing recommendations, for example, modifying the dose of the ongoing study participants and/or changing the starting dose for future participants. Dose Level A: 0.2 mg/kg twice weekly Dose Level B: 0.6 mg/kg twice weekly Dose Level C: 0.2 mg/kg once weekly Dose Level D: 0.6 mg/kg once weekly Dose Level E: 1.8 mg/kg once weekly |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
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| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. | 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. | 52 weeks (Treatment Period) |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 52 weeks (Treatment Period) |
| Area under the Plasma Concentration versus Time Curve (AUC) of INZ-701 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Trial Specialist | Contact | +1 800-983-4587 | medinfo@bmrn.com | |
| Medical Director, MD | Contact | +1 800-983-4587 | medinfo@bmrn.com |
| Name | Affiliation | Role |
|---|---|---|
| Medical Director, MD, MD | BioMarin Pharmaceutical | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Rady Children's Hospital | Recruiting | San Diego | California | 92123 | United States |
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Study INZ701-104 is a Phase 1b, open-label study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of INZ-701 in infant study participants with ENPP1 Deficiency or with ABCC6 Deficiency.
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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. |
| 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. | 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. | 52 weeks (Treatment Period) |
| Boston Children's Hospital | Withdrawn | Boston | Massachusetts | 02115 | United States |
| Nationwide Children's Hospital | Recruiting | Columbus | Ohio | 43205 | United States |
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| The Children's Hospital of Philadelphia | Recruiting | Philadelphia | Pennsylvania | 19104 | United States |
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| The University of Utah | Withdrawn | Salt Lake City | Utah | 84108 | United States |
| Hospital Sant Joan de Déu | Terminated | Barcelona | Spain |
| Royal Manchester Children's Hospital | Terminated | Manchester | M13 9WL | United Kingdom |
| ID | Term |
|---|---|
| C562792 | Hypophosphatemic Rickets, Autosomal Recessive, 1 |
| C537440 | Arterial calcification of infancy |
| D011561 | Pseudoxanthoma Elasticum |
| ID | Term |
|---|---|
| D020141 | Hemostatic Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D006474 | Hemorrhagic Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D012868 | Skin Abnormalities |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012871 | Skin Diseases |
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