The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency (ENERGY)
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| ClinicalTrials.gov Identifier: NCT05734196 |
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Recruitment Status :
Recruiting
First Posted : February 17, 2023
Last Update Posted : April 22, 2024
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency Autosomal Recessive Hypophosphatemic Rickets Generalized Arterial Calcification of Infancy | Drug: INZ-701 | Phase 1 |
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 INZ701-104 (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.
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 study participants may continue to receive INZ-701, and an End of Treatment (EOT) visit 30 days after the last dose of INZ-701. Upon treatment discontinuation, study participants may continue to be followed for their ongoing disposition for survival outcome at least quarterly, if feasible through the end of the study. If the study site is not able to complete the survival outcome follow-up for up to 26 weeks post-treatment discontinuation, then the study participant can be considered "lost to follow up.
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 8 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Intervention Model Description: | 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. |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | The ENERGY Study: An Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Infant Subjects With Ectonucleotide Pyrophosphatase/ Phosphodiesterase 1 (ENPP1) Deficiency |
| Actual Study Start Date : | May 25, 2023 |
| Estimated Primary Completion Date : | March 1, 2025 |
| Estimated Study Completion Date : | April 1, 2025 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: INZ-701
The first 2 study participants will receive a single 0.2 mg/kg dose of INZ-701 on Day 1. On Day 8, they will commence receiving Dose Level A (0.2 mg/kg twice weekly). 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 to Dose Levels B, C, D, E, or F. Each study participant's safety and PK/PD data will also be reviewed by the DRC during its quarterly review, based upon which the participant's dose may be modified to Dose Levels B, C, D, E, or F as specified in the protocol. 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 Dose Level F: 3.0 mg/kg once weekly |
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.
Other Name: (rhENPP1-Fc). |
- Number of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: 52 weeks (Treatment Period) ]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.
- Incidence of Anti-Drug Antibodies (ADA) [ Time Frame: 52 weeks (Treatment Period) ]For each participant, the presence of ADAs will be assessed and, if present, further evaluation will determine specificity and subtypes.
- Left Ventricular Ejection Fraction [ Time Frame: 52 weeks (Treatment Period) ]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.
- Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels [ Time Frame: 52 weeks (Treatment Period) ]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.
- Area under the Plasma Concentration versus Time Curve (AUC) of INZ-701 [ Time Frame: 52 weeks (Treatment Period) ]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.
- Maximum Plasma Concentration (Cmax) of INZ-701 [ Time Frame: 52 weeks (Treatment Period) ]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.
- ENPP1 Activity [ Time Frame: 52 weeks (Treatment Period) ]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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | up to 1 Year (Child) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Caregiver(s) must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, per International Conference on Harmonisation (ICH) Good Clinical Practice (GCP).
- Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency with biallelic mutations (ie, homozygous or compound heterozygous) performed by a College of American Pathologists/Clinical Laboratory Improvement Amendments (CAP/CLIA) certified laboratory or local equivalent.
- Study participant must be male or female from birth (newborn) to <1 year of age at Baseline (Day 1).
- Study participant must weigh ≥ 0.5 kg at the time of the first dose of INZ-701
- In the opinion of the Investigator, the subject must be able to complete all aspects of the study
- Study participant's caregiver(s) must agree to provide access to their child's relevant medical records
Exclusion Criteria:
- In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality (outside of those considered associated with the diagnosis of ENPP1 Deficiency) that precludes study participation or may confound interpretation of study results, including known uncontrolled thyroid disease or unrelated connective tissue, bone, mineral, or muscle disease
- Care has been withdrawn or subject is receiving end of life care or hospice only
- Known malignancy
- Known intolerance to INZ-701 or any of its excipients
- Concurrent participation in another non-Inozyme interventional study and/or receipt of any other investigational new drug within 5 half-lives of the last dose of the other investigational product or from 4 weeks prior to the first dose of INZ-701, whichever is longer, or use of an investigational device, through completion of participation in the study
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05734196
| Contact: Inozyme Clinical Trial Information | +1 857 330 4340 | clinicaltrials@inozyme.com |
| United States, California | |
| Rady Children's Hospital | Recruiting |
| San Diego, California, United States, 92123 | |
| Contact: Nathaly Sweeney, MD 858-966-5818 nsweeney@rchsd.org | |
| Contact: Sarah Lazar, MPH 8585761700 slazar@health.ucsd.edu | |
| United States, Massachusetts | |
| Boston Children's Hospital | Recruiting |
| Boston, Massachusetts, United States, 02115 | |
| Contact: Alayna Dutcher 617-355-0741 Alayna.dutcher@childrens.harvard.edu | |
| Contact: Andrea Hale, RN, MPH 617-919-2867 andrea.hale@childrens.harvard.edu | |
| United States, Ohio | |
| Nationwide Children's Hospital | Recruiting |
| Columbus, Ohio, United States, 43205 | |
| Contact: Bimal Chaudhari, MD 614-722-3535 bimal.chaudhari@nationwidechildrens.org | |
| Contact: Marina Artemova, PhD 614-722-2655 marina.artemova@nationwidechildrens.org | |
| United States, Pennsylvania | |
| The Children's Hospital of Philadelphia | Recruiting |
| Philadelphia, Pennsylvania, United States, 19104 | |
| Contact: Maximilian Krumpholz 267-432-0511 krumpholm1@chop.edu | |
| Contact: Rachel Walega 267-586-5969 WALEGAR1@chop.edu | |
| United States, Utah | |
| The University of Utah | Recruiting |
| Salt Lake City, Utah, United States, 84108 | |
| Contact: Carrie Bailey, BS 801-587-3605 ped_mgr@lists.hsc.utah.edu | |
| United Kingdom | |
| Royal Manchester Children's Hospital | Recruiting |
| Manchester, United Kingdom, M13 9WL | |
| Contact: Amish Chinoy, MD 0161 701 7901 amish.chinoy@mft.nhs.uk | |
| Contact: Victoria Hamilton, RN 0161 701 8313 victoria.hamilton@mft.nhs.uk | |
| Study Director: | Alex Lai, MD | Inozyme Pharma |
| Responsible Party: | Inozyme Pharma |
| ClinicalTrials.gov Identifier: | NCT05734196 |
| Other Study ID Numbers: |
INZ701-104 |
| First Posted: | February 17, 2023 Key Record Dates |
| Last Update Posted: | April 22, 2024 |
| Last Verified: | April 2024 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
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ectonucleotide pyrophosphatase/phosphodiesterase1 deficiency hypopyrophosphatemia ENPP1 Generalized Arterial Calcification of Infancy |
GACI Autosomal Recessive Hypophosphatemic Rickets Type 2 ARHR2 |
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Rickets Rickets, Hypophosphatemic Familial Hypophosphatemic Rickets Calcinosis Vascular Calcification Calcium Metabolism Disorders Metabolic Diseases Bone Diseases, Metabolic Bone Diseases Musculoskeletal Diseases Vitamin D Deficiency Avitaminosis Deficiency Diseases Malnutrition |
Nutrition Disorders Hypophosphatemia Phosphorus Metabolism Disorders Hypophosphatemia, Familial Renal Tubular Transport, Inborn Errors Kidney Diseases Urologic Diseases Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Urogenital Diseases Male Urogenital Diseases Metal Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn |