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Het ENERGY 3-onderzoek: evaluatie van de werkzaamheid en veiligheid van INZ-701 bij kinderen met ENPP1-deficiëntie

28 september 2026 bijgewerkt door: Inozyme Pharma

Het ENERGY 3-onderzoek: een gerandomiseerde, gecontroleerde, open-label fase 3-studie om de werkzaamheid en veiligheid van INZ-701 te evalueren bij kinderen met ectonucleotidepyrofosfatase/fosfodiesterase 1 (ENPP1)-deficiëntie

The primary purpose of Study INZ701-106 (The ENERGY 3 Study) is to assess the efficacy and safety of INZ-701 in children with ENPP1 Deficiency.

Studie Overzicht

Toestand

Beëindigd

Conditie

Interventie / Behandeling

Gedetailleerde beschrijving

INZ-701 is an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy (ERT) in development for the treatment of ENPP1 Deficiency, an ultra-rare genetic disorder with an incidence of 1 in 64,000 pregnancies.

Study INZ701-106 (The ENERGY 3 Study) is a multi-center, randomized in a 2:1 ratio, controlled, open-label Phase 3 study to evaluate the efficacy and safety of INZ-701 in children with ENPP1 Deficiency.

The study will consist of a Screening Period of up to 52 days (including a washout period of up to 7 days for prohibited medications post-Randomization) and a Randomized Treatment Period (INZ-701 or control) of 52 weeks, followed by an Open-label Extension Period during which all study participants may receive INZ-701, and an End of Study (EOS) Safety visit 30 days after the last dose of INZ-701.

Studietype

Ingrijpend

Inschrijving (Werkelijk)

27

Fase

  • Fase 3

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

  • Australië
      • South Brisbane, Australië, 4101
        • Queensland Children's Hospital
  • Canada
      • Montreal, Canada, H3T 1C5
        • Centre Hospitalier Universitaire (CHU) Sainte-Justine
  • Frankrijk
      • Le Kremlin-Bicêtre, Frankrijk, 94270
        • Hôpital Bicêtre, Service d'endocrinologie et diabète de l'enfant (Childhood Endocrinology and Diabetes Department)
  • Saoedi-Arabië
      • Riyadh, Saoedi-Arabië, 12713
        • King Faisal Specialist Hospital and Research Centre
  • Turkije (Türkiye)
      • Istanbul, Turkije (Türkiye), 34764
        • Umraniye Training and Research Hospital
      • Sarıçam, Turkije (Türkiye), 01330
        • Cukurova Universitesi Tip Fakultesi
  • Verenigd Koninkrijk
      • Manchester, Verenigd Koninkrijk, M13 9WL
        • Royal Manchester Children's Hospital
  • Verenigde Arabische Emiraten
      • Dubai, Verenigde Arabische Emiraten, 30726
        • Al Jalila Children's Specialty Hospital
  • Verenigde Staten
    • Illinois
      • Chicago, Illinois, Verenigde Staten, 60611
        • Ann & Robert H. Lurie Children's Hospital
    • Pennsylvania
      • Philadelphia, Pennsylvania, Verenigde Staten, 19104
        • The Children's Hospital of Philadelphia
    • Texas
      • Fort Worth, Texas, Verenigde Staten, 76104
        • Cook Children's Medical Center

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

1 jaar tot 12 jaar (Kind)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

Inclusion Criteria

Study participants must meet all of the following inclusion criteria:

  1. Caregiver's written informed 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)
  2. Study participant's assent in accordance with local regulations
  3. A confirmed postnatal 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 regional equivalent
  4. Males and females ≥1 year and <13 years of age at Study Day 1
  5. Open growth plates of the distal femur and proximal tibia in both legs
  6. Plasma PPi concentration of <1400 nM at Screening
  7. 25(OH)D levels of ≥12 ng/mL at Screening
  8. Radiographic evidence of skeletal abnormalities based on an RSS ≥2
  9. Women of childbearing potential (WOCBP, as defined in Clinical Trials Coordination Group [CTCG 2024]) must have a negative serum pregnancy test at Screening and must not be breastfeeding
  10. Males who are sexually active must agree to use condoms from the period following first dose of INZ-701 through 30 days after the last dose of INZ-701
  11. WOCBP and partners of fertile males who are WOCBP must be using or must agree to use a highly effective form of (as per CTCG) from at least 1 month before the first dose of INZ-701 through 30 days after the last dose of INZ-701 (greater than 5 half-lives of INZ-701)
  12. In the opinion of the Investigator, able to complete all aspects of the study

Exclusion Criteria

Study participants meeting any of the following exclusion criteria will not be eligible to participate in the study:

  1. In the opinion of the Investigator, has clinically significant disease or laboratory abnormality not associated with ENPP1 Deficiency that will preclude study participation and/or may confound the interpretation of study results
  2. If receiving any of the following prohibited medications as indicated in the protocol: systemic corticosteroids (>5 mg prednisone equivalent per day), anti-FGF23 and oral and/or IV bisphosphonates
  3. Unable or unwilling to discontinue calcitriol or other active forms of vitamin D3 (or analogs) within 7 days prior to Study Day 1 and/or oral phosphate supplements within 36 hours prior to Study Day 1 if randomized to the INZ-701 arm
  4. Planned orthopedic surgery or other procedures that may confound the interpretation of study results during the 52-week RTP
  5. Known intolerance to INZ-701 or any of its excipients
  6. A positive COVID-19 test within 5 days prior to Randomization, only if required as per local regulations or institutional policy
  7. Previous treatment with INZ-701
  8. Concurrent participation in another interventional clinical study and/or has received an investigational drug within 5 half-lives of the last dose or within 4 weeks prior to the first dose of INZ-701, whichever is longer, or use of an investigational device

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: INZ-701

Subjects randomized to the INZ-701 arm will be administered a 2.4 mg/kg once weekly dose by subcutaneous (SC) injection for the duration of the 52-week Randomized Treatment Period (RTP). RTP will be followed by a Low-dose Open-label Extension Period during which all study participants will receive INZ-701 at a dose of 2.4 mg/kg QW while the primary analysis is pending. If dose escalation criteria are met, all participants will transition to the Randomized Extension Period (REP) where participants who were originally randomized to INZ-701 during RTP will transition to a higher dose regimen of INZ-701 at 1.8 mg/kg twice weekly. Participants who were originally randomized to the active control arm during RTP will continue receiving INZ-701 at 2.4 mg/kg QW in the REP.
Geneesmiddel: 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.

Andere namen:
  • (rhENPP1-Fc).
Actieve vergelijker: Control Arm (Conventional Therapy)

Subjects randomized to the control arm will continue taking their conventional therapy as clinically indicated by their treating physician for the duration of the 52-week Randomized Treatment Period.
Geneesmiddel: Control Arm (Conventional Therapy)

Conventional therapy is defined as oral phosphate supplements and calcitriol or other active forms of vitamin D3 (or analogs). No other agents for treatment of ENPP1 Deficiency are allowed in the control arm.

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) concentration through Week 52
Tijdsspanne: 52 weeks (Baseline through Week 52)

For each subject, plasma PPi will be measured via a series of blood samples obtained throughout the study, comparing the subject's baseline value over time.
52 weeks (Baseline through Week 52)
Change from Baseline in skeletal abnormalities as measured by the Radiographic Global Impression of Change (RGI-C) global score through Week 52
Tijdsspanne: 52 weeks (Baseline through Week 52)

The RGI-C is an overall radiographic score which can be used to monitor response to a therapeutic intervention comparing scores from 2 time points. A determination of healing on a scale of 0 to +3 with 0 being no change or healing and +3 being complete healing; worsening is also measured on a scale of 0 to -3 with 0 being no change and -3 being severe worsening.
52 weeks (Baseline through Week 52)

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Change from Baseline in rickets as measured by Rickets Severity Score (RSS) total score through Week 52
Tijdsspanne: Baseline, Week 26, Week 52

The RSS assesses rickets severity by utilizing a scoring system that uses a scale from 0 to 4 for the wrists and 0 to 6 for the knees, to generate a total score of 0 to 10, where 0 is normal and 10 is the worst score possible ie, most severe skeletal abnormalities observed radiographically.
Baseline, Week 26, Week 52
Change from Baseline in growth Z-score (height/body length and weight) through Week 52
Tijdsspanne: Baseline, Day 29, Week 8, Week 13, Week 26, Week 39, Week 52

A Z-score represents the degree to which that particular measurement for that individual differs from the reference value in the general population.

(height/body length and weight) through Week 52
Baseline, Day 29, Week 8, Week 13, Week 26, Week 39, Week 52
Area under the Plasma Concentration versus Time Curve (AUC) of INZ-701
Tijdsspanne: 52 weeks (Randomized Treatment Period)

For each subject, variation of concentration of INZ-701 in the plasma will be measured via a series of blood samples obtained throughout the study, comparing the subject's baseline value over time.
52 weeks (Randomized Treatment Period)
Maximum Plasma Concentration (Cmax) of INZ-701
Tijdsspanne: 52 weeks (Randomized Treatment Period)

For each subject, the maximum concentration of INZ-701 in the plasma will be measured via a series of blood samples obtained throughout the study, comparing the subject's baseline value over time.
52 weeks (Randomized Treatment Period)
Change from Baseline in ENPP1 activity (µM/min) through week 52
Tijdsspanne: 52 weeks (Randomized Treatment Period)

For each subject, the activity of INZ-701 (µM/min) in the serum will be assessed through hydrolysis of a substrate to the enzyme, via a series of blood samples obtained throughout the study, comparing the subject's baseline value over time.
52 weeks (Randomized Treatment Period)

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Inozyme Pharma

Medewerkers

BioMarin Pharmaceutical

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

5 november 2023

Primaire voltooiing (Werkelijk)

29 januari 2026

Studie voltooiing (Werkelijk)

19 augustus 2026

Studieregistratiedata

Eerst ingediend

28 augustus 2023

Eerst ingediend dat voldeed aan de QC-criteria

18 september 2023

Eerst geplaatst (Werkelijk)

21 september 2023

Updates van studierecords

Laatste update geplaatst (Werkelijk)

1 oktober 2026

Laatste update ingediend die voldeed aan QC-criteria

28 september 2026

Laatst geverifieerd

1 september 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • INZ701-106

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Ja

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

product vervaardigd in en geëxporteerd uit de V.S.

Nee

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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