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    Summary
    EudraCT Number:2020-004000-33
    Sponsor's Protocol Code Number:INZ701-201
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-04-28
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedFrance - ANSM
    A.2EudraCT number2020-004000-33
    A.3Full title of the trial
    A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 Followed by an Open-Label Long-Term Extension Period in Adults with ABCC6 Deficiency Manifesting as Pseudoxanthoma elasticum (PXE)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of INZ-701 in Adults with ABCC6 Deficiency causing Pseudoxanthoma elasticum (PXE)
    A.4.1Sponsor's protocol code numberINZ701-201
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorInozyme Pharma, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportInozyme Pharma, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationInozyme Pharma, Inc.
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street Address321 Summer Street, Suite 400
    B.5.3.2Town/ cityBoston
    B.5.3.3Post code MA 02210
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1 857 330 4340
    B.5.6E-mailclinicaltrials@inozyme.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameINZ-701
    D.3.2Product code INZ-701
    D.3.4Pharmaceutical form Lyophilisate for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNRECOMBINANT HUMAN ECTONUCLEOTIDE PYROPHOSPHATASE/PHOSPHODIESTERASE 1 FUSED TO THE FC FRAGMENT OF IGG1
    D.3.9.2Current sponsor codeINZ-701
    D.3.9.3Other descriptive nameRECOMBINANT HUMAN ECTONUCLEOTIDE PYROPHOSPHATASE/PHOSPHODIESTERASE 1 FUSED TO THE FC FRAGMENT OF IGG1
    D.3.9.4EV Substance CodeSUB194463
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Treatment of patients with ABCC6 Deficiency Manifesting as Pseudoxanthoma elasticum (PXE)
    E.1.1.1Medical condition in easily understood language
    ABCC6 Deficiency causing a condition called Pseudoxanthoma elasticum
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10037150
    E.1.2Term Pseudoxanthoma elasticum
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Assess the safety and tolerability of multiple ascending subcutaneous (SC) doses of INZ-701
    E.2.2Secondary objectives of the trial
    • Assess the immunogenicity of multiple ascending SC doses of INZ-701.
    • Study the pharmacokinetics (PK) of INZ-701 administered as multiple SC doses.
    • Study the long-term safety and pharmacodynamic (PD) changes in pyrophosphate (PPi) and associated biomarkers of ABBC6 Deficiency after administration of multiple SC doses of INZ-701.

    Exploratory Objectives:

    • Assess changes in calcification of arteries and organs (including the eye).
    • Assess changes in clinical measures associated with underlying ABCC6 Deficiency, including skeletal, cardiac, vascular, hearing, ophthalmologic, and renal parameters.
    • Assess changes in physical function.
    • Assess changes in patient, clinician, and caregiver reported outcomes and quality
    of life.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, following International Conference on Harmonisation (ICH) Good Clinical Practice (GCP).
    2. Clinical diagnosis of PXE supported by prior or concurrent genetic identification of biallelic Abcc6 mutations
    3. Male or female, ages 18 to <65 years of age at Screening
    4. Plasma PPi < lower limit of normal (LLN) at screening
    5. Women of child-bearing potential (WOCBP as defined in CTFG 2014) must have a negative serum pregnancy test at Screening and negative urine pregnancy tests at all additional pregnancy tests during the study.
    6. WOCBP and partners of fertile males who are WOCBP must agree to use 2 highly effective forms of contraception (per CTFG 2014) from at least 1 month before the first dose of INZ-701 through 30 days after last dose of INZ-701 (greater than 5 half-lives of INZ-701). WOCBP and partners of fertile males who are WOCBP must also agree to not donate ova from the period following the first dose of INZ-701 through 30 days after last dose of INZ-701
    7. 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. Males must also agree to not donate sperm from the period following the first dose of INZ-701 through 30 days after last dose of INZ-701.
    8. In the opinion of the Investigator, must be willing and able to complete all aspects of the study
    9. Agree to provide access to relevant medical records
    E.4Principal exclusion criteria
    1. In the opinion of the Investigator and/or Sponsor, presence of any clinically significant disease (outside of those considered associated with the diagnosis of ABCC6 Deficiency) that precludes study participation or may confound interpretation of study results, including uncontrolled thyroid disease or unrelated connective tissue, bone, mineral, ophthalmologic, or muscle disease
    2. Advanced eye disease requiring anti-VEGF treatment at Screening
    3. Clinically significant abnormal laboratory result at screening, including but not limited to elevations of aspartate aminotransferase, alanine aminotransferase, bilirubin, or creatinine greater than 2 times the upper limit of normal, 25 (OH) Vitamin D levels <20 ng/mL, parathyroid hormone (PTH) more than 20% above the upper limit of normal, and hyper- or hypocalcemia
    4. Known active fungal, bacterial, and/or viral infection including human immunodeficiency virus, hepatitis B virus, hepatitis C virus, or COVID-19 virus. A negative COVID-19 test result is required within 5 days of first dose of INZ-701
    5. Known intolerance to any INZ-701 excipient
    6. Unable or unwilling to discontinue the use of any prohibited medication (examples include bisphosphonates, calcimimetics, antacids, systemic corticosteroids, pyrophosphate containing medications), as provided in protocol Section 9.4
    7. Receipt of any other investigational new drug within 5 half-lives of the last dose of the other investigational drug 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
    8. Last symptoms from a COVID-19 vaccination within 14 days prior to the first dose of INZ-701 or as described in the Inozyme COVID-19 Vaccine Guidance Document
    9. Women who are breastfeeding
    E.5 End points
    E.5.1Primary end point(s)
    Safety and Immunogenicity:

    Safety assessments will be summarized at Baseline and at each observed time point. Safety variables include:
    • Changes from baseline in the terminal elimination half-life (clearance) of INZ701
    • Changes from baseline in the ENPP1 enzyme activity and mass ratio (neutralizing anti-drug antibodies [ADA])
    • Incidence, frequency, and severity of adverse events (AEs), treatment-related AEs, and serious adverse events (SAEs)
    • Vital signs and weight
    • Physical examinations
    • Estimated glomerular filtration rate (eGFR)
    • Laboratory tests including chemistry, hematology, and urinalysis, including additional biochemical parameters of interest
    • Anti-INZ-701 antibody testing and dose-limiting toxicities (DLTs)
    • Incidence of any anti-drug antibodies (ADA)
    • Incidence of treatment emergent adverse events (TEAEs) associated with hypersensitivity reactions
    • Concomitant medications
    • Electrocardiogram (ECG)

    Pharmacokinetic Endpoints:

    • INZ-701 plasma concentration-time profiles and determination of noncompartmental PK parameters (including Tmax, Cmax, AUClast, AUCtau, AUCinf, T1/2, Cmin, Cl/F, and V/F)
    • Assess linearity between ascending INZ-701 SC doses and PK parameters

    Pharmacodynamic Endpoints:

    • Change from baseline for plasma PPi levels
    • Changes from baseline for renal clearance of PPi, Pi, calcium normalized to blood and urine creatinine
    • Changes from baseline for blood and urine biomarkers
    − Serum 1,25(OH)2D, plasma ionized and total calcium, parathyroid hormone
    − Bone biomarkers: serum alkaline phosphatase (ALP), bone specific alkaline phosphatase (BALP), carboxy terminal cross-linked telopeptide of type I collagen (CTX), and procollagen type 1 N-terminal propeptide (P1NP), serum phosphate, plasma intact fibroblast growth factor 23 (FGF23), tubular maximum reabsorption rate of phosphate adjusted for glomerular filtration rate(TmP/GFR)

    Efficacy Endpoints:

    Skeletal, calcification, and hearing evaluations that were conducted within 2 months of enrollment can be used for Baseline if no prohibited drugs, as described in Protocol Section 9.4, were used during this period prior to enrollment and if conducted consistent with the protocol
    • Skeletal
    − Na18F-PET/CT
    − X-rays
    • Arterial and organ calcification
    − Na18F-PET/CT
    − Echocardiogram
    − Renal ultrasound
    • Ophthalmological
    − Angioid streaks, choroidal and subretinal neovascularization, and breaks in Bruch’s membrane as assessed by optical coherence tomography (OCT), color fundus photography, and autofluorescence photography
    − Best corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study letter charts
    − Dark Adaptation
    • Cardiovascular and peripheral vascular reactivity function
    − Echocardiogram
    − ECG
    − Ankle-brachial pressure index (ABI)
    − Pulse Wave Velocity (PWV)
    − Carotid ultrasound
    • Neurological function
    − Neurological exam
    • Pulmonary function
    − Spirometry
    • Performance outcomes
    − 2-minute walk test (2MWT), 6-minute walk test (6MWT)
    − Handheld dynamometry, grip strength, range of motion
    − Hearing tests: Physical exam and otoscopy, immittance audiometry (tympanometry), Pure Tone Audiometry (PTA), High Frequency Audiometry (HFA)
    • Baseline patient, clinician, and caregiver outcomes
    − Patient, Clinician, Caregiver Global Impression scales
    − Patient Reported Outcomes Measurement Information System (PROMIS) Pain Interference, Pain Intensity, Fatigue, Cognitive Function, and Physical Function - Upper Extremity
    − Vision Function Questionnaire
    − Work Productivity and Activity Impairment Questionnaire (WPAI)
    − Edinburgh Claudication Questionnaire (ECQ)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety assessments will be summarized at Baseline and at each observed time point. All assessments for Efficacy are collected at Baseline and at timepoints noted in the Schedules of Events.
    E.5.2Secondary end point(s)
    Please see above.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Please see above.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Tolerability of multiple ascending doses.
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA2
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 9
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 6
    F.4.2.2In the whole clinical trial 9
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    The Extension Period may be extended upon the decision of the Sponsor and approval of regulatory authorities.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-06-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-06-10
    P. End of Trial
    P.End of Trial StatusOngoing
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