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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06046820




Registration number
NCT06046820
Ethics application status
Date submitted
28/08/2023
Date registered
21/09/2023
Date last updated
1/05/2025

Titles & IDs
Public title
The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency
Scientific title
The ENERGY 3 Study: A Randomized, Controlled, Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Children With Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency
Secondary ID [1] 0 0
INZ701-106
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Ectonucleotide Pyrophosphatase/Phosphodiesterase1 Deficiency 0 0
Autosomal Recessive Hypophosphatemic Rickets 0 0
Generalized Arterial Calcification of Infancy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Diet and Nutrition 0 0 0 0
Other diet and nutrition disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - INZ-701
Treatment: Drugs - Control Arm (Conventional Therapy)

Experimental: 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 and the Open-label Extension Period.

Active comparator: 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.


Treatment: Drugs: 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.

Treatment: Drugs: 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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) concentration through Week 52
Timepoint [1] 0 0
52 weeks (Baseline through Week 52)
Secondary outcome [1] 0 0
Change from Baseline in skeletal abnormalities as measured by the Radiographic Global Impression of Change (RGI-C) global score through Week 52
Timepoint [1] 0 0
Baseline, Week 26, Week 52
Secondary outcome [2] 0 0
Change from Baseline in rickets as measured by Rickets Severity Score (RSS) total score through Week 52
Timepoint [2] 0 0
Baseline, Week 26, Week 52
Secondary outcome [3] 0 0
Change from Baseline in growth Z-score (height/body length and weight) through Week 52
Timepoint [3] 0 0
Baseline, Day 29, Week 8, Week 13, Week 26, Week 39, Week 52
Secondary outcome [4] 0 0
Area under the Plasma Concentration versus Time Curve (AUC) of INZ-701
Timepoint [4] 0 0
52 weeks (Randomized Treatment Period)
Secondary outcome [5] 0 0
Maximum Plasma Concentration (Cmax) of INZ-701
Timepoint [5] 0 0
52 weeks (Randomized Treatment Period)
Secondary outcome [6] 0 0
Change from Baseline in ENPP1 activity (µM/min) through week 52
Timepoint [6] 0 0
52 weeks (Randomized Treatment Period)

Eligibility
Key inclusion criteria
Inclusion Criteria

Study participants must meet all of the following inclusion criteria:

1. Caregiver's written or electronic 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-hydroxyvitamin D (25[OH]D) levels of =12 ng/mL at Screening
8. Radiographic evidence of skeletal abnormalities based on an RSS =2
9. Female participants of childbearing potential must have a negative serum pregnancy test at Screening and must not be breastfeeding
10. Study participants of childbearing potential who are sexually active must agree to use a highly effective form of contraception in accordance with Clinical Trials Facilitation and Coordination Group (CTFG) guidance and local guidelines for the duration of the study
11. In the opinion of the Investigator, able to complete all aspects of the study
Minimum age
1 Year
Maximum age
12 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
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-fibroblast growth factor 23 (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 that may confound the interpretation of study results during the 52-week Randomized Treatment Period
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 Randomization, whichever is longer, or use of an investigational device

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
5/11/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/02/2026
Actual
Sample size
Target
Accrual to date
Final
27
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment postcode(s) [1] 0 0
4101 - South Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Colorado
Country [2] 0 0
United States of America
State/province [2] 0 0
Illinois
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
Ohio
Country [5] 0 0
United States of America
State/province [5] 0 0
Pennsylvania
Country [6] 0 0
United States of America
State/province [6] 0 0
Texas
Country [7] 0 0
Canada
State/province [7] 0 0
Montréal
Country [8] 0 0
France
State/province [8] 0 0
Le Kremlin-Bicêtre
Country [9] 0 0
Saudi Arabia
State/province [9] 0 0
Riyadh
Country [10] 0 0
Spain
State/province [10] 0 0
Barcelona
Country [11] 0 0
Turkey
State/province [11] 0 0
Istanbul
Country [12] 0 0
Turkey
State/province [12] 0 0
Sariçam
Country [13] 0 0
United Arab Emirates
State/province [13] 0 0
Dubai
Country [14] 0 0
United Kingdom
State/province [14] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Inozyme Pharma
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Kurt Gunter, MD
Address 0 0
Inozyme Pharma, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT06046820