The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Inozyme Pharma Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

24 May 2024 → ongoing

Decision date (initial)

2024-03-11

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Inozyme Pharma, Inc.

External identifiers

EU CT number

2023-507382-26-00

ClinicalTrials.gov

NCT06046820

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Safety, Therapy, Efficacy

To determine if INZ-701 increases PPi levels
To determine if INZ-701 improves skeletal abnormalities as measured by the RGI-C

Secondary objectives 4

1. To determine if INZ-701 improves rickets as measured by the Rickets Severity Score (RSS)
2. To determine if INZ-701 increases growth Z-score of height/body length and weight
3. To characterize the PK and ENPP1 activity of INZ-701
4. Please refer to the protocol for Tertiary and Safety objectives

Conditions and MedDRA coding

Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency

Study design 4 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-003232-PIP01-22

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 12

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 contraception (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 8

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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Change from Baseline in plasma PPi concentration through Week 52
2. RGI-C global score through Week 52

Secondary endpoints 4

1. Change from Baseline in RSS total score through Week 52
2. Change from Baseline in growth Z-score (height/body length and weight) through Week 52
3. Measurement of INZ-701 serum concentration and enzyme activity
4. Please refer to the protocol for Tertiary and Safety endpoints

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Inozyme Pharma Inc.

Sponsor organisation

Inozyme Pharma Inc.

Address

321 Summer Street

City

Boston

Postcode

02210-1725

Country

United States

Scientific contact point

Organisation

Inozyme Pharma Inc.

Contact name

Jack Brownrigg, MBChB, PhD

Public contact point

Organisation

Inozyme Pharma Inc.

Contact name

Clinical Trial Information

Third parties 13

Locations

2 EU/EEA countries · 2 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 94 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

10 submissions — initial application plus substantial / non-substantial modifications