The ENERGY Study: Evaluation of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Infants with ENPP1 Deficiency or ATP-binding Cassette Sub-family C Member 6 (ABCC6) 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

21 Oct 2024 → 10 Apr 2025

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

2023-507384-20-00

ClinicalTrials.gov

NCT05734196

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Pharmacodynamic, Safety, Pharmacokinetic, Therapy, Dose response

To assess the safety and tolerability of INZ-701

Secondary objectives 2

1. To study the PK and PD activity of INZ-701
2. Please refer to the protocol for Exploratory objectives

Conditions and MedDRA coding

Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency or ATP-binding Cassette Sub-family C Member 6 (ABCC6) Deficiency

Study design 1 period

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration, 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 7

1. Caregiver(s) must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, following International Council for Harmonisation (ICH) Good Clinical Practice (GCP).
2. Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency with biallelic mutations (ie, homozygous or compound heterozygous) performed using an assay that meets CE-marked requirements, or by a College of American Pathologists/Clinical Laboratory Improvement Amendments (CAP/CLIA) certified laboratory or a local equivalent
3. Study participant must be male or female from birth to <1 year of age at Baseline (Day 1)
4. Study participant must weigh ≥0.5 kg at the time of the first dose of INZ-701 in this study
5. In the opinion of the Investigator, the study participant must be able to complete all aspects of the study
6. Study participant’s caregiver(s) must agree to provide access to their child’s relevant medical records
7. Study participants must have clinical manifestations of GACI or GACI-2, which may include, but are not limited to, pathologic ectopic calcification, heart failure, respiratory distress, edema, cyanosis, hypertension, and cardiomegaly

Exclusion criteria 6

1. 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 or ABCC6 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
2. Care has been withdrawn or subject is receiving end of life care or hospice only
3. Known malignancy
4. Known intolerance to INZ-701 or any of its excipients
5. Concurrent participation in another non-Inozyme interventional study
6. Receipt of any non-Inozyme investigational new drug within 5 half-lives of the last dose of the other investigational product or within 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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 7

1. Adverse events (incidence, frequency, and severity of AEs, TEAEs, AESIs, and SAEs)
2. Vital signs and weight
3. Laboratory tests including chemistry, hematology, and if feasible, urine tests
4. Immunogenicity
5. Concomitant medications
6. Left ventricular ejection fraction via echocardiogram
7. Electrocardiogram

Secondary endpoints 4

1. Measurement of INZ-701 serum concentration-time profiles and PK parameters
2. Change from Baseline over time in PPi levels through Week 52
3. Measurement of ENPP1 activity
4. Please refer to the protocol for Exploratory endpoints

Investigational products

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

Test 2

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

Kurt C. Grunter

Public contact point

Organisation

Inozyme Pharma Inc.

Contact name

Clinical Trial Information

Third parties 10

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

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

Application history

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