A Long-term Follow-up Study of Participants who Received INZ-701 in a previous clinical study

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Inozyme Pharma Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

Trial duration

8 Oct 2024 → ongoing

Decision date (initial)

2024-10-04

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Inozyme Pharma Inc.

External identifiers

EU CT number

2024-512715-42-00

ClinicalTrials.gov

NCT06462547

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

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

To assess the long-term safety of INZ-701

Secondary objectives 2

1. To confirm drug levels of INZ-701 with long-term use
2. To assess the effect of long-term INZ-701 treatment on plasma PPi concentrations

Conditions and MedDRA coding

Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency and ATP-binding cassette sub-family C member 6 (ABCC6) Deficiency

Study design 3 periods

Regulatory references

Scientific advice from competent authorities

Medicines And Healthcare Products Regulatory Agency, Agence Nationale De Sante Publique, European Medicines Agency, Federal Institute For Drugs And Medical Devices, Medicines Evaluation Board

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 8

1. Provide written or electronic informed consent after the nature of the study has been explained, and prior to any research-related procedures, per International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP)
2. Provide assent in accordance with local regulations, if <18 years of age
3. Male or female, greater than 1 year of age
4. Must have completed the protocol-required safety and PK/PD and/or efficacy period(s) of the INZ-701-101, INZ701-201, INZ701-104, or INZ701-106 clinical studies in ENPP1 or ABCC6 Deficiency, as confirmed by the Sponsor
5. 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
6. Males who are sexually active must agree to use condoms from the period following the first dose of INZ-701 through 30 days after the last dose of INZ-701
7. WOCBP and partners of fertile males who are WOCBP must be using or agree to use a highly effective form of contraception (as per CTCG 2024) 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)
8. In the opinion of the Investigator, able to complete all aspects of the study

Exclusion criteria 5

1. In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality not associated with ENPP1 Deficiency or ABCC6 Deficiency, that will preclude study participation and/or may confound interpretation of study results
2. Known intolerance to INZ-701 or any of its excipients
3. Concurrent participation in another interventional clinical study and/or has received an investigational drug other than INZ-701 within 5 half-lives or within 4 weeks prior to the first dose of INZ-701 in this study, whichever is longer, or use of an investigational device
4. Pregnant, trying to become pregnant, or breastfeeding
5. Male participants trying to father a child

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 4

1. Incidence, frequency, and severity of adverse events (AEs), treatment-emergent adverse event (TEAEs), adverse events of special interest (AESIs), and serious adverse events (SAEs)
2. Changes in clinical laboratory tests including chemistry, hematology, and urinalysis resulting in an AE
3. Concomitant medications
4. Titers of anti-drug antibodies (ADA)

Secondary endpoints 2

1. INZ-701 plasma PK parameters measured by immunoassay and enzymatic activity
2. Plasma PPi concentrations based on a validated assay

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

Kurt C. Gunter, MD, FASCP

Public contact point

Organisation

Inozyme Pharma Inc.

Contact name

Clinical Trial Information

Third parties 11

Locations

2 EU/EEA countries · 3 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 77 files

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

Application history

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