Long-term Follow-up (LTFU) Study of Participants in any iECURE Protocol Using an Investigational Product (IP)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Iecure Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male

Therapeutic area

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

Decision date (initial)

2026-06-18

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

iECURE

External identifiers

EU CT number

2024-514190-21-00

ClinicalTrials.gov

NCT06805695

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Others, Efficacy, Pharmacokinetic

To assess the long-term safety of investigational product (IP) in participants who received IP and the long-term safety of those who never received IP regardless of causality

Secondary objectives 2

1. To assess the pharmacokinetics of IP and clinical outcomes in participants who received IP and those who never received IP and are managed only on available standard of care (SOC)
2. To assess potential effects on disease specific biologic markers, developmental milestones and quality of life (QOL)

Conditions and MedDRA coding

Ornithine Transcarbamylase Deficiency (OTC)

Regulatory references

Scientific advice from competent authorities

Spanish Agency Of Medicines And Medical Devices

Plan to share IPD

Yes

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Enrolled in an iECURE parent protocol and have either completed or discontinued that protocol
2. Participant parent(s)/legal authorized representative (LAR) is willing and able to adhere to the protocol requirements.
3. Consent was obtained by the participants parent(s)/LAR (and participant assent, where applicable), prior to any study-related data being collected.

Exclusion criteria 1

1. None

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. • AE and SAE (incidence, severity, seriousness, and relatedness) • Physical exam (including length/height and weight) • Vital signs • Neurologic exam • Blood safety tests including hematology, serum chemistry, liver function tests, coagulation tests, serum PCSK9 • Urinalysis • 12 lead ECG

Secondary endpoints 12

1. 1. Vector PK in blood and shedding in plasma, saliva, urine and feces for dosed participants
2. 2. Percent liver transduction
3. 3. Number of hyperammonemic crises (HAC)
4. 4. Among participants who experience HAC with associated neurological status change and are hospitalized: a. Daily ammonia levels for duration of hospitalization for each event b. Duration of hospitalization for each event c. Requirement for Intensive Care Unit (ICU) care during hospitalization for each event and total time in ICU for each event
5. 5. Number of HAC with the following severities: a. Mild: adjustment of dietary protein intake and oral scavenger medication b. Moderate: cessation of dietary protein intake and initiation of IV scavenger therapy c. Severe: requirement for hemodialysis
6. 6. Scavenger drug dose per body surface area (BSA)
7. 7. Protein allowance g/kg
8. 8. Blood urea nitrogen measurements
9. 9. Time to liver transplant from Day 1 (Parent Protocol) for participants who are dosed and Day -1 (Parent Protocol) for participants who are not dosed to end of study EOS (LTFU Protocol)
10. 10. Transplant free survival a. Time to liver transplant or any-cause death from dosing to EOS
11. 11. Overall survival a. Time to any-cause death from dosing to EOS. Survival measured from Day 1 (Parent Protocol) for participants who are dosed and Day -1 (Parent Protocol) for participants who are not dosed to end of study EOS (LTFU Protocol).
12. • Antibody response to AAV capsid, and to nuclease and therapeutic transgenes • Plasma ammonia, citrulline and glutamine • Urinary nitrogen to urinary creatinine ratio • Future DNA analysis of WBCs collected in a parent trial may be performed only in the event of an observed genomic safety signal • Developmental assessments as measured by age-appropriate Bayley Scale of Infant Development IV (BSID-IV) and Kaufman Assessment Battery for Children – Second Edition Normative Update (KABC-II NU)

Investigational products

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

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Iecure Inc.

Sponsor organisation

Iecure Inc.

Address

1777 Sentry Parkway West Suite 200 Building 14

City

Blue Bell

Postcode

19422-2211

Country

United States

Scientific contact point

Organisation

Iecure Inc.

Contact name

Therapeutic Area Lead - Urea Cycle Disorders

Public contact point

Organisation

Iecure Inc.

Contact name

iECURE - Regulatory Affairs

Third parties 2

Locations

2 EU/EEA countries · 3 investigational sites

By country

Investigational sites

Results and documents

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

Documents 11 files

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

Application history

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