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

iECURE, Inc.13 enrolled

Overview

This LTFU is being conducted to assess long-term safety and durability of response in participants dosed with IP in a parent protocol, and to collect longitudinal natural history in enrolled but not dosed participants who also participated in a parent protocol.

This is a 14.5 year LTFU protocol to assess long term safety and clinical response to IP as well as to capture natural disease history of participants not dosed with IP in a parent protocol. No IP is provided as part of this protocol. Participants will remain on standard of care medication as indicated and prescribed by their physicians.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Ornithine Transcarbamylase Deficiency Ornithine Transcarbamylase Deficiency Disease Urea Cycle Disorders, Inborn

Interventions

No InterventionOTHER

No Intervention

Eligibility

Sex: ALLMin age: 7 MonthsMax age: 15 Months

Medical Language ↔ Plain English

Inclusion Criteria: 1. Enrolled in an iECURE parent protocol and have either completed or discontinued that protocol 2. Participant parent(s)/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\. Participants who enroll into an interventional drug or gene therapy clinical trial utilizing an IP other than the IP provided in the parent protocol will be excluded from this protocol.

Locations (1)

Great Ormond Street Hospital

London, United Kingdom

RECRUITING

Outcomes

Primary Outcomes

AE and SAE (incidence, severity, seriousness, and relatedness)

Safety

Time frame: Over 14.5 years post dosing with ECUR-506

Change from baseline over 14.5 years post infusion in length

Length measured in cenitmeters

Time frame: Over 14.5 years post dosing with ECUR-506

Change from baseline over 14.5 years post infusion in weight

Weight measured in kilograms

Time frame: Over 14.5 years post dosing with ECUR-506

Urinalysis (Dip Stick) Evaluations

Urinalysis (Dip Stick) will be evaluated to monitor pathological changes to the participants urine and to monitor levels of Specific Gravity, pH, Glucose, Protein, Blood, Ketones, Bilirubin, Urobilinogen, Nitrite, Leukocyte esterase, red blood cell count, and white blood cell count as they relate to established local lab normal ranges.

Time frame: Over 14.5 years post dosing with ECUR-506

Secondary Outcomes

qPCR measurement to evaluate the clearance of both vectors in blood over time.

Pharmacokinetics

Time frame: Over 14.5 years post dosing with ECUR-506

qPCR measurement to evaluate the clearance of both vectors in saliva over time.

Pharmacokinetics

Time frame: Over 14.5 years post dosing with ECUR-506

qPCR measurement to evaluate the clearance of both vectors in urine over time.

Pharmacokinetics

Time frame: Over 14.5 years post dosing with ECUR-506

Central Contacts

George Diaz, M.D., Ph.D.

CONTACT

1-877-694-3558 medinfo@iecure.com

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

qPCR measurement to evaluate the clearance of both vectors in feces over time.

Pharmacokinetics

Time frame: Over 14.5 years post dosing with ECUR-506

Percent Liver Transduction

Pharmacokinetics

Time frame: Over 14.5 years post dosing with ECUR-506

Number of hyperammonemic crises (HAC)

Pharmacodynamics and Efficacy

Time frame: Over 14.5 years post dosing with ECUR-506

Among participants who experience HAC with associated neurological status change and are hospitalized: Asses daily ammonia levels for duration of hospitalization for each event

Pharmacodynamics and Safety

Time frame: Over 14.5 years post dosing with ECUR-506

Among participants who experience HAC with associated neurological status change and are hospitalized: Assess duration of hospitalization for each event.

Pharmacodynamics and Safety

Time frame: Over 14.5 years post dosing with ECUR-506

Among participants who experience HAC with associated neurological status change and are hospitalized: Assess requirement for ICU care.

Pharmacodynamics and Safety

Time frame: Over 14.5 years post dosing with ECUR-506

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

Pharmacodynamics and Safety

Time frame: Over 14.5 years post dosing with ECUR-506

Scavenger drug dose per body surface area (BSA)

Efficacy

Time frame: Over 14.5 years post dosing with ECUR-506

Protein allowance g/kg

Efficacy

Time frame: Over 14.5 years post dosing with ECUR-506

Concentration of blood urea nitrogen measurements

Pharmacodynamics

Time frame: Over 14.5 years post dosing with ECUR-506

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)

Efficacy

Time frame: Over 14.5 years post dosing with ECUR-506

Transplant free survival

Efficacy

Time frame: Over 14.5 years post dosing with ECUR-506

Time to liver transplant or any cause of death from dosing to EOS.

Efficacy

Time frame: Over 14.5 years post dosing with ECUR-506

Overall Survival: 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).

Efficacy

Time frame: Over 14.5 years post dosing with ECUR-506

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).

Efficacy

Time frame: Over 14.5 years post dosing with ECUR-506