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NCT Number: NCT06805695

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

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.

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Key information

Conditions

Ornithine Transcarbamylase Deficiency Amino Acid Metabolism, Inborn Errors Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked Hyperammonemia Liver Diseases Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Ornithine Carbamoyltransferase Deficiency Disease Ornithine Transcarbamylase Deficiency Disease Pathologic Processes Pathological Conditions, Signs and Symptoms Urea Cycle Disorders, Inborn

Age range

7 month–15 month

Sex eligibility

All sexes

Study type

Observational

Primary location

Great Ormond Street Hospital

London, United Kingdom

Location status: Recruiting

Location contact

Christopher Jackson

CONTACT

[email protected]

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

  • 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.

Treatment and study plan

No intervention

Other

No Intervention

Primary outcomes

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

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

    Safety

  2. Change from baseline over 14.5 years post infusion in length

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

    Length measured in cenitmeters

  3. Change from baseline over 14.5 years post infusion in weight

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

    Weight measured in kilograms

  4. Urinalysis (Dip Stick) Evaluations

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

    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.

Secondary outcomes

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

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

    Pharmacokinetics

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

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

    Pharmacokinetics

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

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

    Pharmacokinetics

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

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

    Pharmacokinetics

  5. Percent Liver Transduction

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

    Pharmacokinetics

  6. Number of hyperammonemic crises (HAC)

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

    Pharmacodynamics and Efficacy

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

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

    Pharmacodynamics and Safety

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

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

    Pharmacodynamics and Safety

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

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

    Pharmacodynamics and Safety

  10. 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

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

    Pharmacodynamics and Safety

  11. Scavenger drug dose per body surface area (BSA)

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

    Efficacy

  12. Protein allowance g/kg

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

    Efficacy

  13. Concentration of blood urea nitrogen measurements

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

    Pharmacodynamics

  14. 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)

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

    Efficacy

  15. Transplant free survival

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

    Efficacy

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

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

    Efficacy

  17. 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).

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

    Efficacy

  18. 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).

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

    Efficacy

Other outcomes

  1. Number of participants with antibodies to hOTC in blood.

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

    Pharmacodynamics

  2. Number of participants with antibodies to M2PCSK9 in blood.

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

    Pharmacodynamics

  3. Number of participants with antibodies to AAVrh79 in blood.

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

    Pharmacodynamics

  4. Concentration of plasma ammonia

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

    Pharmacodynamics

  5. Concentration of plasma citrulline

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

    Pharmacodynamics

  6. Concentration of plasma glutamine

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

    Pharmacodynamics

  7. Urinary excretion of phenylacetate metabolites.

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

    Urinary phenylactate (mcg/mL), urinary phenylactylglutamine (mcg/mL), urinary phenylacetate/ phenylglutamine ratio.

  8. Urinary excretion of orotic acid metabolites

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

    Urinary orotic acid (mmol/mol creatinine), urinary uracil (mmol/mol creatinine)

  9. Future DNA analysis of WBCs collected in a parent trial may be performed only in the event of an observed genomic safety signal

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

    Safety

  10. Developmental assessments as measured by age-appropriate Bayley Scale of Infant Development IV (BSID-IV)

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

    Total Raw Scores, Growth Score Values (GSVs), and Age Equivalent Scores will be analyzed; Raw Scores range 0-162; GSVs range 428-599; Age Equivalent Scores range 1-42 months; a higher score reflects a higher level of skill.

  11. Kaufman Assessment Battery for Children - Second Edition Normative Update (KABC-II NU)

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

    Total Raw Scores, Growth Score Values (GSVs), and Age Equivalent Scores will be analyzed; Raw Scores range 0-162; GSVs range 428-599; Age Equivalent Scores range 1-42 months; a higher score reflects a higher level of skill.

  12. Quality of life as measured by an age-appropriate Pediatric Quality of Life Inventory Infant Scales

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

    Quality of Life Outcomes

Study contacts

Contact information is provided by the study sponsor or research team.

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

CONTACT

[email protected]

1-877-694-3558

Sponsors and collaborators

Lead sponsor

iECURE, Inc.

Industry

Registry information

Important dates

Study start
2024
Primary completion
2041
Study completion
2041
First posted
Feb 3, 2025
Registry last updated
Feb 3, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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