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

NCT06805695 | Recruiting DMC FDA Drug

• 1 other identifier: ECUR-LTFU
• Study Type: observational
• Target: 13
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

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

Keywords

Amino Acid Metabolism, Inborn Errors; Ammonia; Brain Diseases; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Central Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; High Ammonia; Hyperammonemia; Inborn; Inborn Errors; Liver Disease; Liver Transplant; Metabolism; Metabolic Diseases; Metabolism, Inborn Errors; Neonatal; Nervous System Diseases; NH4; Ornithine; Ornithine Transcarbamylase Deficiency; OTC; OTC Deficiency; OTCD; Transcarbamylase; UCD; Urea Cycle Disorders; Long Term Follow Up; Long-Term Follow-Up; LTFU

Outcome Measures

Primary Outcomes (4)

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

  Safety

  Over 14.5 years post dosing with ECUR-506

• Change from baseline over 14.5 years post infusion in length

  Length measured in cenitmeters

  Over 14.5 years post dosing with ECUR-506

• Change from baseline over 14.5 years post infusion in weight

  Weight measured in kilograms

  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.

  Over 14.5 years post dosing with ECUR-506

Secondary Outcomes (18)

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

  Over 14.5 years post dosing with ECUR-506

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

  Over 14.5 years post dosing with ECUR-506

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

  Over 14.5 years post dosing with ECUR-506

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

  Over 14.5 years post dosing with ECUR-506

• Percent Liver Transduction

  Over 14.5 years post dosing with ECUR-506

Other Outcomes (12)

• Number of participants with antibodies to hOTC in blood.

  Over 14.5 years post dosing with ECUR-506

• Number of participants with antibodies to M2PCSK9 in blood.

  Over 14.5 years post dosing with ECUR-506

• Number of participants with antibodies to AAVrh79 in blood.

  Over 14.5 years post dosing with ECUR-506

Study Arms (2)

Enrolled and Dosed

Previously dosed in a previous iECURE study.

Other: No Intervention

Enrolled but Not Dosed

Enrolled in a previous iECURE study, but not dosed.

Other: No Intervention

Interventions

No Intervention OTHER

No Intervention

Enrolled and Dosed; Enrolled but Not Dosed

Eligibility Criteria

• Age: 7 Months - 15 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

All participants from an iECURE parent treatment protocol, whether they received IP or not, will be consented to enter this LTFU study. Enrollment into the LTFU will begin upon either premature discontinuation from, or completion of, the parent treatment protocol.

You may qualify if:

• 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.

You may not qualify if:

• \. 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.

Sponsors & Collaborators

• iECURE, Inc.: lead

Study Sites (1)

Great Ormond Street Hospital

London, United Kingdom

RECRUITING

MeSH Terms

Conditions

Ornithine Carbamoyltransferase Deficiency Disease; Urea Cycle Disorders, Inborn; Amino Acid Metabolism, Inborn Errors; Brain Diseases; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Central Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Hyperammonemia; Liver Diseases; Metabolic Diseases; Metabolism, Inborn Errors; Nervous System Diseases

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Digestive System Diseases

Study Officials

• George Diaz, M.D., Ph.D

  iECURE, Inc.

  STUDY DIRECTOR

Central Study Contacts

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

CONTACT

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

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 20, 2024
• First Posted: February 3, 2025
• Study Start: December 23, 2024
• Primary Completion (Estimated): July 1, 2041
• Study Completion (Estimated): July 1, 2041
• Last Updated: February 3, 2025

Record last verified: 2025-01

Locations

GB (1)