RecruitingNot Applicable

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

United Kingdom13 participantsStarted 2024-12-23

Plain-language 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.

Who can participate

Age range7 Months – 15 Months

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

What they're measuring

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

Timeframe: Over 14.5 years post dosing with ECUR-506

Change from baseline over 14.5 years post infusion in length

Timeframe: Over 14.5 years post dosing with ECUR-506

Change from baseline over 14.5 years post infusion in weight

Timeframe: Over 14.5 years post dosing with ECUR-506

Urinalysis (Dip Stick) Evaluations

Timeframe: Over 14.5 years post dosing with ECUR-506

Trial details

NCT IDNCT06805695

SponsoriECURE, Inc.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2041-07

Contact for this trial

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

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

View on ClinicalTrials.gov More Ornithine Transcarbamylase Deficiency trials