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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06255782


Additional trial details provided through ANZCTR are available at the end of this record.


Registration number
NCT06255782
Ethics application status
Date submitted
19/12/2023
Date registered
13/02/2024

Titles & IDs
Public title
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency (OTC-HOPE)
Scientific title
A Phase I/II First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of a Single Intravenous (IV) Administration of ECUR-506 in Males Less Than 9 Months of Age With Genetically Confirmed Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency
Secondary ID [1] 0 0
OTC HOPE
Secondary ID [2] 0 0
ECUR-506-OTC-101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Ornithine Transcarbamylase Deficiency 0 0
Ornithine Transcarbamylase Deficiency Disease 0 0
Ornithine Carbamoyltransferase Deficiency (Disorder) 0 0
Urea Cycle Disorders, Inborn 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Neurological 0 0 0 0
Other neurological disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Diet and Nutrition 0 0 0 0
Other diet and nutrition disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - ECUR-506

Experimental: Cohort 1 - Participants will receive the Low Dose of ECUR-506 delivered one time via IV Infusion.

Experimental: Cohort 2 - Participants will receive a higher dose of ECUR-506 delivered one time via IV infusion.

Experimental: Additional Cohorts - Additional Cohorts based on recommendations of DMC review of safety and efficacy data.


Treatment: Other: ECUR-506
ECUR-506 is a gene editing treatment delivering a gene encoding the editing enzyme and an OTC gene.
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Treatment-emergent adverse events (incidence, severity, seriousness, and relatedness)
Timepoint [1] 0 0
Over 24 weeks post infusion
Primary outcome [2] 0 0
Urinalysis evaluations
Timepoint [2] 0 0
Assessed as change from baseline at pre-specified timepoints through Week 24 post infusion.
Secondary outcome [1] 0 0
Percent liver transduction
Timepoint [1] 0 0
Assessed at Week 24
Secondary outcome [2] 0 0
Number of hyperammonemic crises (HAC)
Timepoint [2] 0 0
Over 24 weeks post infusion
Secondary outcome [3] 0 0
qPCR measurement to evaluate the clearance of both vectors in body fluids over time
Timepoint [3] 0 0
Over 24 weeks post infusion
Secondary outcome [4] 0 0
Scavenger drug dose per body surface area (BSA)
Timepoint [4] 0 0
Over 24 weeks post infusion
Secondary outcome [5] 0 0
Protein allowance g/kg
Timepoint [5] 0 0
Over 24 weeks post infusion
Secondary outcome [6] 0 0
Blood urea nitrogen measurements
Timepoint [6] 0 0
Over 24 weeks post infusion

Eligibility
Key inclusion criteria
Key

1. Male sex
2. Gestational age = 37 weeks
3. Age at screening is 24 hours to 7 months*
4. Weight = 3.5 kg and = 10.0 at screening
5. Has received all age-appropriate vaccinations
6. Genetically confirmed OTCD
7. Severe neonatal OTCD defined by hyperammonemic crisis within first week of life
8. Current or historical biochemical profile consistent with OTCD
9. Participant's parent(s)/LAR must be able to comprehend and be willing to provide a signed IRB/IEC-approved ICF.

Key
Minimum age
24 Hours
Maximum age
7 Months
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
1. Neonatal diagnosis of severe to profound Hypoxic Ischemic Encephalopathy due to birth injury
2. Requiring urgent liver transplant due to liver failure as assessed by the PI.
3. Contiguous gene deletion involving the OTC gene
4. Known or suspected major organ injury/dysfunction/anomalies.
5. Treatment with any other gene therapy or gene editing therapy
6. Co-enrollment in any other clinical study with an investigational product prior to or during the duration of this trial would require the participant to be withdrawn from this study
7. Any condition, that in the opinion of the Investigator, would compromise the safety of the participant or study data
8. Documented vertical transmission of HSV, HIV, or HepA/HepB/HepC
9. Documented in-utero teratogen, substance, and/or alcohol exposure, which in the opinion of the Investigator may increase the participant's risk of developmental delays, congenital anomalies, and/or significant medical complications

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
8/04/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/09/2026
Actual
Sample size
Target
13
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United Kingdom
State/province [1] 0 0
London
Country [2] 0 0
United Kingdom
State/province [2] 0 0
Newcastle Upon Tyne

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
iECURE, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
George Diaz, M.D., Ph.D
Address 0 0
iECURE, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
George Diaz, M.D., Ph.D.
Address 0 0
Country 0 0
Phone 0 0
1-877-694-3558
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06255782

Additional trial details provided through ANZCTR
Accrual to date
Recruiting in Australia
Recruitment state(s)
ACT,NSW,NT,QLD,SA,TAS,WA,VIC
Recruitment hospital [1] 188
The Children's Hospital at Westmead
Recruitment hospital [2] 189
The Royal Childrens Hospital
Recruitment postcode(s) [1] 187
2145
Recruitment postcode(s) [2] 188
3052
Funding & Sponsors
Primary sponsor
Commercial sector/Industry
Primary sponsor name
iECURE Inc
Primary sponsor address
1777 Sentry Parkway West
Building 14, Suite 200
Blue Bell PA, 19422
Primary sponsor country
United States of America
Other collaborator category [1] 104
Commercial sector/Industry
Name [1] 104
Fortrea Australia Pty ltd
Address [1] 104
Suite 3.02, Level 3, Building A, 97 Waterloo Road, Macquarie Corporate Centre, Macquarie Park, NSW 2113
Country [1] 104
Australia
Ethics approval
Ethics application status
Approved
Ethics committee name [1] 77
Sydney Children's Hospitals Network Human Research Ethics Committee
Address [1] 77
Email: [email protected] (preferred) Mailing Addresses: The Children’s Hospital at Westmead, Kids Research Building, Corner Hawkesbury Road and Hainsworth Street, Westmead NSW 2145. Locked Bag 4001, Westmead NSW 2145
Country [1] 77
Australia
Date submitted for ethics approval [1] 77
20/10/2023
Approval date [1] 77
24/11/2023
Ethics approval number [1] 77
2023/ETH02015
 
Public notes

Contacts
Principal investigator
Title 445 0
Dr
Name 445 0
Shanti Balsubramaniam
Address 445 0
The Children's Hospital at Westmead, Corner Hawkesbury Road and Hainsworth Street, Westmead NSW 2145, Australia
Country 445 0
Australia
Phone 445 0
+61 02 7825 0201
Fax 445 0
Email 445 0
[email protected]
Contact person for public queries
Title 446 0
Mr
Name 446 0
George Diaz M.D. Ph.D.
Address 446 0
1777 Sentry Parkway West Building 14, Suite 200 Blue Bell PA, 19422
Country 446 0
United States of America
Phone 446 0
+1 877 694 3558
Fax 446 0
Email 446 0
[email protected]
Contact person for scientific queries
Title 447 0
Mr
Name 447 0
George Diaz M.D. Ph.D.
Address 447 0
1777 Sentry Parkway West Building 14, Suite 200 Blue Bell PA, 19422
Country 447 0
United States of America
Phone 447 0
+1 877 694 3558
Fax 447 0
Email 447 0
[email protected]