ECUR-506 for Ornithine Transcarbamylase Deficiency

Recruiting at 10 trial locations

Overseen By George Diaz, M.D., Ph.D.

Age: < 18

Sex: Male

Trial Phase: Phase 1 & 2

Sponsor: iECURE, Inc.

Must not be taking: Gene therapy

Disqualifiers: Hypoxic Ischemic Encephalopathy, Liver failure, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: 1 day

8 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ECUR-506, a gene therapy designed to help baby boys with ornithine transcarbamylase (OTC) deficiency, a genetic condition that can cause dangerous ammonia build-up in the blood. The trial aims to determine the treatment's safety, effectiveness, and optimal dosage. Participants are divided into three groups, each receiving a different amount of the treatment through a one-time IV infusion. Baby boys who have experienced serious symptoms of OTC deficiency, such as high ammonia levels shortly after birth, might be suitable candidates for this trial. As a Phase 1, Phase 2 trial, this research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial information does not specify whether participants must stop taking their current medications. Please consult with the study team for guidance.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that ECUR-506, a gene editing treatment, is being tested for safety and effectiveness. In studies, patients who received ECUR-506 experienced a complete positive response between three to six months after treatment, suggesting the treatment was well-tolerated during that period.

The treatment is administered as a single dose through an IV (intravenous) drip. It is important to note that this study is in the early testing stages, so researchers are closely monitoring safety and patient response.

Since the trial remains in its early stages, ECUR-506 has demonstrated sufficient safety in initial studies to warrant continued testing, but more information is needed to fully understand all potential side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Ornithine Transcarbamylase (OTC) Deficiency, which often involve dietary restrictions and ammonia-reducing medications, ECUR-506 is a gene therapy delivered via a single IV infusion. Researchers are excited about ECUR-506 because it directly targets the root cause of the condition by potentially correcting the underlying genetic defect, offering a more lasting solution. This approach could significantly reduce the need for lifelong dietary management and medication, making it a groundbreaking option for patients with OTC deficiency.

What evidence suggests that ECUR-506 might be an effective treatment for OTC deficiency?

Research has shown that ECUR-506 could be a promising treatment for neonatal onset ornithine transcarbamylase (OTC) deficiency. Early results suggest that ECUR-506 significantly improved symptoms, starting three months after treatment and lasting for at least six months. This experimental treatment is a type of gene therapy targeting the liver to correct the genetic issue causing the disorder. Current evidence supports its potential to effectively manage this serious condition by lowering harmful ammonia levels in the blood. Participants in this trial will receive different dose levels of ECUR-506 to evaluate its safety and efficacy. ^{1 2 3 4 6}

Who Is on the Research Team?

George Diaz, M.D., Ph.D

Principal Investigator

iECURE, Inc.

Are You a Good Fit for This Trial?

This trial is for male babies under 9 months old with a severe form of OTC deficiency, which causes dangerous ammonia build-up in the blood. Babies must weigh between 3.5 and 10 kg, have had all vaccinations appropriate for their age, be born after at least 37 weeks of pregnancy, and have genetically confirmed OTCD.

Inclusion Criteria

My parent or legal guardian can understand and is willing to sign the consent form.

My pregnancy has reached or passed 37 weeks.

I am male.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single intravenous (IV) administration of ECUR-506 at varying dose levels

1 day

1 visit (in-person)

Safety Follow-up

Participants are monitored for treatment-emergent adverse events and other safety parameters

24 weeks

Multiple visits (in-person and virtual)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• ECUR-506

Trial Overview The study tests ECUR-506's safety and tolerability on baby boys with neonatal onset OTC deficiency. It's an early-stage trial (Phase 1/2) where participants receive one dose of the drug intravenously to see how they react to different dose levels.

How Is the Trial Designed?

Group I: Low Dose Level Experimental Treatment 1 Intervention

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

Group II: Intermediate Dose Level Experimental Treatment 1 Intervention

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

Group III: High Dose Level Experimental Treatment 1 Intervention

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

ECUR-506 is already approved in United States, United Kingdom for the following indications:

Approved in United States as ECUR-506 for:

• Neonatal onset ornithine transcarbamylase (OTC) deficiency

Approved in United Kingdom as ECUR-506 for:

• Neonatal onset ornithine transcarbamylase (OTC) deficiency

Who Is Running the Clinical Trial?

iECURE, Inc.

Lead Sponsor

Trials

2

Recruited

30+

Citations

1. iecure.com iecure.com/news/iecure-to-present-otc-hope-clinical-trial-data-of-ecur-506-in-ornithine-transcarbamylase-deficiency-at-the-european-society-of-gene-cell-therapy-annual-congress-and-the-american-society-of-human-gen/

iECURE to Present OTC-HOPE Clinical Trial Data of ECUR ... The OTC-HOPE study is a first-in-human clinical trial of ECUR-506 in baby boys with genetically confirmed neonatal onset OTC deficiency and has ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06255782

An Open-label Study to Investigate ECUR-506 in Male ... This is a Phase 1/2/3, open-label, multicenter, safety, efficacy, and dose finding study of ECUR-506 in male babies with neonatal onset OTC deficiency. The ...

3. sciencedirect.com sciencedirect.com/science/article/pii/S294977442500891X

A first in human, single arm, open label phase 1/2 study ... ECUR-506 is a liver-directed, investigational gene editing product being developed for the treatment of neonatal onset OTCD.

4. investor.precisionbiosciences.com investor.precisionbiosciences.com/news-releases/news-release-details/precision-biosciences-announces-complete-clinical-response-first

Press Releases Treatment with ECUR-506 resulted in a complete clinical response from three months post exposure to the end of study (six months post exposure).

5. iecure.com iecure.com/news/iecure-to-present-initial-otc-hope-clinical-trial-data-of-ecur-506-in-ornithine-transcarbamylase-deficiency-at-6th-international-symposium-on-ucds-and-15th-international-congress-of-inborn-errors-of-m/

iECURE to Present Initial OTC-HOPE Clinical Trial Data of ... The OTC-HOPE study is a first-in-human clinical trial of ECUR-506 in baby boys with genetically confirmed neonatal onset OTC deficiency and has been cleared to ...

6. centerwatch.com centerwatch.com/clinical-trials/listings/NCT06255782/an-open-label-study-to-investigate-ecur-506-in-male-babies-less-than-9-months-of-age-with-neonatal-onset-otc-deficiency

An Open-label Study to Investigate ECUR-506 in Male ... Individuals with OTC deficiency can build-up excess levels of ammonia in their blood, potentially resulting in devastating consequences, ...