Study for Adolescents and Adults with Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810

Arcturus Therapeutics

Status and phase: Terminated; Phase 2

Conditions: OTCD; Ornithine Transcarbamylase Deficiency; OTC Deficiency

Treatments: Other: Placebo; Biological: ARCT-810

Study type: Interventional

Funder types: Industry

Identifiers

NCT05526066

ARCT-810-03

Details and patient eligibility

About

The primary objective is to evaluate the safety and tolerability of repeated doses of intravenously administered ARCT-810.

Full description

This study is a Phase 2, randomized, placebo-controlled study of ARCT-810 in people living with OTC deficiency 12 years of age and older. After an at least 4 week screening and diet stabilization period, participants will be randomized 3:1 to receive ARCT-810 or placebo. Following the first dose and safety evaluation, participants will receive up to an additional 5 doses of ARCT-810 or placebo, each separated by 14 days. The treatment period is followed by a 12-week observation period.

Enrollment: 8 patients

Sex: All

Ages: 12 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

1. Adequate cognitive ability to understand study requirements and give informed consent
2. Males and females aged 12 to 65 years inclusive, at Screening
3. Documented diagnosis of OTC deficiency
4. Clinical stability (no clinical symptoms of hyperammonemia within 1 month, no hospitalizations for metabolic decompensation within 3 months, ≤ 2 hospitalizations within 1 year)
5. Stable protein-restricted diet, dietary supplements, and ammonia scavenger regimen (if applicable) for at least 28 days.
6. BMI = 18.0 - 32.0 kg/m2, inclusive for adults, and >5th percentile for adolescents ≥12 to 17 years
7. Must be willing to adhere to contraception guidelines

Key Exclusion Criteria:

1. History of any OTC gene therapy, or history of liver-derived stem cell therapy in the past 3 years
2. History of other medical conditions that may make the participant unsuitable for inclusion or could interfere with study participation (e.g., uncontrolled hypertension or diabetes, malignancy, HIV, hepatitis B or C)
3. History of severe allergic reaction to liposomal or PEG-containing products
4. Abuse of illicit drugs, medications or alcohol
5. Clinically significant laboratory abnormalities on screening labs

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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8 participants in 2 patient groups, including a placebo group

ARCT-810

Experimental group

Description:

Participants receive an initial intravenous (IV) infusion ARCT-810. If considered safe and well tolerated, participants will receive up to 5 additional IV infusions of ARCT-810 administered at 14-day intervals.

Treatment:

Biological: ARCT-810

Placebo, Normal Saline

Placebo Comparator group

Description:

Participants receive an initial IV infusion of placebo. If considered safe and well tolerated, participants receive up to 5 additional IV infusions of placebo administered at 14-day intervals.

Treatment:

Other: Placebo