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A Phase 1, First-in-Human Study of CITY-RBP4 in Healthy Volunteers and STGD1 Participants

ConditionsStargardt Disease 1STGD1Healthy Volunteers
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age12-45
SponsorCity Therapeutics

About this trial

This is a first-in-human (FIH), multicenter, randomized, double-masked, placebo-controlled adaptive clinical study evaluating the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) of CITY-RBP4, following single-ascending doses and multiple doses, via subcutaneous (SC) administration.

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

Willing and able to provide written informed consent form

Be aged 18 to 45 years (inclusive) at the time of signing the informed consent form

Generally healthy on medical history, exam, labs, and ECG

BCVA 20/25 or better in each eye

Disqualifiers

Clinically significant ocular disease or prior ocular surgery that could interfere with safety assessments

History of vitamin A deficiency, malabsorption, or chronic diarrhea

Clinically significant cardiac, hepatic, renal, pulmonary, neurologic, or psychiatric disease

Positive hepatitis B, hepatitis C, or HIV screening

Trial design

Design model

Sequential

Treatments tested in this trial

  • CITY-RBP4

    Drug

    siRNA (subcutaneous injection)

  • Placebo

    Drug

    Saline (subcutaneous injection)

Treatment groups

48 Participants
are divided into 4 treatment groups
Group A: CITY-RBP4 in Healthy Adults (Part A)Experimental treatment 1 intervention
Group B: In Healthy Adults (Part A)Placebo comparator 1 intervention
Group C: CITY-RBP4 in Participants with STGD1 (Parts B & C)Experimental treatment 1 intervention
Group D: In Participants with STGD1 (Parts B & C)Placebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Incidence, severity, and relationship of treatment-emergent adverse events (TEAEs)

To evaluate the safety and tolerability of single and multiple doses of CITY-RBP4 in healthy adults and participants with Stargardt disease Type 1 (STGD1)

Time frame
Through study completion, up to Day 360

Secondary outcomes

1

Plasma concentrations of CITY-RBP4

To characterize the pharmacokinetic (PK) profile of CITY-RBP4 after single and multiple doses of CITY-RBP4 in healthy adults and participants with STGD1

Time frame
Day 1 to Day 2
2

Urine concentrations of CITY-RBP4

To characterize the pharmacokinetic (PK) profile of CITY-RBP4 after a single dose of CITY-RBP4 in healthy adults and participants with STGD1

Time frame
Part A and B only: Day 1 to Day 2
3

Maximum plasma concentration (Cmax)

To characterize the pharmacokinetic (PK) profile of CITY-RBP4 after single and multiple doses of CITY-RBP4 in healthy adults and participants with STGD1

Time frame
Day 1 to Day 2
4

Time to maximum plasma concentration (Tmax)

To characterize the pharmacokinetic (PK) profile of CITY-RBP4 after single and multiple doses of CITY-RBP4 in healthy adults and participants with STGD1

Time frame
Day 1 to Day 2

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

City Therapeutics

Lead sponsor

Source ClinicalTrials.gov
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