PhaseI/II Investigator-Initiated Trial to Investigate Safety and Efficacy of Ripasudil in Patients With Retinopathy of Prematurity

Phase 1

Completed

• Conditions: Retinopathy of Prematurity
• Interventions: Drug: Ripasudil ophthalmic solution 0.4%

• Registration Number: NCT04621136
• Lead Sponsor: Kyushu University

Brief Summary

This is an open-label, single-arm phase I/II trial to evaluate the safety and efficacy of Rho-associated protein kinase(ROCK) inhibitor Ripasudil eye drops for preterm infants with Retinopathy of Prematurity(ROP).

Detailed Description

Ripasudil eye drops will be administrated to all enrolled preterm infants with zone I/II stage 1 or greater ROP （except for aggressive posterior ROP, Type1 ROP ）.

The safety and efficacy of ripasudil in treated patients will be assessed in comparison to a historical control.

Study Timeline

• Study Start: 2020-11-01
• Study First Submitted: 2020-11-04
• Study First Submitted QC: 2020-11-04
• Study First Posted: 2020-11-09
• Primary Completion: 2022-11-30
• Study Completion: 2022-12-31
• Status Verified: 2023-02
• Last Update Submitted: 2023-02-03
• Last Update Posted: 2023-02-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

• 1. Informed consent signed by parents or legal guardians of the patient
• 2. Patients born with less than or equal to 32 weeks of gestational age and/or less than or equal to 1,500 grams of birth weight

• 3. Patients with the following types of ROP in both eyes

  4. Zone I ROP with stage greater than or equal to 1

  5. Zone II ROP with stage greater than or equal to 1

Exclusion Criteria

• 1. Patients with aggressive posterior ROP in one or both eyes
• 2. Patients with type 1 ROP in one or both eyes
• 3. Patients with a confirmed or suspected chromosomal abnormality or genetic disorder
• 4. History of hypersensitivity to ripasudil (including drugs with similar chemical structure) in patients or their mothers
• 5. Patients or their mothers have participated in another intervention study and have used the study drug of another intervention study within 30 days prior to enrollment in this study or within 5 times the half-life of the study drug
• 6. Patients with an ocular structural abnormality that can affect the evaluation of this clinical study by investigators
• 7. Patients with a clinically significant neurological disease (e.g., intraventricular hemorrhage grade 3 or higher, severe hydrocephalus with significantly increased intracranial pressure)
• 8. Patients with inadequate blood access
• 9. Patients with complications, allergies, or worsening systemic conditions that make it difficult to conduct this trial
• 10. Patients judged unsuitable by investigators

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Ripasudil eye drops	Ripasudil ophthalmic solution 0.4%	Ripasudil eye drops

Primary Outcome Measures

Name	Time	Method
Proportion of patients with Adverse Events(AEs) and Adverse Drug Reactions(ADRs)	throughout the study duration(up to week16)	Safety Assessment

Secondary Outcome Measures

Name	Time	Method
Concentration of ripasudil and its metabolite M1	throughout the study duration(up to week12)	Pharmacokinetics, the population pharmacokinetic(popPK)
Proportion of patients with ROP remission in both eyes	week12 of treatment	Efficacy Assessment： ripasudil-treated patients will be assessed in comparison to the medical records of a historical control group
Proportion of patients whose ROP worsens to Type1 ROP in one or both eyes	week12 of treatment	Efficacy Assessment： ripasudil-treated patients will be assessed in comparison to the medical records of a historical control group

Trial Locations

Locations (3)

University Hospital of Occupational and Environmental Health; 🇯🇵 Kitakyushu, Fukuoka, Japan

Yamaguchi University Hospital; 🇯🇵 Ube, Yamaguchi, Japan

Kyushu University Hospital; 🇯🇵 Fukuoka, Japan