Phase I/II Investigator-Initiated Trial to Investigate Safety and Efficacy of Ripasudil in Patients with Retinopathy of Prematurity

JRCT ID: jRCT2071200047

Registered date:28/10/2020

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Retinopathy of Prematurity
• Date of first enrollment: 04/11/2020
• Target sample size: 24
• Study type: Interventional

Intervention(s)

Phase I
A total of three infants will receive ripasudil eye drops once daily for one week, followed by twice-daily drug administration for two weeks. At the end of the three weeks of treatment for the first patient, a data and safety monitoring board (DSMB) will be held to decide whether the second and third patients can be enrolled into phase I. The DSMB will discuss the safety again at the end of the three-week eye drop treatment for the third patient to determine if phase II can begin. In addition, the three infants who participate in phase I can continue to receive the eye drop treatment for additional 9 weeks (12 weeks in total) if the investigators determine that there are no safety issues with ripasudil.

Phase II
A total of 21 patients from three clinical trial institutions will receive ripasudil eye drops twice daily for 12 weeks. The safety and efficacy will be assessed at the end of the 12-week treatment period. The efficacy of ripasudil will be evaluated in comparison to the results of the historical control. In addition, another follow-up examination will be performed four weeks after the end of the treatment.

Outcome(s)

• Primary Outcome: Proportion of patients with AEs and ADRs

Secondary Outcome

Secondary endpoints
1. Efficacy
a. Proportion of patients whose ROP worsens to type 1 ROP in one or both eyes (phase II)
b. Proportion of patients with ROP remission in both eyes (phase II)
2. Pharmacokinetics of ripasudil (phase I and II)

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: <=
• Gender: Both

Include 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
a. Zone I ROP with stage greater than or equal to 1
b. Zone II ROP with stage greater than or equal to 1

Exclude 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

Related Information

• Primary Sponsor: Arima Mitsuru
• Source(s) of Monetary Support: Japan Agency for Medical Research and Development (AMED)