This study is to find out if ranibizumab is safe and effective compared to laser therapy in preterm babies with ROP, a condition of abnormal blood vessel (arteries and veins) development in the eyes

• Register: EUCTR
• Last refreshed on: 26 February 2018
• Main ID: EUCTR2014-003041-10-EE
• Date of registration: 11/02/2015
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma Services AG
• Public title: This study is to find out if ranibizumab is safe and effective compared to laser therapy in preterm babies with ROP, a condition of abnormal blood vessel (arteries and veins) development in the eyes
• Scientific title: RAINBOW study: a randomized, controlled study evaluating the efficacy and safety of RAnibizumab compared with laser therapy for the treatment of INfants BOrn prematurely With retinopathy of prematurity
• Date of first enrolment: 06/03/2015
• Target sample size: 180
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-003041-10
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: yes; Single blind: no; Double blind: no; Parallel group: yes; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: yes; Other specify the comparator: laser therapy; Number of treatment arms in the trial: 3
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Austria, Belgium, Colombia, Croatia, Czech Republic, Denmark, Egypt, Estonia, Finland, France, Germany, Greece, Hungary, India, Italy, Japan, Lithuania, Malaysia, Mexico, Poland, Romania, Russian Federation, Saudi Arabia, Slovakia, Sweden, Taiwan, Turkey, United Kingdom, United States

Contacts

• Name: DRA
• Affiliation: Novartis Pharma Services Inc, Estonian affiliate

• Name: DRA
• Affiliation: Novartis Pharma Services Inc, Estonian affiliate

Key inclusion & exclusion criteria

Inclusion criteria:
Patients have to fulfill all of the following criteria prior to receiving the first investigational treatment:
1. Signed informed consent from parent(s) or legal guardian(s), in compliance with local requirements
2. Male or female preterm infants with a birth weight of less than 1500 g
3. Bilateral ROP with 1 of the following retinal findings in each eye:
• Zone I, stage 1+, 2+, 3 or 3+ disease, or
• Zone II, stage 3+ disease, or
• Aggressive posterior ROP
Are the trial subjects under 18? yes
Number of subjects for this age range: 180
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
Patients fulfilling any of the following criteria prior to receiving the first investigational treatment are not eligible for inclusion in this study.
Investigational treatment is not clinically appropriate for the following patients:
1. Have ROP disease characteristic in either eye, other than that listed in “Inclusion Criteria” above, at the time of the firstinvestigational treatment
2. Have a history (either the patient or the mother) of hypersensitivity to any of the investigational treatments or to drugs of similar chemical classes
Risk of confounding efficacy and/or safety assessments in the following patients:
3. Have received any previous surgical or nonsurgical treatment for ROP (e.g., ablative laser therapy or cryotherapy, vitrectomy)
4. Have been previously exposed to any intravitreal or systemic anti-VEGF agent (either the patient or the mother during this child’s pregnancy)
5. Have used (either the patient or the mother) other investigational drugs as part of another clinical study (other than vitamins and minerals) within 30 days or within 5 half-lives of the other investigational drug, whichever is longer
6. Have ocular structural abnormalities that are assessed by the Investigator to have a clinically significant impact on study assessments
7. Have active ocular infection within 5 days before or on the day of first investigational treatment
8. Have a history of hydrocephalus requiring treatment
9. Have a history of any other neurological conditions that are assessed by the Investigator to have a significant risk of severe impact on visual function
10. Have any other medical conditions or clinically significant comorbidities or personal circumstances that are assessed by the Investigator to have a clinically relevant impact on study participation, any of the study procedures, or on efficacy assessments (e.g., poor life expectancy, pupil not able to be adequately dilated,unable to comply with the visit schedule)


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Retinopathy of prematurity (ROP)
MedDRA version: 19.1 Level: PT Classification code 10038933 Term: Retinopathy of prematurity System Organ Class: 10015919 - Eye disorders

Therapeutic area: Diseases [C] - Eye Diseases [C11]

Intervention(s)

Trade Name: Lucentis
Product Name: Lucentis
Product Code: RFB002
Pharmaceutical Form: Solution for injection
INN or Proposed INN: RANIBIZUMAB
CAS Number: 347396-82-1
Current Sponsor code: RFB002
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-

Primary Outcome(s)

Secondary Objective: To evaluate
• whether IVT ranibizumab 0.2 mg has superior efficacy to IVT ranibizumab 0.1 mg in the treatment of ROP as measured by the absence of active ROP and absence of unfavorable structural outcomes in both eyes
• whether IVT ranibizumab 0.1 mg has superior efficacy to laser therapy in the treatment of ROP as measured by the absence of active ROP and absence of unfavorable structural outcomes in both eyes 24 weeks after starting investigational treatment
• the time to intervention with a 2nd modality for ROP or development of unfavorable structural outcome, or death
• the recurrence of ROP receiving any post-baseline intervention at 24 weeks or before
• the ocular and systemic safety of IVT ranibizumab 0.1 mg / 0.2 mg
• the systemic PK of IVT ranibizumab
• the effects of investigational treatment on systemic VEGF levels in patients with ROP
• To assess the number of ranibizumab administrations needed in the treatment of patients with ROP

Timepoint(s) of evaluation of this end point: 24 weeks

Main Objective: To demonstrate that intravitreal ranibizumab 0.2 mg has superior efficacy to laser therapy in the treatment of ROP as measured by the absence of active ROP and absence of unfavorable structural outcomes in both eyes 24 weeks after starting investigational treatment.

Primary end point(s): Absence of active ROP and absence of unfavorable structural outcomes in both eyes 24 weeks after starting investigational treatment

Secondary Outcome(s)

Secondary end point(s): • The components that constitute the definition of absence of active ROP and unfavorable structural outcomes in either eye at or before the 24-weeks assessment visit after starting investigational treatment
• Any of the following unfavorable structural outcomes in either eye at 24 weeks after the first investigational treatment: retrolental membrane obscuring the view of the posterior pole, substantial temporal retinal vessel dragging causing abnormal structural features/ macular ectopia, posterior retinal fold involving the macula, retinal detachment involving the macula
• The time after the first investigational treatment to the first occurrence of one of the following:
- Death
- Intervention for ROP with a treatment modality other than the modality of the first investigational treatment
- An unfavorable structural outcome in either eye
• Requirement of none, 1, or 2 ranibizumab re-treatments (whether unilateral or bilateral retreatment)
• Recurrence of ROP requiring any intervention at 24 weeks or before

Timepoint(s) of evaluation of this end point: 24 weeks

Secondary ID(s)

2014-003041-10-IT

CRFB002H2301

Source(s) of Monetary Support

Novartis Pharma Services AG