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Completed

NCT Number: NCT02640664

Rainbow Extension Study

The purpose of this study was to evaluate the long term efficacy and safety of intravitreal ranibizumab compared with laser ablation therapy in patients who were treated for retinopathy of prematurity (ROP) in the core study CRFB002H2301 (NCT02375971)

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Key information

Conditions

Retinopathy of Prematurity (ROP) Congenital, Hereditary, and Neonatal Diseases and Abnormalities Eye Diseases Infant, Newborn, Diseases Infant, Premature, Diseases Retinal Diseases Retinopathy of Prematurity

Age range

6 month–5 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Novartis Investigative Site, Graz, Austria

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About this study

This was a multicenter, open-label extension study where the Visual Acuity (VA) assessment at the child's 5th birthday visit was performed. The study had 2 distinct periods (Epochs). Treatment with study ranibizumab (either as retreatment after ranibizumab had already been injected in the same eye or as switch ranibizumab treatment from study laser therapy administered in the core study) was permitted for eligible eyes with recurrence/worsening of ROP up to and including Week 40 from the baseline visit in the core study (Epoch 1). The remainder of the extension study up to the 5th birthday visit (Epoch 2) was observational, with no study treatment planned to be administered.

In the core study, patients were randomized to 1 of the 3 treatment arms (ranibizumab 0.2 mg, ranibizumab 0.1 mg, and laser). Treatment arm assignment and patient identifier in the extension study remained the same as in the core study.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Signed informed consent from parent(s) or legal guardian(s), in compliance with local requirements
  • The patient successfully completed the core study H2301, as defined by providing assessments at the Visit 112 (the last scheduled visit in the core study) or, if appropriate, at the last of the additional assessment visits as per protocol in H2301, whichever was latest
  • The patient received study treatment in both eyes at baseline of study H2301

Exclusion criteria

  • Patient had a medical condition or personal circumstance which precluded study participation or compliance with study procedures, as assessed by the Investigator
  • Patient had been discontinued from the core study H2301 at any time

Treatment and study plan

ranibizumab

Drug

1 intravitreal injection in both eyes on Day 1 (Baseline), with up to 2 re-treatments allowed for each eye if required

Primary outcomes

  1. Visual Acuity (VA) of the Better-seeing Eye at the Participant's Fifth Birthday Visit - Comparison Between Treatment Arms

    Time frame: at the participant's fifth birthday visit (maximum 5 years and 4 months post core baseline visit)

    The VA assessment at the child's 5th birthday visit was performed using Early Treatment Diabetic Retinopathy Study (ETDRS) methodology. VA measurements were taken in a sitting position at an initial test distance of 3 meters using Lea Symbols charts. Scores represented the number of optotypes (Lea symbols) the participant identified and ranged from 0 to 100, with higher scores indicating better visual acuity. VA was tested in each eye, using the child's current refractive index. The better-seeing eye was defined as the eye with the higher ETDRS score at the 5th birthday visit. If both eyes had the same ETDRS score, then the right eye was assigned as the better-seeing eye.

Secondary outcomes

  1. Number of Participants With Ocular Adverse Events (AEs) Regardless of Study Treatment or Procedure Relationship by Preferred Term

    Time frame: throughout the study, approximately 5 years

    Number of participants with ocular AEs starting during the core study and ongoing at extension baseline, or starting on/after extension baseline were reported.

  2. Number of Participants With Non-ocular Adverse Events (AEs) Regardless of Study Treatment or Procedure Relationship (Greater Than or Equal to 3% in Any Arm) by Preferred Term

    Time frame: throughout the study, approximately 5 years

    Number of participants with non-ocular adverse events regardless of study treatment or procedure relationship (greater than or equal to 3% in any arm) by preferred term were reported.

  3. Visual Acuity (VA) of the Worse-seeing Eye at the Participant's Fifth Birthday Visit - Comparison Between Treatment Arms

    Time frame: at the participant's fifth birthday visit (maximum 5 years and 4 months post core baseline visit)

    The VA assessment at the child's 5th birthday visit was performed using Early Treatment Diabetic Retinopathy Study (ETDRS) methodology. VA measurements were taken in a sitting position at an initial test distance of 3 meters using Lea Symbols charts. Scores represented the number of optotypes (Lea symbols) the participant identified and ranged from 0 to 100, with higher scores indicating better visual acuity. VA was tested in each eye, using the child's current refractive index. The worse-seeing eye was the eye with a lower ETDRS score at the 5th birthday visit. If both eyes had the same ETDRS score, then the left eye was assigned as the worse-seeing eye.

  4. Number of Participants With Absence of Active Retinopathy of Prematurity (ROP) at 40 Weeks Post Core Baseline Visit

    Time frame: at 40 weeks post core baseline visit

    The absence of active ROP in both eyes is defined by the absence of all of the following features: (1) Vessel dilatation of plus disease in at least 2 quardrants (some persisting tortuosity is allowed), (2) Extra-retina vessels extending from the retina into the vitreous and judged to be a sign of active ROP disease.

  5. Number of Participants With Absence of Active Retinopathy of Prematurity (ROP) at 52 Weeks Post Core Baseline Visit

    Time frame: at 52 weeks post core baseline visit

    The absence of active ROP in both eyes is defined by the absence of all of the following features: (1) Vessel dilatation of plus disease in at least 2 quardrants (some persisting tortuosity is allowed), (2) Extra-retina vessels extending from the retina into the vitreous and judged to be a sign of active ROP disease.

  6. Number of Participants With Absence of All Ocular Structural Abnormalities at or Before 40 Weeks Post Baseline Visit

    Time frame: at or before 40 weeks post baseline visit

    The absence of all ocular structural abnormalities is defined by the absence of all of the following fundus features in both eyes at or before the given time point: (1) Substantial temporal retinal vessel dragging causing abnormal structural features/macular Ectopia, (2) Retrolental membrane obscuring the view of the posterior pole, (3) Posterior retinal fold involving the macula, (4) Retinal detachment involving the macula

  7. Number of Participants With Absence of All Ocular Structural Abnormalities at or Before the Participant's Fifth Birthday Visit

    Time frame: at or before the participant's fifth birthday visit (up to maximum 5 years and 4 months post core baseline visit)

    The absence of all ocular structural abnormalities is defined by the absence of all of the following fundus features in both eyes at or before the given time point: (1) Substantial temporal retinal vessel dragging causing abnormal structural features/macular Ectopia, (2) Retrolental membrane obscuring the view of the posterior pole, (3) Posterior retinal fold involving the macula, (4) Retinal detachment involving the macula

  8. Number of Participants With Absence of Individual Ocular Structural Abnormalities at or Before 40 Weeks Post Baseline Visit

    Time frame: at or before 40 weeks post baseline visit

    Number of participants with absence of each structural abnormality in both eyes at or before the given time point: (1) Substantial temporal retinal vessel dragging causing abnormal structural features/macular Ectopia, (2) Retrolental membrane obscuring the view of the posterior pole, (3) Posterior retinal fold involving the macula, (4) Retinal detachment involving the macula, (5) Retinal detachment not involving the macula, (6) Pre-retinal fibrosis

  9. Number of Participants With Absence of Individual Ocular Structural Abnormalities at or Before the Participant's Fifth Birthday Visit

    Time frame: at or before the participant's fifth birthday visit (up to maximum 5 years and 4 months post core baseline visit)

    Number of participants with absence of each structural abnormality in both eyes at or before the given time point: (1) Substantial temporal retinal vessel dragging causing abnormal structural features/macular Ectopia, (2) Retrolental membrane obscuring the view of the posterior pole, (3) Posterior retinal fold involving the macula, (4) Retinal detachment involving the macula, (5) Retinal detachment not involving the macula, (6) Pre-retinal fibrosis, (7) Optic disc pallor, (8) Optic disc swelling, (9) Pigmentary disturbance in the macula, (10) Atrophic changes in the macula

  10. Number of Participants With Absence of All Ocular Structural Abnormalities at or Before Participant's 2 Years Corrected Age Visit

    Time frame: at or before participant's 2 years corrected age visit (up to 2 years and 4 months post core baseline visit)

    The absence of all ocular structural abnormalities is defined by the absence of all of the following fundus features in both eyes at or before the given time point: (1) Substantial temporal retinal vessel dragging causing abnormal structural features/macular Ectopia, (2) Retrolental membrane obscuring the view of the posterior pole, (3) Posterior retinal fold involving the macula, (4) Retinal detachment involving the macula

  11. Number of Participants With Absence of Individual Ocular Structural Abnormalities at or Before Participant's 2 Years Corrected Age Visit

    Time frame: at or before participant's 2 years corrected age visit (up to 2 years and 4 months post core baseline visit)

    Number of participants with absence of each structural abnormality in both eyes at or before the given time point: (1) Substantial temporal retinal vessel dragging causing abnormal structural features/macular Ectopia, (2) Retrolental membrane obscuring the view of the posterior pole, (3) Posterior retinal fold involving the macula, (4) Retinal detachment involving the macula, (5) Retinal detachment not involving the macula, (6) Pre-retinal fibrosis, (7) Optic disc pallor, (8) Optic disc swelling, (9) Pigmentary disturbance in the macula, (10) Atrophic changes in the macula

  12. Number of Participants With Recurrence of ROP up to 40 Weeks Post Baseline Visit in the Core Study

    Time frame: up to 40 weeks post baseline visit in the core study

    Recurrence of ROP was defined as ROP receiving any post-baseline intervention after the 1st study treatment in the core study. In the ranibizumab arms, post-baseline interventions were ranibizumab retreatment or switch to laser. In the laser arm, post-baseline interventions were supplementary laser treatments after 11 days post-baseline, or switch to ranibizumab; supplementary laser treatment within 11 days post-baseline was not counted as recurrence.

  13. Number of Participants With Recurrence of ROP up to 52 Weeks Post Baseline Visit in the Core Study

    Time frame: up to 52 weeks post baseline visit in the core study

    Recurrence of ROP was defined as ROP receiving any post-baseline intervention after the 1st study treatment in the core study. In the ranibizumab arms, post-baseline interventions were ranibizumab retreatment or switch to laser. In the laser arm, post-baseline interventions were supplementary laser treatments after 11 days post-baseline, or switch to ranibizumab; supplementary laser treatment within 11 days post-baseline was not counted as recurrence. Beyond Week 40, participants did not receive any study intervention and no new data was collected after 40 weeks post core baseline visit.

  14. Number of Ranibizumab Injections Received Per Participant Over the Whole Safety Observation Period

    Time frame: up to and including 40 weeks post baseline visit in the core study

    Number of ranibizumab injections received in the treatment of participants with ROP up to and including 40 weeks post baseline visit in the core study were reported.

  15. Refraction Status: Summary of Participants at Participant's 2 Years Corrected Age

    Time frame: at participant's 2 years corrected age (maximum 2 years and 4 months post core baseline visit)

    Summary of participants was reported to evaluate the refraction in each eye at the participant's 2 years corrected age

  16. Refraction Status: Summary of Participants at the Participant's Fifth Birthday Visit

    Time frame: at the participant's fifth birthday visit (maximum 5 years and 4 months post core baseline visit)

    Summary of participants was reported to evaluate the refraction in each eye at the participant's 2 years' corrected age

  17. Change From Baseline in Weight

    Time frame: Baseline of the core study, at the subject's 2 years' corrected age (maximum 2 years and 4 months post core baseline visit) and at the subjects' fifth birthday (maximum 5 years and 4 months post core baseline visit)

    Subject´s weight was reported to evaluate the physical development.

  18. Change From Baseline in Head Circumference

    Time frame: Baseline of the core study and at the subject's 2 years' corrected age (maximum 2 years and 4 months post core baseline visit)

    Subject´s head circumference was reported to evaluate the physical development.

  19. Change From Baseline in Sitting Diastolic Blood Pressure

    Time frame: Baseline of the core study and at the subject's 2 years' corrected age (maximum 2 years and 4 months post core baseline visit)

    Subject´s Sitting Diastolic Blood Pressure was reported to evaluate the physical development.

  20. Change From Baseline in Sitting Systolic Blood Pressure

    Time frame: Baseline of the core study and at the subject's 2 years' corrected age (maximum 2 years and 4 months post core baseline visit)

    Subject´s Sitting Systolic Blood Pressure was reported to evaluate the physical development.

  21. Number of Participants With the Summary of Respiratory Function Status

    Time frame: at the participants' fifth birthday visit (maximum 5 years and 4 months post core baseline visit)

    Number of participants with respiratory function status was reported

  22. Number of Participants With Hearing Impairment of Any Type

    Time frame: at the participants' fifth birthday visit (maximum 5 years and 4 months post core baseline visit)

    Number of participants with hearing function status was reported

  23. Duration of Hospitalization

    Time frame: From baseline of the core study up to 5 years and 4 months post core baseline visit

    Duration of hospitalization (from birth to first hospital discharge home) was reported to evaluate the health status of the subject

  24. Weight at the Time of First Hospital Discharge

    Time frame: From baseline of the core study up to 5 years and 4 months post core baseline visit

    Weight (gram) at the time of first hospital discharge was reported to evaluate the health status of the subject

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

RAINBOW Extension Study: an Extension Study to Evaluate the Long Term Efficacy and Safety of RAnibizumab Compared With Laser Therapy for the Treatment of INfants BOrn Prematurely With Retinopathy of Prematurity

Acronym: RainbowExt

Important dates

Study start
2016
Primary completion
2022
Study completion
2022
First posted
Dec 29, 2015
Registry last updated
Feb 13, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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