Use of Pan-Vascular Endothelial Growth Factor Receptor (Pan-VEGF) Blockade for the Treatment of Retinopathy of Prematurity (ROP) (Compassionate Use BLOCK-ROP)

March 27, 2009 updated by: Children's Hospital Los Angeles

Compassionate Use Pan-VEGF Blockade for the Treatment of ROP (Compassionate Use BLOCK-ROP) Trial

The purpose of this study is to provide access to intravitreal injection of Avastin in high-risk infants who do not otherwise qualify for study NCT00702819, an investigational multi-site study examining Avastin use for retinopathy of prematurity.

Study Overview

Status

Temporarily not available

Conditions

Intervention / Treatment

Detailed Description

Retinopathy of Prematurity (ROP) is a leading cause of blindness in children in developed countries around the world, and an increasing cause of blindness in developing countries. The retina lines the inside of the eye. It functions as "film" within the camera which is the eye. When an infant is born prematurely, the vascular network necessary to nourish the retina has not fully developed. As a consequence, in some infants abnormal vessels proliferate instead of the normal ones - a condition known as ROP. The abnormal vessels carry scar tissue along with them, and may lead to retinal detachment and blindness if the eye is not treated. The Multicenter Trial of Cryotherapy for Retinopathy of Prematurity (CRYO-ROP) Study demonstrated that ablation of the peripheral avascular retina reduced the risk of poor structural and visual outcome due to retinal distortion or detachment in ROP (1980's). The ablated retina is not functional and is not amenable to regeneration. Peripheral retinal ablation is not universally effective in fostering regression of ROP. This is particularly true for an aggressive form of ROP (aggressive posterior ROP, or APROP) which typically afflicts profoundly premature and infirm neonates. In this subset of infants, progression of ROP to bilateral retinal detachment and blindness occurs despite timely and complete peripheral retinal laser ablation.

The development of ROP is largely dependent on vascular endothelial growth factor (VEGF). When an infant is born prematurely the relatively hyperoxic environment the baby is introduced to shuts down the production of VEGF. Retinal maturation is delayed. Subsequently, at a time when intraocular VEGF levels would normally be declining late in the third trimester of pregnancy, abnormally high levels of VEGF are seen due to large areas of avascular retina and associated tissue hypoxia. The availability of FDA-approved drugs for anti-VEGF treatment renders it possible to treat such eyes off-label. Available drugs include pegaptanib sodium (Macugen) for partial blockage of VEGF-A, or drugs such as ranibizumab (Lucentis) and bevacizumab (Avastin), which cause complete blockage of VEGF-A. As VEGF is required in the developing retina for normal angiogenesis, our goal is not to penetrate tissue, but to block the excessive levels of VEGF trapped within the overlying vitreous which is responsible for the abnormal vasculature in ROP.

For purposes of this study we have chosen bevacizumab (Avastin), which will: a) attain complete blockage (vs. Macugen) of intravitreal VEGF-A, and; b) which is limited in its ability to penetrate tissues because it is a full antibody (vs. Lucentis, an antibody fragment specifically designed for better tissue penetration), and is more likely to restore VEGF homeostasis within the developing retina.

There is a nearly identical multi-site trial (NCT00702819) currently recruiting, which Childrens Hospital Los Angeles is a part of. However, that study has limiting enrollment criteria; this compassionate-use study was created to provide access to bevacizumab (Avastin) for high-risk infants who do not qualify for study NCT00702819.

Study Type

Expanded Access

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Childrens Hospital Los Angeles

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Inborn babies at CHLA NICU
  • Outborn babies transferred to CHLA NICU
  • Zone 1 or 2 ROP
  • Adequate/appropriate laser ablation
  • Failed standard laser treatment (persistent Plus disease at a minimum of 1 week post-laser)
  • Post-menstrual age greater than 30 weeks

Exclusion Criteria:

  • Zone 3 ROP
  • Inadequate initial laser treatment
  • Most recent laser treatment less than 1 week
  • Evidence of tractional retinal detachment (exudative retinal detachment may be included in study group)
  • Post-menstrual age less than 30 weeks
  • Health not allowing for full protocol participation (determined by neonatologist)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Children's Hospital Los Angeles

Investigators

  • Principal Investigator: Thomas Lee, M.D., Children's Hospital Los Angeles

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

March 26, 2009

First Submitted That Met QC Criteria

March 26, 2009

First Posted (Estimate)

March 27, 2009

Study Record Updates

Last Update Posted (Estimate)

March 30, 2009

Last Update Submitted That Met QC Criteria

March 27, 2009

Last Verified

March 1, 2009

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 09-00044

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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