Retrospective Case Series of Trans-scleral Cryotherapy for Retinal Hemangioblastoma

NCT04458935 | Active Not Recruiting

• 2 other identifiers: 99992012920-EI-N129
• Study Type: observational
• Target: 25
• Locations: 1 country
• Sites: 1

Brief Summary

Background: Retinal hemangioblastoma (RH) is a tumor. It grows from the retina in the eye. It can threaten a person s vision. Trans-scleral cryotherapy is used to destroy the tumors and minimize the long-term risks of vision loss. RH is a rare condition, often occurring in people with von Hippel-Lindau disease. There are no clinical trials to study how well the treatment works. Researchers want to study the medical records of people with RH who were treated at the NIH eye clinic to learn more. Objective: To analyze clinical data collected over a 20-year span to study consecutive cases of RH managed with trans-scleral cryotherapy at the NIH. Eligibility: People who took part in NIH natural history protocols for which cryotherapy of RH was performed as a standard care measure. Design: Researchers will collect and study data from participants medical charts. Participants will not be contacted because no new data is needed. Researchers were granted a waiver of informed consent for use of these medical records. To protect patient privacy, participants will be assigned an ID number. Their data will be entered into a spreadsheet in a coded fashion. The key to this code will be kept in a secure file. No patient identifying information will be used in the analysis or the publication....

Conditions

Retinal Hemangioblastoma; Von Hippel-Lindau Disease

Keywords

Vascular Tumor; Vision Loss; Natural History

Outcome Measures

Primary Outcomes (1)

• To perform a retrospective analysis of consecutive cases of retinal hemangioblastoma (RH) managed with trans-scleral cryotherapy at the NIH

  Some key measures of interest will include treatment outcomes (rate of control of tumors; visual function measures; post-treatment lesion characteristics; sequelae and complications) and association of outcomes with eye and lesion characteristics.

  ongoing

Study Arms (1)

Participants

Participants with retinal hemangioblastoma (RH) managed with trans-scleral cryotherapy at the NIH.

Eligibility Criteria

• Age: 7 Years - 120 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Data (medical history and images) was collected under NEI and non-NEI natural history protocols allowing administration of standard care measures to participants.

You may qualify if:

• Participants were enrolled in NEI and non-NEI natural history protocols, but in all cases, cryotherapy was administered as a standard care measure (not as an investigative treatment).
• Patients were identified by searching the NIH eye clinic EMR for all of those manifesting one or more retinal hemangioblastomas (with or without associated von Hippel-Lindau disease) treated with cryotherapy.

You may not qualify if:

• None

Sponsors & Collaborators

• National Eye Institute (NEI): lead

Study Sites (1)

National Eye Institute (NEI)

Bethesda, Maryland, 20892, United States

Location

MeSH Terms

Conditions

von Hippel-Lindau Disease; Vascular Neoplasms; Vision Disorders

Condition Hierarchy (Ancestors)

Neurocutaneous Syndromes; Nervous System Diseases; Angiomatosis; Vascular Diseases; Cardiovascular Diseases; Ciliopathies; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Soft Tissue Neoplasms; Neoplasms by Site; Neoplasms; Sensation Disorders; Neurologic Manifestations; Eye Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Emily Y Chew, M.D.

  National Eye Institute (NEI)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 3, 2020
• First Posted: July 7, 2020
• Study Start: July 2, 2020
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): June 30, 2026
• Last Updated: February 5, 2026

Record last verified: 2026-02-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)