Sickle Cell AnemiaNCT00618644
Ranibizumab for Neovascularization in Sickle Cell Retinopathy
A study testing Ranibizumab in people with Sickle Cell Anemia and Retinopathy, run by Kresge Eye Institute at 1 site in United States.
Trial summary
The purpose of this study is to determine the ocular and non-ocular safety of a single dose of ranibizumab in treating neovascularization secondary to sickle cell retinopathy. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Anemia, Retinopathy
- What is being tested?
- Ranibizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2010to Jun 2011
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with sickle cell anemia and retinopathy
- Over age 18 years
- Non-pregnant
Who cannot join
- Pregnant
- Glaucoma
- Patients using anticoagulants (e.g., warfarin)
- Retinal detachment
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase I Study to Evaluate the Ocular and Non Ocular Safety of Ranibizumab in Treating Neovascularization Secondary to Sickle Cell Retinopathy
The purpose of this study is to determine the ocular and non-ocular safety of a single dose of ranibizumab in treating neovascularization secondary to sickle cell retinopathy.
Read the detailed description
In the U.S., about 10% of African Americans have an abnormal hemoglobin gene. About 8% of African Americans are heterozygous for Hemoglobin S. In the United States, sickle cell anemia primarily occurs in the black population, with approximately 0.2% of African American children afflicted by this disease. It may be associated with other hemoglobinopathies as well. The prevalence in adults is lower because of the decrease in life expectancy. Systemically, the sickle cell anemia variation (SS) produces the most symptoms. With respect to the eye, the sickle cell disease mutation (SC) produces the most effects. Overall, the sickle cell trait expression (AS) produces the fewest complications.
- Among patients with SC or SThal, the incidence of proliferation sickle cell retinopathy is 33% and 14% respectively.
- Proliferative sickle cell retinopathy is the major cause of vision loss in sickle cell disease.
For sickle cell retinopathy, the commonly used therapeutic modalities include laser retinal photocoagulation, retinal cryotherapy, and vitrectomy/membranectomy depending on the severity of the disease. The most effective therapeutic modality with minimal postoperative complications appears to be scatter laser retinal photocoagulation.
A single case study of bevacizumab was found to effective in short term regression of neovascularization and improving vision after a single injection. Further study with ranibizumab is warranted.
Recent clinical trials (Marina and Anchor) have demonstrated that ranibizumab is effective in treating patients with CNV with age-related macular degeneration. Retinopathy in sickle cell disease has also been linked to VEGF. Therefore, patients with sickle cell retinopathy should respond to ranibizumab therapy.
This is an open-label single dose, phase I study of intravitreally administered ranibizumab in patients with sickle cell retinopathy.
Consented, enrolled subjects will receive a single open-label intravitreal injection of 0.5 mg ranibizumab.
Three subjects from one site in the United States will be enrolled.
Patients will receive one dose of 0.5 mg ranibizumab administered intravitreally.
- Conditions
- Sickle Cell Anemia, Retinopathy
- Being tested
- Ranibizumab
- Medical subject headings
- Anemia, Sickle Cell, Retinal Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RanibizumabDrug
Also called Lucentis (ranibizumab)
Ranibizumab 0.5 mg intravitreal injection
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Ranibizumab injection Receives: Drug: Ranibizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Ocular safety of a single dose of ranibizumab
Measured: Three months
Secondary outcomes (2)
Change in vision status
Measured: Three months
To evaluate ocular hemorrhage
Measured: Three months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2008 (estimated)
- Study startJan 2010
- Primary completionJun 2011
- Study completionJun 2011
Why it stopped: Withdrawn due to lack of eligible participants
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell AnemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kresge Eye Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Wayne State University, Genentech, Inc.
- Investigators
- Vinay Shah, MD, Principal investigator, Kresge Eye Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00618644
- Sponsor's protocol ID
- 08-08
- Other IDs
- FVF4232s
- Status last verified
- Sep 2012
- First submitted
- 7 Feb 2008
- Last updated
- 27 Sep 2012
- Results posted
- No
- Data monitoring committee
- No
Publications
Al-Abdulla NA, Haddock TA, Kerrison JB, Goldberg MF. Sickle cell disease presenting with extensive peri-macular arteriolar occlusions in a nine-year-old boy. Am J Ophthalmol. 2001 Feb;131(2):275-6. doi: 10.1016/s0002-9394(00)00778-9.
PubMed 11228315McLeod DS, Merges C, Fukushima A, Goldberg MF, Lutty GA. Histopathologic features of neovascularization in sickle cell retinopathy. Am J Ophthalmol. 1997 Oct;124(4):455-72. doi: 10.1016/s0002-9394(14)70862-1.
PubMed 9323937Witkin AJ, Rogers AH, Ko TH, Fujimoto JG, Schuman JS, Duker JS. Optical coherence tomography demonstration of macular infarction in sickle cell retinopathy. Arch Ophthalmol. 2006 May;124(5):746-7. doi: 10.1001/archopht.124.5.746. No abstract available.
PubMed 16682603Chalam KV, Shah VA. Macular infarction a presentation of sickle cell crisis. Eye (Lond). 2004 Dec;18(12):1277-8. doi: 10.1038/sj.eye.6701409. No abstract available.
PubMed 15105819Avery RL. Regression of retinal and iris neovascularization after intravitreal bevacizumab (Avastin) treatment. Retina. 2006 Mar;26(3):352-4. doi: 10.1097/00006982-200603000-00016. No abstract available.
PubMed 16508438Rosenfeld PJ, Brown DM, Heier JS, Boyer DS, Kaiser PK, Chung CY, Kim RY; MARINA Study Group. Ranibizumab for neovascular age-related macular degeneration. N Engl J Med. 2006 Oct 5;355(14):1419-31. doi: 10.1056/NEJMoa054481.
PubMed 17021318Siqueira RC, Costa RA, Scott IU, Cintra LP, Jorge R. Intravitreal bevacizumab (Avastin) injection associated with regression of retinal neovascularization caused by sickle cell retinopathy. Acta Ophthalmol Scand. 2006 Dec;84(6):834-5. doi: 10.1111/j.1600-0420.2006.00779.x. No abstract available.
PubMed 17083555
Study at a glance
From the registry record, last updated by the sponsor on 27 Sep 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study to Evaluate the Ocular and Non Ocular Safety of Ranibizumab in Treating Neovascularization Secondary to Sickle Cell Retinopathy
- ClinicalTrials.gov ID
- NCT00618644
- Acronym / sponsor ID
- 08-08
- Conditions
- Sickle Cell Anemia, Retinopathy
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Withdrawn due to lack of eligible participants
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Kresge Eye InstituteUniversity, hospital or other
- Collaborators
- Wayne State University, Genentech, Inc.
- Study start
- Jan 2010
- Primary completion
- Jun 2011 (actual)
- Study completion
- Jun 2011 (actual)
- First posted
- 20 Feb 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine the ocular and non-ocular safety of a single dose of ranibizumab in treating neovascularization secondary to sickle cell retinopathy.
Detailed description
In the U.S., about 10% of African Americans have an abnormal hemoglobin gene. About 8% of African Americans are heterozygous for Hemoglobin S. In the United States, sickle cell anemia primarily occurs in the black population, with approximately 0.2% of African American children afflicted by this disease. It may be associated with other hemoglobinopathies as well. The prevalence in adults is lower because of the decrease in life expectancy. Systemically, the sickle cell anemia variation (SS) produces the most symptoms. With respect to the eye, the sickle cell disease mutation (SC) produces the most effects. Overall, the sickle cell trait expression (AS) produces the fewest complications.
- Among patients with SC or SThal, the incidence of proliferation sickle cell retinopathy is 33% and 14% respectively.
- Proliferative sickle cell retinopathy is the major cause of vision loss in sickle cell disease.
For sickle cell retinopathy, the commonly used therapeutic modalities include laser retinal photocoagulation, retinal cryotherapy, and vitrectomy/membranectomy depending on the severity of the disease. The most effective therapeutic modality with minimal postoperative complications appears to be scatter laser retinal photocoagulation.
A single case study of bevacizumab was found to effective in short term regression of neovascularization and improving vision after a single injection. Further study with ranibizumab is warranted.
Recent clinical trials (Marina and Anchor) have demonstrated that ranibizumab is effective in treating patients with CNV with age-related macular degeneration. Retinopathy in sickle cell disease has also been linked to VEGF. Therefore, patients with sickle cell retinopathy should respond to ranibizumab therapy.
This is an open-label single dose, phase I study of intravitreally administered ranibizumab in patients with sickle cell retinopathy.
Consented, enrolled subjects will receive a single open-label intravitreal injection of 0.5 mg ranibizumab.
Three subjects from one site in the United States will be enrolled.
Patients will receive one dose of 0.5 mg ranibizumab administered intravitreally.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- Double blind (participant, investigator)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: RanibizumabRanibizumab injection |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Patients with sickle cell anemia and retinopathy
- Over age 18 years
- Non-pregnant
Key exclusion criteria (4 listed)
- Pregnant
- Glaucoma
- Patients using anticoagulants (e.g., warfarin)
- Retinal detachment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RanibizumabAlso: Lucentis (ranibizumab)Ranibizumab 0.5 mg intravitreal injection | Drug | 1 |
Primary outcome
Ocular safety of a single dose of ranibizumab
Time frame: Three months
2 secondary outcomes
Change in vision status
Time frame: Three months
To evaluate ocular hemorrhage
Time frame: Three months.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Sep 2012.
- Background: PubMed 11228315Al-Abdulla NA, Haddock TA, Kerrison JB, Goldberg MF. Sickle cell disease presenting with extensive peri-macular arteriolar occlusions in a nine-year-old boy. Am J Ophthalmol. 2001 Feb;131(2):275-6. doi: 10.1016/s0002-9394(00)00778-9.
- Background: PubMed 9323937McLeod DS, Merges C, Fukushima A, Goldberg MF, Lutty GA. Histopathologic features of neovascularization in sickle cell retinopathy. Am J Ophthalmol. 1997 Oct;124(4):455-72. doi: 10.1016/s0002-9394(14)70862-1.
- Background: PubMed 16682603Witkin AJ, Rogers AH, Ko TH, Fujimoto JG, Schuman JS, Duker JS. Optical coherence tomography demonstration of macular infarction in sickle cell retinopathy. Arch Ophthalmol. 2006 May;124(5):746-7. doi: 10.1001/archopht.124.5.746. No abstract available.
- Background: PubMed 15105819Chalam KV, Shah VA. Macular infarction a presentation of sickle cell crisis. Eye (Lond). 2004 Dec;18(12):1277-8. doi: 10.1038/sj.eye.6701409. No abstract available.
- Background: PubMed 16508438Avery RL. Regression of retinal and iris neovascularization after intravitreal bevacizumab (Avastin) treatment. Retina. 2006 Mar;26(3):352-4. doi: 10.1097/00006982-200603000-00016. No abstract available.
- Background: PubMed 17021318Rosenfeld PJ, Brown DM, Heier JS, Boyer DS, Kaiser PK, Chung CY, Kim RY; MARINA Study Group. Ranibizumab for neovascular age-related macular degeneration. N Engl J Med. 2006 Oct 5;355(14):1419-31. doi: 10.1056/NEJMoa054481.
- Background: PubMed 17083555Siqueira RC, Costa RA, Scott IU, Cintra LP, Jorge R. Intravitreal bevacizumab (Avastin) injection associated with regression of retinal neovascularization caused by sickle cell retinopathy. Acta Ophthalmol Scand. 2006 Dec;84(6):834-5. doi: 10.1111/j.1600-0420.2006.00779.x. No abstract available.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Vinay Shah, MD principal investigator
Kresge Eye Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Ranibizumab in people with Sickle Cell Anemia and Retinopathy, run by Kresge Eye Institute at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 27 Sep 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Ocular safety of a single dose of ranibizumab (time frame: Three months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00618644, which also lists 7 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Sep 2012.