CNTF Implants for CNGB3 Achromatopsia
| Status: | Completed |
|---|---|
| Conditions: | Ocular |
| Therapuetic Areas: | Ophthalmology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 11/18/2012 |
| Start Date: | July 2012 |
| End Date: | December 2015 |
| Contact: | Amy E Turriff |
| Email: | turriffa@mail.nih.gov |
| Phone: | (301) 443-2634 |
A Phase I/II Study of the NT-501 Intraocular Implant Releasing Ciliary Neurotrophic Factor (CNTF) in Participants With CNGB3 Achromatopsia
Background:
- Achromatopsia is an inherited condition that causes vision loss because cells in the
retina do not work properly. It causes loss of acuity, sensitivity to light, and loss
of color vision. There are no effective treatments for achromatopsia.
- Four genes currently are known to cause achromatopsia. One of these, the CNGB3 gene, is
the cause in about 50 percent of people.
- CNTF is a natural chemical found in the body that promotes survival and function of
nerve cells. CNTF has been shown to be effective in treating retinal disease in animals
and can slow vision loss.
- CNTF has also been studied in over 250 people with retinal disease other than
achromatopsia. In these studies, a CNTF implant was placed into the eye during a
simple surgery. The implant releases CNTF inside the eye, near the retina. These
studies suggested that a CNTF implant might help vision in some eye diseases.
Objectives:
- To learn whether a CNTF implant is safe for people with CNGB3 achromatopsia.
- To learn whether CNTF can improve visual acuity or color vision, and whether it may
reduce sensitivity to light in people with CNGB3 achromatopsia.
Eligibility:
You may be able to take part in this study if you:
- Are at least 18 years old.
- Test positive for mutations in the CNGB3 gene and have no mutations in another
achromatopsia gene.
- Have 20/100 vision or worse in at least one eye.
- Are not pregnant or nursing.
Design:
- To determine if you can take part, we will ask about your medical history and do a
physical examination and an eye examination. Blood and urine samples will be taken.
- This study requires 11 visits to the National Eye Institute over 3 years.
- One visit will be for the implant surgery. The implant will be placed in one eye only.
- Study visits will take place 1 day after implant surgery, and again 1 week later and 1
month, 3 months, 6 months, 1 year, 1.5 years and 3 years later. These visits will help
us evaluate the safety and benefit of the implant on your eye.
- At the 3 year visit, you can choose to keep the CNTF implant in your eye, or you can
have us remove it.
Objective: The objective of this study is to evaluate the safety of ocular NT-501 device
with encapsulated NT-201 cells releasing Ciliary Neurotrophic Factor (CNTF) to the retina of
participants affected with CNGB3 achromatopsia.
Study Population: Five participants affected with CNGB3 achromatopsia will be enrolled, with
one eye treated per participant.
Design: This is a Phase I/II, prospective, single-center study. One eye of each participant
will receive a vitreous NT-501 device implant releasing CNTF. The study will be completed
once the final participant has received three years of follow-up.
Outcome Measures: The primary outcome is the number and severity of adverse events and
systemic and ocular toxicities at six months post-implantation. Additional safety of ocular
CNTF implants in participants with CNGB3 achromatopsia will be determined from assessment of
retinal function, ocular structure and occurrence of adverse events at all time points.
Secondary outcomes include changes in visual function including visual acuity and color
vision, electroretinogram (ERG) responses, and retinal imaging with optical coherence
tomography (OCT).
- INCLUSION CRITERIA:
To be eligible, the following inclusion criteria must be met, where applicable.
- Participant must be 18 years of age or older.
- Participant must carry two alleles for CNGB3 gene mutations and no CNGA3 sequence
variations as confirmed in a CLIA-certified laboratory.
- Participant must understand and sign the protocol informed consent.
- Both female participants of childbearing potential (see Appendix 1 for definition)
and male participants able to father children must have (or have a partner who has)
had a hysterectomy or vasectomy, be completely abstinent from intercourse, or must
agree to use contraception during the first six months following implantation.
Acceptable forms of contraception include: hormonal contraception (i.e., birth
control pills, injected hormones, dermal patch or vaginal ring), intrauterine device,
barrier methods (diaphragm, condom) with spermicide, or surgical sterilization
(tubal ligation).
EXCLUSION CRITERIA
A participant is not eligible if any of the following exclusion criteria are present.
- Participant has a history of other ocular disease likely to contribute significantly
to visual loss (e.g., optic neuropathy, glaucoma, uveitis, or other retinal disease).
- Participant is judged by the investigator as not sufficiently healthy to safely
undergo ophthalmic surgery.
- Participant is on anticoagulant therapy that cannot be safely stopped
peri-operatively at the implant procedure. Patients on warfarin will always be
excluded. Patients on aspirin will be asked to stop the medication at least seven
days prior to the surgery (when not contraindicated by the underlying medical
condition). The stoppage period for other anticoagulant medications is based on the
best clinical judgment of the investigator surgeon and is variable depending on the
patient's medical condition and the type of medication.
- Participant has had diagnosis or treatment of a malignancy (excluding non-melanoma
skin cancer) within the previous five years.
- Participant has received investigational treatment in another clinical study related
to an ocular condition in the last six months.
- Participant is pregnant, lactating or planning to become pregnant in the first six
months following implantation.
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
Bethesda, Maryland 20892
301-496-4000
National Institutes of Health Clinical Center The National Institutes of Health (NIH) Clinical Center in...
Click here to add this to my saved trials
