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Methotrexate For The Prevention and Treatment of Proliferative Vitreoretinopathy in Pediatric Patients
Trial ID: NCT04830878
Rhegmatogenous retinal detachment (RRD) is a sight-threatening condition. Children with RRD usually present late with clinical features of longstanding RRD, specifically a serious condition named: proliferative vitreoretinopathy (PVR). Therefore, children with RRD often have poorer outcomes. The objective of this study is to investigate the efficacy and safety of methotrexate in the treatment and prevention of PVR. Methotrexate is a medication that has been used to treat inflammatory conditions in children and adults for a long time and it has been recently used to treat PVR in adults.
A Phase I, Multicenter, Uncontrolled, Open Label Study Assessing the Efficacy and Safety of a Combination of Systemic and Intravitreal Injections of Methotrexate for the Prevention and Treatment of Proliferative Vitreoretinopathy in Pediatric Patients With RheGmatogenous Retinal DetacHmenT (the SIGHT Study)
1. Ability to get informed consent from a parent or a legal guardian of the child and
attend all study visits.
2. Males or females 18 years old or younger.
3. Subject is undergoing vitrectomy for either i. initial retinal detachment with or
without PVR ii. recurrent retinal detachment due to grade A or higher of proliferative
4. Female subjects of childbearing potential must not be pregnant or breast-feeding, must
have a negative serum pregnancy test at screening, and must be willing to undergo
pregnancy tests throughout the study.
5. Female subjects of childbearing potential and male subjects able to father children
must (a) abstain from intercourse throughout the course of the study or (b) agree to
practice acceptable methods of contraception throughout the course of the study (i.e.,
intrauterine device, oral contraceptives, barrier method, or other contraception
deemed adequate by the investigator).
1. Ocular or periocular infection in either eye including (but not limited to):
1. History of herpetic infection in the study eye(s) or adnexa.
2. Presence of known active or inactive toxoplasmosis or toxoplasmosis scar in
3. History of cytomegalovirus infection or clinical evidence of active
cytomegalovirus infection at screening and/or Day 1.
2. Pupillary dilation inadequate for quality stereoscopic fundus photography in the study
3. Media opacity that would limit clinical visualization in the study eye(s) and, in the
opinion of the investigator, could not be repaired or improved during the RD surgery.
4. Other planned eye surgery during the course of the trial
5. Corneal opacity in the study eye(s) that would preclude reliable assessment of the
6. Uncontrolled glaucoma in the study eye(s), evidenced by an intraocular pressure (IOP)
> 21 mmHg while on maximum medical therapy, or chronic hypotony (unmeasurable eye
7. Subjects should not be currently undergoing treatment with one of the following at the
time of RD diagnosis: systemic steroids, methotrexate, azathioprine, or mycophenolate
mofetil (or an equivalent drug, e.g., mycophenolic acid) or other immunomodulatory
8. Malignancy in remission for less than 5 years prior to study participation.
9. Allergy or hypersensitivity to investigational product or other study related
10. Any recent systemic infection (excluding common cold) within 30 days of Day 1.
11. Known to be immunocompromised.
12. History of other disease, metabolic dysfunction, physical examination finding, or
clinical laboratory finding giving reasonable suspicion of a disease condition that
contraindicates the use of an investigational drug, might affect the interpretation of
the results of the study, or renders the subject at high risk for treatment
13. Any uncontrolled systemic disease, except stable syndromic conditions.
14. Females who are pregnant or lactating and females of child-bearing potential who are
not using adequate contraceptive precautions (i.e., intrauterine device, oral
contraceptives, barrier method, or other contraception deemed adequate by the
15. Participation in other investigational drug (oral or topical therapy) or device
clinical trials within 30 days prior to Day 1 and/or participation in other
investigational drug (intravitreal injection therapy) within 3 months or 5 half-lives
(whichever is longer) prior to Day 1 or planning to participate in other
investigational drug or device clinical trials during a time which would overlap with
the duration of the study. This includes both ocular and non-ocular clinical trials.
Exposure to investigational biologics should be discussed with the investigators.
16. In addition, the investigator may declare a subject ineligible for any sound reason.
17. Chest X-ray within 3 months prior to initiation of systemic MTX that shows active
pulmonary diseases of any etiology.
School of Medicine
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Stanford, CA 94305
Edward Wood, MD