Retinitis Pigmentosa ve Leber Konjenital Amorozisi için OCU400'ün Güvenliliğini ve Etkinliğini Değerlendirme Çalışması

Sade dilde özet

Bu çalışma, OCU400 adlı bir gen terapisini, retinitis pigmentosa ve Leber konjenital amorozisi (Leber congenital amaurosis) gibi kalıtsal göz hastalıkları olan kişilerde inceliyor. Çalışmaya, NR2E3 ya da RHO gen mutasyonu bulunan retinitis pigmentosa hastaları ile CEP290 mutasyonu bulunan Leber konjenital amoroz hastaları (yetişkinler ve 6-17 yaş çocuklar) alınmaktadır. Çalışma Faz 1/Faz 2 aşamasında yürütülmüş; şu an katılımcı almamakta ancak devam etmektedir (active, not recruiting).

Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.

Deney bilgileri

Faz

PHASE1/PHASE2

Durum

Devam ediyor, katılım kapalı

Sponsor

Ocugen

Müdahale

OCU400 Low Dose, OCU400 Med Dose, OCU400 High Dose, OCU400 Second Eye Dosing

Başlangıç

24.01.2022

Tahmini bitiş

01.03.2027

Ülkeler

United States

İlgili genler

Merkezler

• Phoenix, United States
• La Jolla, United States
• Miami, United States
• Atlanta, United States
• Portland, United States
• Philadelphia, United States
• Dallas, United States

Uygunluk kriterleri

Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.

Diagnosis and main criteria for inclusion:

Subjects meeting all inclusion criteria and none of the exclusion criteria are eligible for study participation.

Inclusion Criteria for Adult RP:

1. Males or females ≥ 18 years of age at the time of informed consent.
2. Confirmed genetic diagnosis of biallelic autosomal recessive NR2E3 mutations or autosomal dominant NR2E3 mutation for Subgroup 1 or autosomal dominant RHO mutations for Subgroup 2.
3. For the sentinel subject of Cohort 1-3, BCVA ≤ 20/160 in study eye or visual field less than 20° in any meridian, as measured by a III4e isopter or equivalent in study eye.
4. For non-sentinel subject, BCVA ≤ 20/50 or visual field less than 20° in any meridian, as measured by a III4e isopter or equivalent in study eye.
5. Able to perform a Multi-Luminance Mobility Testing (MLMT) using study eye, but unable to pass the MLMT at 1 lux, the lowest luminance level tested.

Exclusion Criteria for Adult RP:

1. Subject lacks evidence of outer nuclear layer.
2. Considered unsuitable for any reason that may either place the subject at increased risk during participation or interfere with the interpretation of the study outcomes by the Investigator, or the Sponsor after reviewing medical, ocular, and psychiatric history, clinical examination, and laboratory evaluation, as determined by the Investigator.
3. Previous treatment with a gene therapy or cell therapy product.
4. Previous treatment with any investigational drug or device within one year.
5. Any contraindications for subretinal injection.
6. Cataract Surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months.
7. Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception within the Treatment Observation Period.
8. Any medical condition with life expectancy \< 6 years.

Inclusion Criteria for Adult LCA:

1. Males or females at least 18 years of age at the time of informed consent.
2. Clinical diagnosis of LCA and confirmed genetic diagnosis of CEP290 mutation.
3. Best corrected visual acuity (BCVA) equal to or worse than LogMAR +0.7 but equal to or better than LogMAR 3.8 (light perception) in the study eye.
4. Detectable outer nuclear layer in the macular region as determined by spectral-domain optical coherence tomography (SD-OCT).

Exclusion Criteria for Adult LCA:

1. Any symptom of central nervous system involvement/disease that would impact the ability to measure visual function.
2. Considered unsuitable for any reason that may either place the patient at increased risk during participation or interfere with the interpretation of the study safety and efficacy outcomes by the Investigator, after reviewing medical, ocular, and psychiatric history, clinical examination, and laboratory evaluation, as determined by the Investigator.
3. Any contraindications for subretinal injection.
4. Any intraocular surgery within 6 months.
5. Active ocular/intraocular infection (e.g., conjunctivitis, keratitis, scleritis, endophthalmitis).
6. Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception within the Treatment Observation Period.

Inclusion Criteria for Pediatric RP:

1. Males or females 6 - 17 years of age (inclusive) at the time of parental permission and/or assent, whichever is applicable.
2. Confirmed genetic diagnosis of biallelic autosomal recessive NR2E3 mutations or autosomal dominant NR2E3 mutation for Subgroup 1 or autosomal dominant RHO mutations for Subgroup 2.
3. BCVA ≤ 20/32 or visual field less than 20° in any meridian, as measured by a III4e isopter or equivalent in study eye.
4. Able to perform a Multi-Luminance Mobility Testing (MLMT) using study eye, but unable to pass the MLMT at 1 lux, the lowest luminance level tested.

Exclusion Criteria for Pediatric RP:

1. Subject lacks evidence of outer nuclear layer as determined by spectral-domain optical coherence tomography (SD-OCT).
2. Considered unsuitable for any reason that may either place the subject at increased risk during participation or interfere with the interpretation of the study outcomes by the Investigator, or the Sponsor after reviewing medical, ocular, and psychiatric history, clinical examination, and laboratory evaluation, as determined by the Investigator.
3. Previous treatment with a gene therapy or cell therapy product.
4. Previous treatment with any investigational drug or device within one year.
5. Any contraindications for subretinal injection.
6. Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months.
7. Breast-feeding, pregnancy, or inability to practice strict contraception within the Treatment Observation Period for subjects of childbearing potential.
8. Active ocular/intraocular infection (e.g., conjunctivitis, keratitis, scleritis, endophthalmitis).
9. Any medical condition with life expectancy \< 6 years.

Inclusion Criteria for Pediatric LCA:

1. Males or females 6 - 17 years of age (inclusive) at the time of parental permission and/or assent, whichever is applicable.
2. Clinical diagnosis of LCA and confirmed genetic diagnosis of CEP290 mutation.
3. Best corrected visual acuity (BCVA) equal to or worse than LogMAR +0.7 but equal to or better than LogMAR 3.8 (light perception) in the study eye.
4. Detectable outer nuclear layer in the macular region as determined by spectral-domain optical coherence tomography (SD-OCT).

Exclusion Criteria for Pediatric LCA:

1. Any symptom of central nervous system involvement/disease that would impact the ability to measure visual function.
2. Considered unsuitable for any reason that may either place the subject at increased risk during participation or interfere with the interpretation of the study safety and efficacy outcomes by the Investigator, after reviewing medical, ocular, and psychiatric history, clinical examination, and laboratory evaluation, as determined by the Investigator.
3. Any contraindications for subretinal injection.
4. Any Intraocular surgery within 6 months.
5. Active ocular/intraocular infection (e.g., conjunctivitis, keratitis, scleritis, endophthalmitis).
6. Breast-feeding, pregnancy, or inability to practice strict contraception within the Treatment Observation Period for subjects of childbearing potential.