Interventional Study of Implantation of hESC-derived RPE in Patients With RP Due to Monogenic Mutation

• Register: ClinicalTrials.gov
• Last refreshed on: 11 December 2023
• Main ID: NCT03963154
• Date of registration: 22/05/2019
• Prospective Registration: Yes
• Primary sponsor: Centre d'Etude des Cellules Souches
• Public title: Interventional Study of Implantation of hESC-derived RPE in Patients With RP Due to Monogenic Mutation
• Scientific title: STREAM: A Phase 1/2, Open-label, Safety, Tolerability and Preliminary Efficacy Study of Implantation Into One Eye of hESC-derived RPE in Patients With Retinitis Pigmentosa Due to Monogenic Mutation
• Date of first enrolment: August 19, 2019
• Target sample size: 7
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03963154
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

France

Contacts

• Name: Stéphane BERTIN, MD
• Affiliation: Centre des 15-20

Key inclusion & exclusion criteria

Inclusion Criteria:

- Documented diagnosis of retinitis pigmentosa based on a genetic test confirming the
presence of a monogenic mutation that affects a gene involved in the visual signalling
process specifically at the level of RPEs, namely RPE65 or LRAT, or MerTK

- 18 years old = Age = 65 years old

For patient of the first cohort:

- Visual acuity = 20/200 in the best eye (legally blind)

- For patient of the second cohort:

- 20/63 > Visual acuity > 20/200 in the worst eye And

- Visible photoreceptor outer nuclear layer (ONL) on a spectral domain optical coherence
tomography (OCT) scan

For the two cohorts:

- Negative serum pregnancy test in women of childbearing potential (a woman who is two
years post-menopausal confirmed by a physician or surgically sterile is not considered
to be of childbearing potential)

- Female patients of childbearing potential (if sexually active), committed to use two
methods of contraception starting from the enrollment, during Mycophenolate Mofetil
(MMF) treatment and for 6 weeks after the last dose of MMF

- Sexually active men (including vasectomized men) committed to use condoms during from
the first day of MMF treatment and for at least 90 days after cessation of treatment

- Signed informed consents by the patient or legal guardian(s). For patients unable to
give consent, authorization to participate to the study will be collected close to
their legally authorized representative

- Affiliated to or a beneficiary of a health care system

Exclusion Criteria:

- - Patient unable or unwilling to comply with the protocol requirements

- History of allergy or sensitivity to one of the products used during the study

- Patients with known serious allergies to the fluorescein

- Patients with a contraindication to general anesthesia

- Prior treatment with a gene or cell therapy product

- Patients with chronic hepatitis B or C, i.e. positive hepatitis B surface antigen or
hepatitis C RNA viral load positive

- Patients infected with Human immunodeficiency virus (HIV)

- Anti-HLA antibodies positive at screening

- Pregnancy or breastfeeding

- Presence of any ocular disease or ocular media opacity which in the opinion of the
investigator precludes accurate evaluation

- Participation in another drug or device clinical study within last 6 months prior to
baseline

- Patients known to be affected by pathologies for which the symptoms or associated
treatments can alter the visual function and/or affect the retina

- Systemic corticosteroid therapy or other immunosuppressive / immunomodulating or
anti-retroviral drugs within 2 months prior to baseline

- Patients with a contraindication to immunosuppressive/immunomodulating therapy (MMF)
such as severe chronic renal impairment, severe digestive system disease, Lesch-Nyhan
disease, Kelley-Seegmiller syndrome…

- Acute illness or infection within 4 weeks of the anticipated administration of study
medication which may interfere with study assessments and
immunosuppressive/immunomodulating therapy

- Any other condition or history that, in the opinion of the Investigator, may
compromise the safety or compliance of the patient or would preclude the patient from
successful completion of the study



Age minimum: 18 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Retinitis Pigmentosa

Intervention(s)

Biological: Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (RPE)

Primary Outcome(s)

Safety assessment [Time Frame: From Baseline until Week 56]

Secondary Outcome(s)

Change in leakage or perfusion [Time Frame: At baseline and weeks 24, 48, and 56]

Position of therapeutic patch [Time Frame: From baseline until Week 56]

Change in thickness of RPE layer [Time Frame: At weeks 4, 8, 16, 24, 36, 48 and 56]

Placement of the therapeutic patch [Time Frame: From Baseline until Week 56]

Secondary ID(s)

STREAM

Source(s) of Monetary Support

Please refer to primary and secondary sponsors