Phase 1 Study of C.001 in Retinal Degeneration
A Phase I Single-Center Open-label Study of C.001 in Patients With Significant Retinal Pigment Epithelium Mediated Maculopathy (RMM)
This is a first-in-human Phase 1 study evaluating the safety and tolerability of C.001 in patients with retinal diseases involving degeneration of the retinal pigment epithelium.
Participants will receive a single administration of C.001 delivered by subretinal injection. The study will evaluate three dose levels in a sequential manner.
The primary objective is to assess the safety of C.001 over a 3-month period following administration. Participants will be followed for up to 12 months to further evaluate safety and explore changes in visual function and retinal structure.
Studieoversigt
Status
Betingelser
Intervention / Behandling
Detaljeret beskrivelse
This is a Phase 1, single-center, open-label, dose-escalation study designed to evaluate the safety and tolerability of C.001 administered via subretinal injection.
Participants will be enrolled into three sequential dose cohorts. A sentinel dosing strategy will be used, with safety review by an independent Data Safety Monitoring Board (DSMB) prior to escalation to higher dose levels. A minimum observation period of 4 weeks will be required between dose escalations.
Participants will be followed for up to 12 months after treatment for safety assessments and exploratory evaluation of visual and anatomical outcomes.
Undersøgelsestype
Tilmelding (Anslået)
Fase
- Fase 1
Kontakter og lokationer
Studiekontakt
- Navn: Clinical Trial Administrator
- Telefonnummer: 999-999-9999
- E-mail: trials@celliotherapeutics.com
Studiesteder
-
-
California
-
Beverly Hills, California, Forenede Stater, 90210
- Rekruttering
- Retina Vision Consultants
-
Kontakt:
- Pradeep Prasad, MD
- Telefonnummer: (310) 269-8565
- E-mail: trials@celliotherapeutics.com
-
Palo Alto, California, Forenede Stater, 94301
- Ikke rekrutterer endnu
- Palo Alto Medical Foundation
-
Kontakt:
- Ehsan Rahimy, MD
- Telefonnummer: (650) 853-2974
- E-mail: trials@celliotherapeutics.com
-
Sacramento, California, Forenede Stater, 95825
- Ikke rekrutterer endnu
- Retinal Consultants Medical Group
-
Kontakt:
- David Telander, MD
- Telefonnummer: (916) 454-4861
- E-mail: trials@celliotherapeutics.com
-
-
Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
- Voksen
- Ældre voksen
Tager imod sunde frivillige
Beskrivelse
Inclusion Criteria:
- Age ≥18 years
- Diagnosis of retinal disease associated with retinal pigment epithelium degeneration (e.g., geographic atrophy or Stargardt disease)
- Visual acuity within protocol-defined limits
- Stable ocular condition prior to enrollment
- Medically suitable for ophthalmic surgery and anesthesia
- Willingness to comply with study procedures and follow-up
Exclusion Criteria:
- Active or prior choroidal neovascularization (CNV)
- Recent myocardial infarction or significant uncontrolled cardiovascular disease
- Uncontrolled glaucoma
- Significant ocular inflammatory disease
- Recent intraocular surgery within protocol-defined timeframe
- Active systemic infection
- Poorly controlled diabetes (e.g., HbA1c >8.5%)
- Participation in another investigational study within 3 months prior to screening
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: Ikke-randomiseret
- Interventionel model: Sekventiel tildeling
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
|
Eksperimentel: Arm 1
C.001Low Dose
|
C.001 administered once via subretinal injection
|
|
Eksperimentel: Arm 2
C.001 Medium Dose
|
C.001 administered once via subretinal injection
|
|
Eksperimentel: Arm 3
C.001 High Dose
|
C.001 administered once via subretinal injection
|
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Tidsramme |
|---|---|
|
Incidence of Ocular and Systemic Adverse Events
Tidsramme: 3 months
|
3 months
|
Sekundære resultatmål
Resultatmål |
Tidsramme |
|---|---|
|
Change in visual function measured in EDTRS letters
Tidsramme: 3 months
|
3 months
|
|
Findings of anatomic change on optical coherence tomography (OCT)
Tidsramme: 3 months
|
3 months
|
Samarbejdspartnere og efterforskere
Sponsor
Samarbejdspartnere
Datoer for undersøgelser
Studer store datoer
Studiestart (Faktiske)
Primær færdiggørelse (Anslået)
Studieafslutning (Anslået)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Nøgleord
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- CT-C.001-001
- CLIN2-19416 (Andet bevillings-/finansieringsnummer: California Institute of Regenerative Medicine)
Plan for individuelle deltagerdata (IPD)
Planlægger du at dele individuelle deltagerdata (IPD)?
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
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