A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy with TTX-381 for the Ocular Manifestations Associated with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease
This is a first-in-human, open-label, single ascending dose study of TTX-381 for the treatment of ocular manifestations of CLN2 (Batten disease).
• A participant is eligible to be included in the study only if all of the following criteria apply:
• Has biallelic CLN2 mutations.
• Has decreased leukocyte TPP1 activity.
• Has clinical signs or symptoms consistent with CLN2 disease (eg, developmental delay, developmental decline, seizure, vision loss, or other signs/symptoms) OR an older sibling with confirmed CLN2 diagnosis.
• Is currently receiving biweekly ICV ERT treatment with cerliponase alfa.
• Meets the following baseline disease condition according to age and CRT as assessed by SD-OCT and confirmed by CRC:
• Participants in the phase of accelerated decline in CRT:
⁃ CRT at baseline ≤210 μm and
⁃ CRT at baseline ≥140 μm in both eyes and
⁃ Age ≤84 months,
∙ Is willing to adhere to the protocol and 5-year visit schedule.
‣ Sexually active female participants of childbearing potential (following menarche) or fertile male participants (following puberty) must be willing to use a medically accepted form of contraception from Screening Visit 2 until 6 weeks after vector administration.
• OR
• Was previously administered TTX-381.
• Upon retrospective review, met the above criteria at the time of administration of TTX-381. IDMC may consider exceptions to this when weighing whether to retrospectively enroll a participant who has received TTX-381.
• Has been recommended for enrollment into the clinical trial by IDMC