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

Status: Recruiting
Location: See location...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY

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).

Eligibility
Participation Requirements
Sex: All
Minimum Age: 1
Maximum Age: 7
Healthy Volunteers: f
View:

• 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

Locations
Other Locations
United Kingdom
Greater Ormond Street Hospital
RECRUITING
London
Contact Information
Primary
Tern Therapeutics Patient Advocacy
patientadvocacy@terntx.com
202-644-8488
Time Frame
Start Date: 2023-05-17
Estimated Completion Date: 2030-07-30
Participants
Target number of participants: 16
Treatments
Experimental: Cohort 1: Main Treatment Arm
2×10\^10 GC/eye
Experimental: Cohort 2: Main Treatment Arm
6×10\^10 GC/eye
Experimental: Expansion Cohort
Expansion cohort, dose level 2×10\^10 GC/eye as determined by Independent Data Monitoring Committee.
Sponsors
Leads: Tern Therapeutics, LLC

This content was sourced from clinicaltrials.gov