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Gene Therapy

Gene Therapy for Batten Disease (CLN5-200 Trial)

Phase 1 & 2
Recruiting
Research Sponsored by Neurogene Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Molecular genetic diagnosis of the CLN5 gene
Impaired motor and/or language function and/or impaired visual acuity
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 5 years (multiple visits)
Awards & highlights

CLN5-200 Trial Summary

This trial is testing a gene therapy for children with a rare disease called CLN5.

Who is the study for?
This trial is for children aged 3 to 9 with a confirmed genetic diagnosis of CLN5 Batten Disease who can walk (with or without help) and comply with study procedures like MRI scans. They must not have been in another drug study recently, have certain infections or allergies, need major surgery soon, or be on ventilatory support.Check my eligibility
What is being tested?
The trial tests NGN-101 gene therapy in a single dose escalation format. It's an open-label study where all participants receive the treatment. The aim is to see how different doses are tolerated and how effective they are against CLN5 Batten Disease symptoms.See study design
What are the potential side effects?
Potential side effects may include reactions related to the immune system due to gene therapy, discomfort from procedures required by the trial such as lumbar puncture or MRI under sedation/anesthesia, and general risks associated with gene therapies.

CLN5-200 Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My condition is due to a CLN5 gene mutation.
Select...
I have difficulties with movement, speaking, or seeing clearly.
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I am between 3 and 9 years old.
Select...
I have been diagnosed with CLN5 disease.

CLN5-200 Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~5 years (multiple visits)
This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years (multiple visits) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of Serious Adverse Events (SAEs)
Incidence of Treatment Emergent Adverse Events (TEAEs)
Incidence of clinical laboratory abnormalities
+2 more
Secondary outcome measures
Change in Caregiver global impression of change
Change in Hamburg Scale, Motor and Language domain scores
Change in Spectral Domain-Optical Coherence Tomography (SD-OCT)
+3 more

CLN5-200 Trial Design

3Treatment groups
Experimental Treatment
Group I: Cohort 3Experimental Treatment1 Intervention
The study treatment is a higher dose of recombinant serotype 9 adeno-associated virus encoding a codon- optimized human CLN5 transgene (hCLN5opt).
Group II: Cohort 2Experimental Treatment1 Intervention
The study treatment is a higher dose of recombinant serotype 9 adeno-associated virus encoding a codon-optimized human CLN5 transgene (hCLN5opt).
Group III: Cohort 1Experimental Treatment1 Intervention
The study treatment is a recombinant serotype 9 adeno-associated virus encoding a codon-optimized human CLN5 transgene (hCLN5opt).

Find a Location

Who is running the clinical trial?

Neurogene Inc.Lead Sponsor
5 Previous Clinical Trials
134 Total Patients Enrolled
Xiomara Q. Rosales, MDStudy DirectorNeurogene Inc.
Leslie Jacobsen, MDStudy DirectorNeurogene Inc.
1 Previous Clinical Trials
4 Total Patients Enrolled

Media Library

NGN-101 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05228145 — Phase 1 & 2
Neuronal Ceroid Lipofuscinosis Research Study Groups: Cohort 2, Cohort 1, Cohort 3
Neuronal Ceroid Lipofuscinosis Clinical Trial 2023: NGN-101 Highlights & Side Effects. Trial Name: NCT05228145 — Phase 1 & 2
NGN-101 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05228145 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the projected outcomes of this research endeavor?

"During the span of 5 years, this research study will track and analyse Treatment Emergent Adverse Events (TEAEs) to determine its efficacy. Secondary objectives include a comparative analysis between baseline scores on the Unified Batten Diseases Rating Scale (UBDRS), Hamburg Scale, Motor and Language Domain Scores, as well as Caregiver Global Impression of Change throughout the duration of the trial."

Answered by AI

Do I meet the criteria to join this experiment?

"This medical research is seeking 3 participants, aged between three to nine years old, who are diagnosed with neuronal ceroid-lipofuscinoses. Additional criteria that must be met include: molecular genetic confirmation of the CLN5 gene; impaired motor and/or language functioning; visual acuity impairment; written consent from a parent or guardian (and assent by the participant); compliance with assessments such as laboratory sample collection, lumbar puncture, electroencephalogram etc.; ambulation capabilities even if requiring assistance like braces or walker for support; residency within an hour's drive radius of the study site for 6 months post"

Answered by AI

Is recruitment for this research project still open to participants?

"Clinicaltrials.gov confirms that this medical trial is actively seeking enrolment, having originally been posted on January 31st 2022 and recently updated on November 17th 2022."

Answered by AI

Does the study accept participants who are in their eighties?

"This clinical trial has an age criteria with a 3 year old minimum and 9 years old maximum."

Answered by AI

How many individuals is this trial accommodating?

"Affirmative. The information available on clinicaltrials.gov confirms that this medical trial, which was first listed on January 31st 2022, is actively enlisting individuals for participation. Around 3 volunteers are necessary to be enlisted from 1 distinct healthcare setting."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Gene Therapy Study for Children With CLN5 Batten Disease
2022. "Gene Therapy Study for Children With CLN5 Batten Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05228145.
~4 spots leftby Nov 2028
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