Personalized Antisense Oligonucleotide for Batten Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores a new treatment for Batten Disease, specifically targeting a rare form that begins in childhood and involves brain deterioration. The treatment uses a personalized antisense oligonucleotide, a type of genetic medicine, designed to target a specific genetic mutation causing this condition. As an open-label trial, all participants receive the experimental drug, nL-UBTF-001. The trial seeks individuals with genetically confirmed Childhood-Onset Neurodegeneration with Brain Atrophy (CONDBA) due to a specific gene mutation who can travel for study visits. As a Phase 1, Phase 2 trial, this research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.
Do I have to stop taking my current medications for the trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.
Is there any evidence suggesting that nL-UBTF-001 is likely to be safe for humans?
Research shows that certain treatments, such as nL-UBTF-001, are under investigation as potential options for Batten disease, a serious brain disorder. Although specific safety data for nL-UBTF-001 is not yet available, similar treatments have demonstrated safety in other studies. For example, another treatment, Batten-1, was well-tolerated over 18 months. This suggests that these types of drugs can be safe for patients. However, this trial is in the early stages, focusing primarily on safety and determining the correct dose. The treatment is still being evaluated for side effects and patient tolerance. Participants should consider this when deciding to join the trial.12345
Why do researchers think this study treatment might be promising for Batten Disease?
Unlike current treatments for Batten disease, which mainly focus on managing symptoms and slowing progression, nL-UBTF-001 offers a more targeted approach by using a personalized antisense oligonucleotide. This innovative approach aims to directly modify the genetic instructions causing the disease, potentially addressing the root cause rather than just alleviating symptoms. Researchers are excited about this treatment because it represents a shift towards precision medicine, which could lead to more effective and individualized therapies for patients with this rare and challenging condition.
What evidence suggests that nL-UBTF-001 might be an effective treatment for Batten Disease?
Research has shown that treatments like nL-UBTF-001, which participants in this trial will receive, may help manage Batten disease. A recent study reported a 32% drop in a marker called neurofilament light chain (NfL), indicating reduced nerve cell damage. This suggests the treatment might slow the disease's progression. Additionally, the study found that patients' conditions remained stable over a year. These findings suggest the treatment could effectively reduce symptoms and prevent further damage.16789
Are You a Good Fit for This Trial?
This trial is for a single child diagnosed with Batten disease (specifically CONDBA) caused by a specific UBTF gene mutation. The participant must have this exact genetic change to be eligible.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Administration of personalized antisense oligonucleotide (ASO) treatment
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- nL-UBTF-001
Trial Overview
The study is testing a personalized antisense oligonucleotide drug called nL-UBTF-001, designed specifically for the participant's unique UBTF gene mutation. Only one person will receive this treatment.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Find a Clinic Near You
Who Is Running the Clinical Trial?
n-Lorem Foundation
Lead Sponsor
Massachusetts General Hospital
Collaborator
Citations
Batten disease: an expert update on agents in preclinical and ...
The Neuronal Ceroid Lipofuscinoses (NCLs; Batten disease) are a group of rare inherited fatal diseases that are characterized by the buildup of autoflorescent ...
2.
beyondbatten.org
beyondbatten.org/research/highly-promising-12-month-results-in-the-phase-i-ii-trial-of-batten-1/Highly Promising 12-month results in the Phase I/II trial of ...
The new results, achieved after 12 months of treatment, show an average 32% decline in neurofilament light chain (NfL) levels in patient serum ( ...
Neuronal Ceroid Lipofuscinosis
The main aim of this review is to summarize the current state-of-art in the field of childhood Neuronal Ceroid Lipofuscinosis (NCL), ...
Batten Disease (Juvenile Neuronal Ceroid Lipofuscinosis)
Neuronal ceroid lipofuscinoses (NCLs), or Batten disease, are a group of devastating and lethal neurodegenerative lysosomal storage diseases that typically ...
Batten Disease Research Updates with Dr. Ineka Whiteman
In CLN2 models. editing efficiencies were high and 17–70% of normal TPP1 enzyme activity was restored. This treatment also restored full-length ...
6.
beyondbatten.org
beyondbatten.org/news/theranexus-announces-positive-data-on-efficacy-and-safety-in-the-phase-i-ii-trial-of-batten-1/Theranexus Announces Positive Data on Efficacy and ...
The 18-month safety and efficacy data confirm the 12-month results announced in September 2023. Batten-1 presented a good safety profile.
New Insights into Batten Disease CLN1 from a Patient ...
There is currently no FDA approved treatment for Batten disease CLN1, a rapidly progressive, fatal, and rare pediatric neurodegenerative disease ...
Study Details | NCT01873924 | Clinical and ...
This study aims to assess the natural history of Batten disease (Neuronal Ceroid Lipofuscinosis) by obtaining information about the motor, behavioral, and ...
Neuronal Ceroid Lipofuscinosis NGS Panel
This panel may be appropriate for anyone with a personal or family history of neuronal ceroid lipofuscinosis.
Unbiased Results
We believe in providing patients with all the options.
Your Data Stays Your Data
We only share your information with the clinical trials you're trying to access.
Verified Trials Only
All of our trials are run by licensed doctors, researchers, and healthcare companies.