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Neurogenic, a biopharmaceutical company developing therapies for Batten disease, is trying to learn more about the progression of the CLN5 subtype disease from a caregiver’s perspective.

To this end, Neurogene invites caregivers of children living with CLN5, or deceased within the past 12 months, to participate in a one-time telephone interview about their experiences, according to an email provided by the company to Batten Disease News.

The goal is to better understand the symptoms of the disorder, its progression and its impact on children and their families, all from the perspective of their caregivers.

The study, called Qualitative interviews with caregivers of children with CLN5-specific Batten disease, is recruiting U.S. caregivers of children with CLN5, a form of late childhood Batten disease. Neurogene collaborates with RTI health solutions (RTI-HS), a non-profit research organization, which will conduct the interviews.

Each participant will be interviewed by telephone by two representatives of RTI-HS for approximately 90 minutes, or an hour and a half. For their efforts, caregivers will each receive a $ 150 electronic gift card.

During the interviews, which will be recorded, participants will be asked to describe their experiences in caring for a child with CLN5. The questions will focus on the caregiver and child’s experiences with the diagnosis of the disease, including the path leading up to it and what happened afterwards. They will be asked about the observed signs and symptoms of the disorder, any developmental delay and the impact of CLN5 on the child, caregivers and family members.

At the end of the study, a summary report will be written based on the interview transcripts, according to Neurogene. The names of participants and other identifying information will not be used. Once the report is written, the audio recordings will be destroyed.

Those interested in participating in the telephone interview are asked to call Danielle, filter at L&E Research, at 720-647-3228. Once qualified for the study, participants will be called for an interview on a day and at a time convenient to them.

Neurogene, which develops therapies for Batten disease and other rare neurological disorders, last month invited families affected by CLN5 to participate in a virtual discussion group about their experiences and perspectives.

The information gleaned from the study and focus group will be used to inform the clinical development plan for Neurogene’s CLN5 gene therapy. The therapy, which aims to provide patients with a working copy of the human CLN5 gene, was designated an orphan drug last July by the U.S. Food and Drug Administration.

Children with CLN5 and CLN7, another form of late infantile batten, are also recruited by Neurogene for a natural history study (NCT03822650). This study, which seeks approximately 30 participants of all ages, will examine the clinical features of the subtypes and the natural progression of symptoms.

Taking place at the University of Rochester Medical Center in New York, this observational study is expected to run until 2026. Those interested in participating should write to [email protected] or call 585-275-4762.

Late infantile Batten disease typically affects children aged 2 to 4 years and is characterized by seizures and loss of motor and cognitive skills. Children with CLN5 often develop normally until the first symptoms, which are usually problems with movement and regression in development. Other features of the disease include loss of vision and speech problems.

Batten disease is thought to affect two to four in 100,000 US residents and about one in 100,000 people worldwide.

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