A Phase 3, Randomized, Double-Blind, Dose-Ranging Evaluation of Transcutaneous Vagus Nerve Stimulation (tVNS) to Reduce Temper Outbursts in People With Prader-Willi Syndrome (PWS)

The goal of the VNS4PWS clinical study is to test the efficacy, safety, and acceptability of transcutaneous vagus nerve stimulation (tVNS) treatment in people with PWS.

开始日期2024-01-08

申办/合作机构

Foundation For Prader-Willi Research, Inc.

[+1]

NCT05939453 / RecruitingN/AIIT

A Randomized, Double-Blind, Controlled Trial of Bright Light Therapy on All-Cause Excessive Daytime Sleepiness in Prader-Willi Syndrome

This is a placebo controlled clinical trial to assess the utility of light therapy as a sufficient treatment for excessive daytime sleepiness in patients with Prader-Willi Syndrome

开始日期2023-10-01

申办/合作机构

Maimonides Medical Center

[+1]

NCT05938543 / RecruitingN/AIIT

Noninvasive Neuromodulation of a Novel Cerebellar Satiety Circuit in Prader-Willi Syndrome

This study uses a noninvasive technique called transcranial magnetic stimulation (TMS) to study hyperphagia and satiety in Prader-Willi syndrome.
TMS is a noninvasive way of stimulating the brain, using a magnetic field to change activity in the brain. The magnetic field is produced by a coil that is held next to the scalp. In this study, the investigators will be stimulating the brain to learn more about how TMS might improve hyperphagia in Prader-Willi syndrome.

开始日期2023-09-01

申办/合作机构

The Brigham & Women's Hospital, Inc.

[+1]

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项与 Foundation For Prader-Willi Research, Inc. 相关的文献（医药）

2024-04-01·Neurotherapeutics

Neuromodulation for the treatment of Prader-Willi syndrome – A systematic review

Review

作者: Ma, Ruoyu ; Strong, Theresa V ; Halpern, Casey H ; Qiu, Liming ; Miller, Jennifer L ; Okun, Michael S ; Gunduz, Aysegul ; Foote, Kelly D ; Chang, Andrew ; Wexler, Anna

Prader-Willi syndrome (PWS) is a complex, genetic disorder characterized by multisystem involvement, including hyperphagia, maladaptive behaviors and endocrinological derangements. Recent developments in advanced neuroimaging have led to a growing understanding of PWS as a neural circuit disorder, as well as subsequent interests in the application of neuromodulatory therapies. Various non-invasive and invasive device-based neuromodulation methods, including vagus nerve stimulation (VNS), transcranial direct current stimulation (tDCS), repetitive transcranial magnetic stimulation (rTMS), and deep brain stimulation (DBS) have all been reported to be potentially promising treatments for addressing the major symptoms of PWS. In this systematic literature review, we summarize the recent literature that investigated these therapies, discuss the underlying circuits which may underpin symptom manifestations, and cover future directions of the field. Through our comprehensive search, there were a total of 47 patients who had undergone device-based neuromodulation therapy for PWS. Two articles described VNS, 4 tDCS, 1 rTMS and 2 DBS, targeting different symptoms of PWS, including aberrant behavior, hyperphagia and weight. Multi-center and multi-country efforts will be required to advance the field given the low prevalence of PWS. Finally, given the potentially vulnerable population, neuroethical considerations and dialogue should guide the field.

2024-03-01·Pharmacogenomics

Pharmacogenomics for Prader-Willi syndrome: caregiver interest and planned utilization

Article

作者: Richards, Jaimie L ; Denton, Jessica J ; Brown, Donna ; Strong, Theresa V ; Bar-Peled, Yael ; Worthey, Elizabeth

Aim: The study aim was to determine caregiver interest and planned utilization of pharmacogenomic (PGx) results for their child with Prader-Willi syndrome. Methods: Caregivers consented to PGx testing for their child and completed a survey before receiving results. Results: Of all caregivers (n = 48), 93.8% were highly interested in their child's upcoming PGx results. Most (97.9%) planned to share results with their child's medical providers. However, only 47.9% of caregivers were confident providers would utilize the PGx results. Conclusion: Caregivers are interested in utilizing PGx but are uncertain providers will use these results in their child's care. More information about provider comfort with PGx utilization is needed to understand how PGx education would benefit providers and ultimately patients with PGx results.

2023-12-01·Molecular Genetics & Genomic Medicine

Caregiver‐based perception of disease burden in Schaaf‐Yang syndrome

Article

作者: Dötsch, Laura ; Matesevac, Lisa ; Strong, Theresa V ; Schaaf, Christian P

AbstractBackgroundSchaaf‐Yang syndrome (SYS) is a neurodevelopmental disorder caused by truncating variants in the paternally expressed MAGEL2 gene in the Prader‐Willi syndrome‐region on chromosome 15q. In addition to hypotonia and intellectual disability, individuals with SYS are frequently affected by neonatal contractures and autism spectrum disorder. In this study, we focus on the burden of disease on patients and their families for the first time.MethodsBased on the online SYS Patient Voices Survey the perspective of 81 primary caregivers on SYS was assessed.ResultsThe perceived severity of muscular and developmental manifestations dominated the evaluation of the phenotype in early childhood, while behavioral issues were considered more impactful later in life. Importantly, an apprehension toward symptoms with a later onset was observed in caregivers of younger children. Available therapeutic options, while mostly effective, did not sufficiently alleviate the total burden of disease. Overall, parents stated that caring for an individual with SYS was very challenging, affecting their daily lives and long‐term planning.ConclusionOur study demonstrates the necessity for treatments that, adapted to age and in accordance with the caregivers' prioritization, improve the patients' medical condition and thus facilitate their and their families' social participation.

项与 Foundation For Prader-Willi Research, Inc. 相关的新闻（医药）

2023-05-05

·Pharmaceutical Technology

Palobiofarma and FPWR partner to develop Prader Willi syndrome therapy

Palobiofarma’s PBF-999 has been developed to treat solid tumours such as colon cancer. Credit: Nemeziya / Shutterstock.com. Spanish biopharmaceutical company Palobiofarma has collaborated with the Foundation for Prader Willi Research (FPWR) to develop its PBF-999 to treat Prader Willi syndrome (PWS). The company has received a new venture philanthropy award as a convertible loan from FPWR. Recommended Reports Reports Von Willebrand Factor (VWF) Drugs in Development by Therapy Areas and Indications, Stages, MoA, R... GlobalData Reports LOA and PTSR Model - Ocrelizumab in Relapsing Remitting Multiple Sclerosis (RRMS) GlobalData View all It intends to utilise the funding from FPWR to partially fund its ongoing Phase II clinical trial of PBF-999, a dual adenosine A2a receptor antagonist/phosphodiesterase 10 (PDE-10) inhibitor. The PDE-10 inhibitor has been developed to treat ‘cold tumours,’ such as colon cancers. It has already been assessed in three clinical trials. Following observations of an appetite-reducing effect using PBF-999, Palobiofarma designed a clinical trial to assess its efficacy and safety in reducing hyperphagia (excessive hunger) in PWS adults. Palobiofarma CEO Dr Julio Castro stated: “We are excited to receive this funding from FPWR, which will enable us to continue our mission of developing a treatment to help patients with PWS.” Led by Dr Assumpta Caixàs, the clinical trial is being conducted at the university hospital Parc Taulí in Sabadell, Spain. FPWR executive director Susan Hedstrom stated: “Effective treatments for PWS hyperphagia are sorely needed and we are pleased to support Palobiofarma’s efforts on this front.” In 2018, Palobiofarma commenced recruitment of participants for a Phase I clinical trial of PBF-999 in patients with solid tumours. Conducted at the Vall D Hebron hospital in Barcelona, Spain, the trial was designed to assess the tolerability and safety of PBF-999.

临床1期临床2期

2023-04-27

·BioSpace

Palobiofarma Receives Venture Philanthropy Award from Foundation for Prader-Willi Research to Advance PBF-999 Development for Prader-Willi Syndrome Treatmenta

[Pamplona, Covina] - The Foundation for Prader Willi Research (FPWR) has provided a new venture philanthropy award, in the form of a convertible loan, to Palobiofarma S.L., a Spanish biopharmaceutical company focused on developing innovative treatments for serious diseases. Palobiofarma has a robust pipeline of promising drug candidates that are currently undergoing clinical development for a range of diseases, including cancer, respiratory, and metabolic disorders supported by an experienced team. FPWR’s philanthropic investment will be used to partially fund Palobiofarma's ongoing Phase 2 clinical trial to develop PBF-999 for the treatment of Prader Willi syndrome (PWS). PBF-999 is a phosphodiesterase 10 inhibitor that has been evaluated in three previous clinical studies. Observations of an appetite-reducing effect have led Palobiofarma to design a clinical study to test this compound in adults with PWS to evaluate safety and efficacy in the reduction of hyperphagia (excessive hunger). The clinical study is being conducted in the University Hospital Parc Taulí in Sabadell, Spain under the leadership of Dra. Assumpta Caixàs. "We are excited to receive this funding from FPWR, which will enable us to continue our mission of developing a treatment to help patients with PWS," said Dr. Julio Castro, CEO of Palobiofarma. "Effective treatments for PWS hyperphagia are sorely needed and we are pleased to support Palobiofarma’s efforts on this front," said Susan Hedstrom, Executive Director of the Foundation for Prader-Willi Research. The Foundation for Prader-Willi Research is a 501(c)(3) non-profit organization dedicated to eliminating the challenges of Prader-Willi syndrome through the advancement of research and therapeutic development. By providing funding and resources to companies like Palobiofarma, FPWR is helping to develop new treatments for people with PWS.

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