Evidence map›Paper›PMID 31918761›Full record

ArticleTrials2020

Evaluation of the safety and tolerability of a nutritional Formulation in patients with ANgelman Syndrome (FANS): study protocol for a randomized controlled trial.

Donna L Herber, Edwin J Weeber, Dominic P D'Agostino, Jessica Duis

Registry-linked trialOpen access · goldAbstract readClinical Trial Protocol
PubMed Full text at PMC Publisher
In one paragraph

Article in Trials, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03644693 (Evaluation of the Safety and Tolerability of a Nutritional Formulation in Angelman Syndrome), which is not on this map. Cited by 6 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed, 1 pooled it
1.6field-weighted citation impact, top 17% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT03644693 nacompletednot on this map

Evaluation of the Safety and Tolerability of a Nutritional Formulation in Angelman Syndrome

TypeinterventionalSponsorUniversity of Colorado, DenverRan2018 to 2020Enrolled15ConditionsAngelman SyndromeArmsNutritional Formulation Containing Exogenous Ketones, Placebo Formulation
3 · Its place in the literature

Who cites it

6 citing papers in PubMed, 1 synthesis or guideline pooled it, 13 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Review
  4. Review
  5. Imprinting disorders.Nature reviews. Disease primers · 2023
    Review
  6. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors at 3 institutions in 1 country.

Donna L HerberDisruptive Nutrition, LLC, 300 West Morgan Street, Suite 1510, Durham, NC, 27701, USA.
Edwin J WeeberPTC Therapeutics, Inc., 100 Corporate Court, South Plainfield, NJ, 07080-2449, USA.
Dominic P D'AgostinoMorsani College of Medicine, Department of Molecular Pharmacology and Physiology, University of South Florida, 12901 Bruce B Downs Blvd, Tampa, FL, 33612, USA.
Jessica DuisDivision of Medical Genetics & Genomic Medicine, Department of Pediatrics, Vanderbilt University Medical Center, Nashville, TN, 37232-2578, USA. jessica.duis@vumc.org.ORCID http://orcid.org/0000-0001-6605-1028
Florida Institute for Human and Machine Cognition · USPTC Therapeutics (United States) · USVanderbilt University Medical Center · US

Funding

Foundation for Angelman Syndrome Therapeutics (US) FAST Trac 2018-001
6 · The paper itself

Abstract

backgroundKetogenic and low-glycemic-index diets are effective in treating drug-resistant seizures in children with Angelman syndrome. Cognition, mobility, sleep, and gastrointestinal health are intrinsically linked to seizure activity and overall quality of life. Ketogenic and low-glycemic diets restrict carbohydrate consumption and stabilize blood glucose levels. The ketogenic diet induces ketosis, a metabolic state where ketone bodies are preferentially used for fuel. The use of exogenous ketones in promoting ketosis in Angelman syndrome has not been previously studied. The study formulation evaluated herein contains the exogenous ketone beta-hydroxybutyrate to rapidly shift the body towards ketosis, resulting in enhanced metabolic efficiency. METHODS/

designThis is a 16-week, randomized, double-blind, placebo-controlled, crossover study to assess the safety and tolerability of a nutritional formula containing exogenous ketones. It also examines the potential for exogenous ketones to improve the patient's nutritional status which can impact the physiologic, symptomatic, and health outcome liabilities of living with Angelman syndrome. DISCUSSION: This manuscript outlines the rationale for a study designed to be the first to provide data on nutritional approaches for patients with Angelman syndrome using exogenous ketones.

trial registrationClinicalTrials.gov, ID: NCT03644693. Registered on 23 August 2018. Last updated on 23 August 2018.

Indexed as

Diet, KetogenicRandomized Controlled Trials as Topic3-Hydroxybutyric AcidAngelman SyndromeCross-Over StudiesDiet, Carbohydrate-RestrictedDouble-Blind MethodGlycemic IndexHumansKetonesNutritional Status3-Hydroxybutyric AcidKetonesAngelman syndromeChildrenExogenous ketoneKetogenic dietKetone esterKetone supplementKetosisPlacebo-controlled trial

Identifiers

PMID31918761
PMCPMC6953273
OpenAlexW3000657063

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.