N/A N=10 Effects of Exenatide on Overweight Adolescents With Prader-Willi Syndrome
Prader-Willi Syndrome
Primary: Change in Weight — -.5 kg — p=.07
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 3 N=126 A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome
Prader-Willi Syndrome
Primary: Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13) — -5.94; -4.27 score on a scale — p=0.1983
N/A N=39 Early-onset Obesity and Cognitive Impairment in Children With Pseudohypoparathyroidism
Pseudohypoparathyroidism · Albright Hereditary Osteodystrophy
Primary: Intelligence Quotient — 85.9; 104.3; 103.2 units on a scale
Phase 2 N=19 A Study of GLWL-01 in Patients With Prader-Willi Syndrome
Prader-Willi Syndrome
Primary: Post-treatment Total Score on the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) — 15.9; 14.7 score on a scale
Phase 2 N=15 Intranasal Oxytocin for Infants With Prader-Willi Syndrome
Prader-Willi Syndrome
Primary: Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a — 8; 4; 1; 1 Participants — p=0.930
Phase 3 N=56 Effect of Liraglutide for Weight Management in Paediatric Subjects With Prader-Willi Syndrome
Metabolism and Nutrition Disorder · Obesity
Primary: Change in Body Mass Index (BMI) Standard Deviation Score (SDS) From Baseline to 16 Weeks — -0.18; -0.18; -0.50; -0.48 SDS score
Phase 3 N=11 Growth Hormone Use in Adults With Prader-Willi Syndrome
Prader-Willi Syndrome
Primary: Body Composition — 51.2; 48.5; 52.1 percentage of body fat — p=<0.05
Phase 2 N=32 Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Prader-Willi Syndrome
Prader-Willi Syndrome · Hyperphagia
Primary: Change in Efficacy of Peptide Intranasal Oxytocin (IN-OXT) — -6.08; -6.64 score on a scale
Phase 2 N=38 Treatment of Hyperphagia Behavioral Symptoms in Children and Adults Diagnosed With Prader-Willi Syndrome
Hyperphagia in Prader-Willi Syndrome
Primary: Change From Baseline in Hyperphagia in Prader-Willi Syndrome (PWS) Questionnaires- Responsiveness (HPWSQ-R) Total Score at End-of-treatment (Day 15) — -15.6; -8.9 score…
Phase 2 N=23 Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
Prader-Willi Syndrome · Hyperphagia
Primary: Hyperphagia Questionnaire (HQ)- Total Score — 27.91; 30.58; 27.4; 23.33 score on a scale — p=0.059
Phase 2 N=18 Evaluation of Tolerance, Suckling and Food Intake After Repeated Nasals Administrations of Oxytocin in PWS Infants
Prader Willi Syndrome
Primary: Occurence of Adverse Event — 10; 9; 6 number of adverse events
Phase 2 N=65 A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension
Prader-Willi Syndrome
Primary: Excessive Daytime Sleepiness — -3.5; -5.0; -3.9 score on a scale
Phase 2 N=6 Growth Hormone Treatment in Children With Phelan McDermid Syndrome
Phelan McDermid Syndrome
Primary: ABC - Social Withdrawal Subscale — 10.31; 4.6; 14.8; 6.2 score on a scale
Phase 2 N=40 Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome
Prader-Willi Syndrome
Primary: Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE) - Period 2 — 4; 11; 6; 11 Participants
Phase 2 N=12 Understanding the Role of Gut Microbiota in Hyperphagia in Prader-Willi Syndrome
Prader-Willi Syndrome · Obesity
Primary: Microbiome Analysis: Alpha Diversity — 4.89 Shannon index
Phase 3 N=130 Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome
Prader-Willi Syndrome
Primary: Hyperphagia Behavior — -3.439; -5.372; -2.237 score on a scale — p=0.3493
Phase 2 N=23 An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)
Phelan-McDermid Syndrome
Primary: Safety and Tolerability — 17; 17; 1; 3 Participants
Phase 2 N=18 Piloting Treatment With Intranasal Oxytocin in Phelan-McDermid Syndrome
Phelan-McDermid Syndrome
Primary: Change in Aberrant Behavior Checklist — -7.44; -2.42; -5; -1.71 score on a scale
Phase 2 N=9 Co-administration of Tesofensine/Metoprolol in Subjects With Prader-Willi Syndrome (PWS)
Confirmed Genetic Diagnosis of Prader-Willi Syndrome
Primary: Percent Change From Baseline to End of Treatment in Mean Body Weight — -4.09; -0.38; 3.56; 3.00 Percent (%) change in mean body weight — p=0.1045
N/A N=31 Pilot Study of Startle-response Test to Assess Transcranial Direct Current Stimulation-induced Modulation of Hyperphagia in Prader-Willi Syndrome
Hyperphagia · Prader-Willi Syndrome
Primary: Amplitude of Eyeblink Startle Responses — 0.006; 0.001; 0.008; 0.001 microvolts
Phase 2 N=19 Clinical Trial in 22q13 Deletion Syndrome(Phelan-McDermid Syndrome)
22q13 Deletion Syndrome · Phelan-McDermid Syndrome
Primary: Change in Aberrant Behavior Checklist - Social Withdrawal (ABC-SW) Subscale - Study 1 — 15.78; 11.22; 7.61; 9.70 score on a scale
N/A N=70 Intrauterine Environment in Polycystic Ovary Syndrome (PCOS) Probands
Polycystic Ovary Syndrome
Primary: Estradiol Level in Female Offspring — 18606; 6467 pg/ml — p=0.049
Phase 2 N=6 AMO-01 to Treat Adolescents and Adults With Phelan-McDermid Syndrome (PMS) and Co-morbid Epilepsy
Phelan-McDermid Syndrome · Epilepsy
Primary: Number of Adverse Events — 19 events
N/A N=90 Dietary Supplements for the Treatment of Angelman Syndrome
Angelman Syndrome · Nervous System Diseases
Primary: Average Change in Functioning in Specific Areas of Development, Including Speech and Communications Skills, Cognitive Abilities and Daily Living Skills — 5.7; 4.7; 5.8…
Phase 2 N=28 An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001)
Pitt Hopkins Syndrome
Primary: Safety and Tolerability — 15; 0; 4; 12 Participants
N/A N=23 Obstructive Sleep Apnea and Neurocognitive and Cardiovascular Function in Children With Down Syndrome
Down Syndrome · Obstructive Sleep Apnea Syndrome
Primary: Change in Epworth Sleepiness Scale From Baseline to End of Study — -1; -0.5; 1 units on a scale
Phase 3 N=12 Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity
Bardet-Biedl Syndrome · POMC Deficiency Obesity · PCSK1 Deficiency Obesity
Primary: Percentage of Participants With Greater Than or Equal to (≥) 0.2 Reduction of BMI Z-Score From Baseline to Week 52 — 85.7; 80.0 percentage of participants
Phase 3 N=33 Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS
Prader-Willi Syndrome
Primary: Change From Baseline to Month 12 in Lean Body Mass Measured by Dual-Energy X-ray Absorptiometry (DEXA): Adult Cohort — 3.09 Percentage of body mass
N/A N=42 A Pilot Study to Evaluate the Hypoglossal Nerve Stimulator in Adolescents With Down Syndrome and Obstructive Sleep Apnea
Sleep Apnea, Obstructive · Down Syndrome
Primary: Serious Adverse Events — 7 participants
N/A N=17 Use of Partial Body Weight Support Play Environment to Encourage Mobility and Exploration in Infants With Down Syndrome
Down Syndrome · Trisomy 21
Primary: Change in Movement Counts Per 15 Seconds From Baseline (Week 1) to Mid Study (Week 5) — -34.46; -88.62 movement counts per 15 seconds