Patients will receive standard of care behavior and nutritional interventions. They will not receive cyproheptadine.

Patients will receive standard of care behavior and nutritional interventions. They will receive cyproheptadine every day for a total of two months. Standard dose of 0.25 mg/kg divided BID will be used.

Patients will receive standard of care behavior and nutritional interventions. They will receive cyproheptadine every day for two weeks cycled with no cyproheptadine given for two weeks for a total of two months. Patients on cycled dosing will be given cyproheptadine for two weeks, then no medication for two weeks; repeating this cycle for the two month duration of study

No Cyproheptadine Treatment

Patients will receive standard of care behavior and nutritional interventions. They will receive cyproheptadine every day for a total of two months. Standard dose of 0.25 mg/kg divided BID will be used.
Cyproheptadine

Continuous Cyproheptadine

Patients will receive standard of care behavior and nutritional interventions. They will receive cyproheptadine every day for two weeks cycled with no cyproheptadine given for two weeks for a total of two months. Patients on cycled dosing will be given cyproheptadine for two weeks, then no medication for two weeks; repeating this cycle for the two month duration of study
Cyproheptadine

Cycled Cyproheptadine

Total

Sex: Female, Male

Region of Enrollment

Participants' guardians will complete the Mealtime Behavior Questionnaire (MBQ). This is a validated, 31-item, parent-report questionnaire assessing the mealtime behavior structure in young children above 2 years. The questionnaire measures variables including: 1) food refusals/avoidance; 2) food manipulation; 3) mealtime aggression and 4) choking/gagging/vomiting related to meals. Each behavior is assigned a frequency scale with 1 corresponding to "never" and 5 corresponding to "always." The MBQ then consist of a total score and four subcategory scores (listed above).

Feeding Behavior Questionnaire

Change in skin fold thickness as measured by centimeters and z-score percentiles

Anthropometrics: Skin Fold Thickness

Change in mid-arm circumference as measure by centimeters and z-score percentiles

Anthropometrics: Mid-arm Circumference

Change in BMI as measured by kilograms divided by meters squared and z-score percentiles

Anthropometrics: BMI

Change in height as measured in centimeters and z-score percentiles

Anthropometrics: Height

Change in weight as measured in kilograms and z-score percentiles

Anthropometrics: Weight

Patients will answer affirmative or negative to a descriptive list of side effects commonly experienced when taking cyproheptadine. Examples of these descriptive variables include nausea, emesis, etc. Percentage of patients experiencing each side effect will be calculated and compared across the course of study.

Percentage of Participants Experiencing Side Effects From Cyproheptadine

Change in calorie intake will be recorded

24 Hour Diet Recall

Overall Study

Praveen Goday

Feeding Behaviors

Cyproheptadine

Effects of Cyproheptadine on Growth and Behavior in Pediatric Feeding Disorders

Professor of Pediatric Gastroenterology

Medical College of Wisconsin

Diabetes Mellitus

Stanford University School of Medicine

The Effects of High and Low Blood Glucose Values on the Brain in Children With Type 1 Diabetes Mellitus

Neurocognitive and Neuroanatomical Studies in Young Children With Type 1 Diabetes Mellitus

FSIQ, Memory score, Verbal comprehension score

Tandy Aye

Assistant Professor

Stanford University

William E And Aenid R Weisgerber Foundation

These patients were randomized to receive the placebo in Phase A and study drug (NAC) in Phase B of the trial

Placebo Phase A/NAC Phase B

These patients were randomized to receive study drug (NAC) in Phase A and placebo in Phase B

NAC Phase A/Placebo Phase B

NAC Phase

Placebo Phase

Oxysterol Levels

Dr. Forbes D Porter, Senior Investigator

Niemann-Pick Disease, Type C

900mg effervescent tablet; Dosed as 15 mg/kg/day (maximum dose 900 mg per day) for one week, advanced to 30 mg/kg/day (maximum dose 1800 mg per day) for the second week, and then advanced to 60 mg/kg/day (maximum dose 2700 mg) for the remainder of the trial (6 additional weeks).

cystic fibrosis/癲癇性発作

Effects of Cyproheptadine on Growth and Behavior in Pediatric Feeding Disorders

The Effects of High and Low Blood Glucose Values on the Brain in Children With Type 1 Diabetes Mellitus

Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine

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