Using domperidone to help young people with severe stomach issues

Compassionate Use of Domperidone for Refractory Gastroparesis

This study is testing if the medication domperidone can help young people aged 12 to 21 with severe stomach problems that haven't improved with other treatments.

Quick facts

• Phase: NA
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: 12 Years to 21 Years
• Sex: All
• Sponsor: Children's Mercy Hospital Kansas City (other)
• Locations: 1 site (Kansas City, Missouri)
• Trial ID: NCT04699591 on ClinicalTrials.gov

What this trial studies

This program allows children aged 12 to 21 with refractory gastroparesis and GERD symptoms to receive domperidone after failing standard treatments. Participants will undergo a thorough medical evaluation, including physical exams and ECGs, to ensure safety before starting the medication. Domperidone will be administered four times daily, with regular follow-ups to monitor health and medication effects. The study aims to provide a potential solution for those suffering from severe gastrointestinal motility disorders.

Who should consider this trial

Good fit: Ideal candidates are males and females aged 12 to 21 with severe gastrointestinal symptoms that have not responded to standard therapies.

Not a fit: Patients who have not been evaluated for other causes of their symptoms or those who are not within the specified age range may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the quality of life for young patients suffering from refractory gastroparesis and GERD.

How similar studies have performed: While the use of domperidone has been explored in other contexts, this specific application for refractory gastroparesis in a pediatric population is relatively novel.

Eligibility criteria

Inclusion Criteria:

1. Male or female
2. Age 12 - 21
3. Symptoms or manifestations secondary to GERD (e.g., persistent esophagitis, heartburn, upper airway signs or symptoms or respiratory symptoms), gastrointestinal motility disorders such as nausea, vomiting, severe dyspepsia or severe chronic constipation that are refractory to standard therapy.
4. Patients must have a comprehensive evaluation to eliminate other causes of their symptoms.
5. Patient has signed informed consent for the administration of domperidone that informs the patient of potential adverse events including:

   * increased prolactin levels
   * extrapyramidal side effects
   * breast changes
   * Cardiac arrhythmias including QT prolongation
   * There is a potential for increased risk of adverse events with the drugs and herbal supplements listed in the addendum (See Addendum on pages 24 and 25)
   * The coordinator/investigator will have a discussion with the family about the use of any of the medications and herbal supplements listed in the addendum to make sure they understand the increased risk of their use and the need to contact the investigators prior to using any of the listed medications and herbal supplements.

Exclusion Criteria:

1. History of, or current, arrhythmias including ventricular tachycardia, ventricular fibrillation and Torsade des Pointes. Patients with minor forms of ectopy (PACs) are not necessarily excluded.
2. Clinically significant bradycardia, sinus node dysfunction, or heart block. Prolonged Tc (QTc\> 450 milliseconds for males, QTc\>470 milliseconds for females).
3. Clinically significant electrolyte disorders.
4. Gastrointestinal hemorrhage or obstruction
5. Presence of a prolactinoma (prolactin-releasing pituitary tumor).
6. Pregnant or breast feeding female
7. Known allergy to domperidone

Where this trial is running

Kansas City, Missouri

• Children's Mercy Hospitals and Clinics — Kansas City, Missouri, United States (RECRUITING)

Study contacts

• Principal investigator: Jose Cocjin, MD — Children's Mercy Hospital Kansas City
• Study coordinator: Corey Schurman, MA, CCRC

Conditions: GERD, Gastroesophageal Reflux, Gastroparesis, reflux, acid reflux