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Endocrine & Hormonal

Congenital Hyperinsulinism Clinical Trials and Treatments

Also called Hyperinsulinism (HI), Congenital HI, Persistent Hyperinsulinemic Hypoglycemia of Infancy (PHHI), Hyperinsulinemic Hypoglycemia, Nesidioblastosis (older term), KATP Hyperinsulinism

In congenital hyperinsulinism the pancreas keeps releasing insulin even when blood sugar is low. Insulin also switches off the body's backup fuels, so the brain is starved twice over.

FDA-approved medicines

1

Proglycem

Active trials

4

3 recruiting now

Next FDA decision

None set

FDA calendar

Medicines are those whose current FDA label names Congenital Hyperinsulinism, checked October 14, 2026. Corticosteroids and immune globulins count once each. Download the data

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About Congenital Hyperinsulinism

In congenital hyperinsulinism the pancreas keeps releasing insulin even when blood sugar is low. Insulin also switches off the body's backup fuels, so the brain is starved twice over. It is the most common cause of lasting low blood sugar in babies, and specialists aim to keep glucose above 70 mg/dL, the Pediatric Endocrine Society target.

Diagnosis rests on a blood sample drawn during a low: insulin that is not switched off, suppressed ketones and fatty acids, and a large glucose rise after a glucagon shot. Genetic testing follows quickly because the gene predicts which treatments will work. Children with changes in GLUD1, HNF4A, HNF1A or HADH usually respond to diazoxide; children with 2 recessive KATP gene changes usually have diffuse disease that does not.

About half of diazoxide-unresponsive children have focal disease, a tiny cluster of abnormal cells that forms when a recessive ABCC8 or KCNJ11 change from the father is unmasked by loss of the mother's copy in a few pancreatic cells. An 18F-DOPA PET scan shows the lesion so a surgeon can remove it, and Children's Hospital of Philadelphia reports a 97% cure rate for focal patients. Diffuse disease that medicines cannot control may need a near-total pancreatectomy, which prevents dangerous lows but, Orphanet notes, usually leads to diabetes within 15 years.

Families juggle glucose checks, tube feeds, emergency glucagon and often a continuous glucose monitor. Orphanet reports that thinking and learning can be affected in up to 30% to 50% of patients, so early, aggressive treatment matters.

Common Symptoms of Congenital Hyperinsulinism

Recognizing the signs of Congenital Hyperinsulinism early can lead to faster diagnosis and better outcomes. Symptoms may vary in severity from person to person. If you or a loved one are experiencing any of the following, consider speaking with a specialist.

  • Low blood sugar in a newborn that keeps coming back or needs a lot of sugar water (dextrose) to control
  • Irritability, jitteriness or unusual sleepiness and floppiness in a baby
  • Poor feeding, or feeding constantly because of extreme hunger
  • Fast heartbeat or sweating
  • Abnormal eye movements, staring spells or seizures
  • Being born large for gestational age (high insulin before birth drives growth)
  • In older children: shakiness, weakness, tiredness, confusion and a racing heart when sugar drops
  • Lows triggered by eating protein, in the GLUD1 (HI/HA) form, which also raises blood ammonia
  • Lows after exercise, in the rare exercise-induced form
  • Loss of consciousness or coma if a low goes untreated

Who Congenital Hyperinsulinism Affects

Congenital HI almost always shows up in newborns and infants. NORD reports that about 60% of babies are diagnosed in the first month and another 30% during the rest of the first year, though milder forms can be found later in childhood, and some adults live with ongoing HI or with diabetes after pancreatic surgery. It affects boys and girls equally and occurs in every ethnic group. Rates are higher where marriage between relatives is common, because the most severe forms are recessive.

Most cases are genetic. The 2024 international guideline reports that up to 90% of children who do not respond to diazoxide carry changes in ABCC8 or KCNJ11, the 2 genes that build the beta cell's KATP channel. A separate, temporary form called perinatal stress-induced hyperinsulinism follows a difficult birth, low oxygen or a mother's diabetes and usually resolves within 2 weeks; it is not the same condition, though a few of these babies need diazoxide for weeks to months. HI also appears as part of Beckwith-Wiedemann, Kabuki, Turner and other syndromes.

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Find trials I might fit Answer a few questions and see which open Congenital Hyperinsulinism trials fit your age, location and treatment history.

FDA-Approved Treatments for Congenital Hyperinsulinism

There is currently 1 FDA-approved medication for Congenital Hyperinsulinism. These therapies represent the current standard of care and may be used alongside or compared against investigational treatments in active clinical trials.

diazoxide
Teva Pharmaceuticals (Proglycem); generics from Par Health and e5 Pharma
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Source: openFDA drug labeling data. This list may not include all treatments. Always consult your doctor.

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Congenital Hyperinsulinism Medicines in Clinical Trials

Investigational medicines Trial Friend tracks for Congenital Hyperinsulinism, with trial results, safety findings and where each one stands.

Help Paying for Congenital Hyperinsulinism Treatment

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Side Effect Explorer

Real-world side effect reports from the FDA Adverse Event Reporting System (FAERS). Includes both FDA-approved drugs and investigational therapies from active clinical trials. Click any drug to see what patients reported.

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Genetic Testing

Genetic testing can confirm a diagnosis, guide treatment decisions, and identify family members who may be at risk.

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Trusted Congenital Hyperinsulinism Resources

Reputable organizations and medical references for learning more about Congenital Hyperinsulinism, including disease registries, foundation resources, and clinical guidelines.

Active Clinical Trials for Congenital Hyperinsulinism

Use this Congenital Hyperinsulinism clinical trial finder to see the 3 studies recruiting patients and 1 opening soon in the United States and worldwide, with eligibility criteria in plain English. All of them test a treatment. These studies play a critical role in advancing care for endocrine & hormonal conditions and may offer access to treatments not yet widely available. Each trial below is sourced directly from ClinicalTrials.gov, with eligibility criteria translated into plain English to help patients and caregivers evaluate whether a study may be a fit.

New to trials? What Placebos and Randomization Actually Mean for You (In Plain English) · 4 min readWorried about cost? The Hidden Costs of Joining a Clinical Trial (And How to Get Help Paying for Them) · 4 min read
TrialsSite mapPipeline timelineMedication checker

Note: Trial recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator before making plans.

4 active trials worldwide
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NOT YET RECRUITINGTreatment trialRecently updatedNCT07795047

Umbilical Cord Glucose

Intervention: For the screening, the interventions will be glucose measurements (and possibly G-BOBH), while follow-up assessments will include Bayley testing, EEG, and MRI.

Sponsor: Odense University Hospital · Radiometer Medical ApS

Congenital hyperinsulinism (HI) causes persistent neonatal hypoglycemia that, if untreated, can lead to neurodevelopmental impairments (NDI) such as cognitive delays, cerebral palsy, epilepsy, and blindness. Despite being preventable with early diagnosis and treatment, 30-50% of ...

Ages up to 8 Months
Expected to start Sep 2026Updated 1 month agoEst. Aug 2028 (~1y 10m)
RECRUITINGTreatment trialPhase 2Updated a few months agoNCT04205604

18FluoroLDOPA PET Imaging for the Detection and Localization of Focal Congenital Hyperinsulinism

Intervention: 18F-Fluoro Dopa Imaging

Sponsor: Miguel Pampaloni

The goal of this project is to determine the role of FDOPA/PET as a pre-operative diagnostic imaging procedure for differentiating focal and diffuse forms of congenital hyperinsulinism and locating focal lesions in the pancreas to guide surgical resection.

Ages 0 Days to 18 Years1 location
Started Nov 2016Updated 5 months agoEst. Dec 2026 (~1 month)
RECRUITINGTreatment trialPhase 3No updates in a whileNCT04706910

18F-DOPA II - PET Imaging Optimization

Intervention: 18F-DOPA, Furosemide Injection

Sponsor: University of Alberta

A single centre non-randomized, non-blinded phase III prospective cohort study of 18F-DOPA PET/CT imaging in specific patient populations:

1. Pediatric patients (less than 18 years old) with congenital hyperinsulinism.

Ages not specified1 location
Started Jan 2021Updated 8 months agoEst. Dec 2027 (~1y 2m)
RECRUITINGTreatment trialPhase 1Hasn't posted an update in over a yearNCT02021604

Fluorodopa F 18 in Congenital Hyperinsulinism and Insulinoma

Intervention: Fluorodopa F 18

Sponsor: Cook Children's Health Care System

Low blood sugars are known to cause brain damage in newborn babies. One of the most common causes of low blood sugars persisting beyond the new born period is a condition called congenital hyperinsulinism (HI). This is a disease whereby the pancreas secretes too much insulin and ...

Ages up to 18 Years1 location
Started Oct 2013Updated 2 years agoEst. Jan 2028 (~1y 3m)
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Active trial locations2 cities in the US

Trial Pipeline

Jan 2021 to Aug 2028
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Can My Child Join a Congenital Hyperinsulinism Clinical Trial While on Current Medications?

This medication conflict checker helps families of children with congenital hyperinsulinism (HI) find out whether current treatments could affect clinical trial eligibility. Select one or more medications below to screen active HI trials for potential conflicts.

Most HI trials are add-on studies: the child stays on diazoxide, octreotide or feeds and the study drug is added on top, so a current medicine is more often a requirement than a barrier. Trials usually ask for a stable dose for a set number of weeks before enrollment, and imaging studies of 18F-DOPA PET have their own rules about recent surgery and sedation. Each trial publishes its own criteria, and they differ.

Diazoxide
Proglycem, generic diazoxide oral suspension — the first-line HI medicine and the usual backbone of standard of care in add-on trials. Studies more often require a stable diazoxide dose for several weeks than exclude it. Some trials enroll only diazoxide-unresponsive children, so a documented response or non-response can decide eligibility either way.
Somatostatin Analogs
octreotide (Sandostatin), octreotide LAR, lanreotide (Somatuline Depot) — allowed as background therapy in most add-on trials if the dose has been stable, since these are standard second-line HI treatments. Trials of new glucagon-type drugs may ask about octreotide because both change liver glucose output. Long-acting monthly shots may need to be timed around study visits.
Glucagon and Dextrose
glucagon rescue kits, IV dextrose, continuous tube feeds of dextrose or maltodextrin — rescue glucagon is almost always permitted and often required as an emergency plan. Trials of glucagon analogs (dasiglucagon, efpegerglucagon) count rescue glucagon use as an outcome rather than a conflict. Children who depend on IV dextrose are usually studied in hospital-based trials with their own rules.
Diuretics and Other Supportive Medicines
chlorothiazide, hydrochlorothiazide, anti-seizure medicines, reflux medicines — the water pill prescribed with diazoxide is rarely an issue. Anti-seizure medicines are usually allowed if the dose is stable; a few trials exclude children with uncontrolled seizures rather than the drugs themselves. Phenytoin can lose effect when combined with diazoxide, which is a prescribing issue, not a trial issue.
Drugs the 2024 guideline advises against may be flagged
The international HI guideline says sirolimus should not be used outside an approved study and that glucocorticoids are not effective and should be avoided. Trials often exclude both, and sirolimus usually needs a washout. If your child takes either, the checker may flag it at low confidence; the study team will want to know why it was started.
A conflict often means a timing rule, not a no
HI trials typically require a stable dose of diazoxide or octreotide for a set period before the first study dose, and surgery or 18F-DOPA PET studies may require a gap after a recent pancreatectomy. The checker shows the trial's own wording so you can see whether it is a washout, a stability window or a true exclusion.
After a pancreatectomy
Children who still have lows after a near-total pancreatectomy were specifically included in the dasiglucagon and ersodetug trials, and children who developed diabetes after surgery are sometimes eligible for different studies. Insulin or pancreatic enzyme replacement started after surgery is usually allowed; tell the study team about every post-surgical medicine.
Don't see your child's medication listed?
The checker covers the medicines most relevant to HI families: diazoxide, somatostatin analogs, glucagon, diuretics, anti-seizure drugs and common supportive medicines. If a medicine is not in the list, it has not come up often in HI trial exclusion criteria. Always tell the study team about everything your child takes, including feeds and supplements.
How the medication conflict checker works: This free tool helps HI families learn whether current medications could affect clinical trial eligibility. It scans the published eligibility criteria of every active congenital hyperinsulinism trial and flags those that may exclude a specific treatment. Matches are grouped by confidence: high confidence means the trial names the exact drug, medium means it references the drug class, and low means it uses broad language that may or may not apply. Always confirm eligibility with the study team, since final decisions depend on the child's gene result, diazoxide response, surgical history and the HI center's assessment.

Across 1,853 open rare disease treatment trials, a third exclude people over a medication they commonly take. See which medications and diseases, in our September 2026 analysis.

Sources: International Guidelines for the Diagnosis and Management of Hyperinsulinism (Horm Res Paediatr, 2024); Proglycem prescribing information (Rev. 10/2026); ClinicalTrials.gov records NCT06208215, NCT03941236, NCT04732416, NCT05088798, NCT04205604, NCT02021604.

Data from ClinicalTrials.gov, U.S. National Library of Medicine.
Always talk to your doctor before considering a clinical trial.

Patient Communities

Connect with other Congenital Hyperinsulinism patients, caregivers, and advocacy groups across Facebook groups, Reddit communities, and YouTube channels. These patient communities offer peer support, shared experiences, caregiver resources, and real-time discussion about Congenital Hyperinsulinism treatments, clinical trial participation, and day-to-day disease management.

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Congenital Hyperinsulinism International
Parent-founded global nonprofit (2005) in Glen Ridge, NJ: family support, mentors, help obtaining medicines and supplies, a CGM access program and free genetic testing through the Open HI Genes Project
CHI Parents' Forum (private group)
Private Facebook group moderated by Congenital Hyperinsulinism International where parents worldwide ask questions and support each other
HI Global Registry
Patient-powered natural history study open to adults with HI, parents and caregivers, and people whose HI resolved or became diabetes; surveys in 8 languages
CHI Centers of Excellence
The 8 centers recognized by Congenital Hyperinsulinism International in 2024, including CHOP, Cook Children's and Great Ormond Street, all offering genetic testing, rapid pathology and trial access
CHI Mentors
Volunteer parents and adults with HI in the US, UK and Netherlands who talk with newly diagnosed families
Congenital Hyperinsulinism International on Facebook
Organization page with news, awareness campaigns and event announcements
Congenital Hyperinsulinism International on YouTube
Family conference talks and educational videos from the organization
CHOP Congenital Hyperinsulinism Center
The largest HI center in the world, with more than 1,900 children treated since 1998, 18F-DOPA PET scanning and family resources including a school guide
Cook Children's Hyperinsulinism Center
Fort Worth center led by Dr. Paul Thornton with 18F-DOPA PET-CT, a dedicated HI unit and parent guides
Children's Hyperinsulinism Charity (UK)
UK charity supporting families, linked to the NHS services at Great Ormond Street and Manchester
NORD Rare Disease Report
Reviewed plain-language medical overview from the National Organization for Rare Disorders, written with CHI, CHOP and Cook Children's clinicians

Events

Events run by Congenital Hyperinsulinism International, the parent-founded nonprofit that organizes the HI family conferences. The 2026 family conferences (Antalya, Turkey, in May and Marseille, France, in September) have already taken place and 2027 dates were not posted when this page was checked, so the one upcoming listing is the organization's annual fundraiser.

The Highlawn
Congenital Hyperinsulinism International
Congenital Hyperinsulinism InternationalFrom congenitalhi.org, checked October 18, 2026
Fundraiser

Sugar Soiree 2026 (Congenital Hyperinsulinism International)

When
Saturday evening, November 21, 2026 (start time on the ticket page)Every year
Where
The Highlawn, West Orange, NJ
Cost
Ticketed; price shown on the registration form
For
Families, supporters and clinicians; a fundraiser rather than an education day

Congenital Hyperinsulinism International's signature annual fundraiser, held at The Highlawn in West Orange, New Jersey, and emceed this year by ABC News correspondent Cheri Preston. Proceeds support the organization's family programs, research and advocacy for people with hyperinsulinism.

Tickets are sold through the organization's online form, which also shows the price. CHI arranged hotel blocks at the Cambria Hotel West Orange and The MC Hotel Montclair with a reservation deadline of October 19, 2026.

Details come from Congenital Hyperinsulinism International's own pages (congenitalhi.org/events and congenitalhi.org/2026-sugar-soiree), checked October 18, 2026. The ticket price is shown only inside the registration form. Confirm times and prices on the organizer's page before you go.

Related Conditions

Conditions that share genes, symptoms or treatments with Congenital Hyperinsulinism, followed by others in the endocrine & hormonal category. Patients with Congenital Hyperinsulinism may find relevant research, shared treatment pathways, or overlapping clinical trials among these related conditions.

Companies Developing Congenital Hyperinsulinism Treatments

1 pharmaceutical company has Congenital Hyperinsulinism in their rare disease portfolio

Frequently Asked Questions About Congenital Hyperinsulinism