Disease · MONDO_0019010

congenital isolated hyperinsulinism

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. congenital isolated hyperinsulinism. Diseases, record MONDO_0019010. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0019010

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Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: congenital isolated hyperinsulinism; Open Targets
Open Targets ID: MONDO_0019010; Open Targets
Other names: Congenital Hyperinsulinism, PHHI, chi, persistent hyperinsulinemic hypoglycemia of infancy; Open Targets
NCI Thesaurus: C122923; NCI Thesaurus
Orphanet: ORPHA:657; Orphanet
Highest stage of its drugs: Phase 3, for 2 of its 7 drugs, from ClinicalTrials.gov; Open Targets

Drugs7

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
ERSODETUG	CHEMBL5314739	Antibody	Phase 3	ClinicalTrials.gov	5	3	3	Drugs and targets
DASIGLUCAGON	CHEMBL4297741		Phase 3	ClinicalTrials.gov	2	2	2	Drugs and targets
DIAZOXIDE	CHEMBL181	Small molecule	Phase 2/3	ClinicalTrials.gov	1	1	1	Drugs and targets
AVEXITIDE	CHEMBL4297949	Protein	Phase 2	TTD	4	3	3	Drugs and targets
EFPEGERGLUCAGON	CHEMBL5314827	Protein	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
SIROLIMUS	CHEMBL413	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets
PASIREOTIDE	CHEMBL3349607	Protein	Unknown	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets9

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes	Source
FKBP1A	Peptidyl-prolyl cis-trans isomerase FKBP1A	1	FK506-binding protein 1A inhibitor	Phase 1/2	https://conductscience.org/library/genes/2280	Drugs and targets
GCGR	Glucagon receptor	1	Glucagon receptor agonist	Phase 3	https://conductscience.org/library/genes/2642	Drugs and targets
GLP1R	Glucagon-like peptide 1 receptor	1	Glucagon-like peptide 1 receptor antagonist	Phase 2	https://conductscience.org/library/genes/2740	Drugs and targets
KCNJ11	ATP-sensitive inward rectifier potassium channel 11	1	Potassium channel, inwardly rectifying, subfamily J, member 11 opener	Phase 2/3	https://conductscience.org/library/genes/3767	Drugs and targets
KCNJ8	ATP-sensitive inward rectifier potassium channel 8	1	Potassium channel, inwardly rectifying, subfamily J, member 8 opener	Phase 2/3	https://conductscience.org/library/genes/3764	Drugs and targets
SSTR1	Somatostatin receptor type 1	1	Somatostatin receptor 1 agonist	Unknown	https://conductscience.org/library/genes/6751	Drugs and targets
SSTR2	Somatostatin receptor type 2	1	Somatostatin receptor 2 agonist	Unknown	https://conductscience.org/library/genes/6752	Drugs and targets
SSTR3	Somatostatin receptor type 3	1	Somatostatin receptor 3 agonist	Unknown	https://conductscience.org/library/genes/6753	Drugs and targets
SSTR5	Somatostatin receptor type 5	1	Somatostatin receptor 5 agonist	Unknown	https://conductscience.org/library/genes/6755	Drugs and targets

Trials12

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT04538989	An Open-Label Multiple Dose Study of RZ358 in Patients With Congenital Hyperinsulinism	Phase 2	23	2025-05-28	Congenital Hyperinsulinism	ERSODETUG	Its record names the disease	ClinicalTrials.gov
NCT00897676	Effect of Exendin-(9-39) on Fasting Adaptation and Protein Sensitivity	Phase 1/2	17	2018-01-18	Congenital Hyperinsulinism	AVEXITIDE	Its record names the disease	ClinicalTrials.gov
NCT00835328	Effect of Exendin (9-39) on Glucose Requirements to Maintain Euglycemia	Phase 1/2	14	2020-05-14	Congenital Hyperinsulinism	AVEXITIDE	Its record names the disease	ClinicalTrials.gov
NCT04172441	Trial Evaluating Efficacy and Safety of Dasiglucagon in Children With Congenital Hyperinsulinism	Phase 2/3	12	2025-03-14	Congenital Hyperinsulinism	DASIGLUCAGON	Its record names the disease	ClinicalTrials.gov
NCT00571324	Effect of Exendin-(9-39) on Glycemic Control in Subjects With Congenital Hyperinsulinism	Phase 1/2	9	2016-11-09	Congenital Hyperinsulinism	AVEXITIDE	Its record names the disease	ClinicalTrials.gov
NCT00994149	Diazoxide In the Management Of Hypoglycemic Neonates	Phase 2/3	100		Infant, Newborn, Diseases, Pregnancy in Diabetics, Infant, Diabetic Mother, Hypoglycemia, Infant, Large for Gestational Age	DIAZOXIDE	Its record names the disease	ClinicalTrials.gov
NCT06208215	RZ358 Treatment for Congenital Hyperinsulinism	Phase 3	56		Congenital Hyperinsulinism	ERSODETUG	Its record names the disease	ClinicalTrials.gov
NCT03941236	Extension Trial Evaluating the Long-term Safety and Efficacy of Dasiglucagon in Children With Congenital Hyperinsulinism	Phase 3	42		Congenital Hyperinsulinism	DASIGLUCAGON	Its record names the disease	ClinicalTrials.gov
NCT04732416	HM15136 (Efpegerglucagon) Treatment for 8 Weeks in Subjects Aged ≥2 Years With Congenital Hyperinsulinism (CHI)	Phase 2	17		Congenital Hyperinsulinism	EFPEGERGLUCAGON	Its record names the disease	ClinicalTrials.gov
NCT02604485	A Single-Dose Open-Label Study of XOMA 358 in Subjects With Congenital Hyperinsulinism	Phase 2	10		Congenital Hyperinsulinism	ERSODETUG	Its record names the disease	ClinicalTrials.gov

1 to 10 of 12

See all 12 trials

Comparisons in its trials3

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

Comparison	Group 1, with the outcome	Group 2, with the outcome	Participants	Trial	Source
Exendin-(9-39) vs Vehicle: deaths from any cause	0 of 16	0 of 16	32	NCT00897676	Trial comparisons
Exendin-(9-39) vs Vehicle: serious adverse events	0 of 16	0 of 16	32	NCT00897676	Trial comparisons
Exendin-(9-39) vs Vehicle: serious adverse events	0 of 9	0 of 9	18	NCT00571324	Trial comparisons

Papers from its trials3

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it	Source
40107271	DERIVED	1	PubMed
35416981	DERIVED	1	PubMed
22855730	DERIVED	1	PubMed

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

Sources 6

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
NLM's MeSHNLM's terms
Open TargetsCC0 1.0
PubMedNLM's terms

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
NLM's MeSHNLM's
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/MONDO_0019010

GET https://conductscience.org/api/diseases/v1/MONDO_0019010/drugs

API reference For AI assistants