BridgeMD
A study to Evaluate the Safety, the Tolerability, the Pharmacokinetics and the Activity of HM15136 administered in pediatric Patients with Congenital Hyperinsulinism (CHI)
EU CTIS ID: 2024-515290-98-00
What this study is testing
Primary Objectives: to evaluate the safety and tolerability profile of HM15136 to evaluate the PK profile of HM15136
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male and female subjects with CHI and persistent hypoglycemia defined as experiencing ≥3 level 1 or level 2 hypoglycemia events per week (blood glucose <70 mg/dL [<3.9 mmol/L]) despite current SoC treatment according to the investigator's evaluation or documentation
- Stable therapy with SoC medications or documented to be nonresponsive to SoC medications with or without nutritional supplementation (nutritional supplementation includes enteral feeding such as gastric carbohydrates [administered orally, via nasogastric tube, or gastrostomy]) as described below: a) Subjects aged ≥12 years on stable therapy with diazoxide or somatostatin analog (ie, octreotide and lanreotide) for at least 3 months prior to screening Note: Subjects treated with octreotide infusion could be included after review by the investigator and medical monitor. b) Children aged 2 to 11 years on stable therapy with diazoxide or octreotide for at least 1 month prior to screening; if on lanreotide, stability should be for at least 3 months prior to screening c) Subjects receiving other medications such as sirolimus or nifedipine could be included after review by the investigator and medical monitor
- Subjects on long-acting somatostatin analog may be enrolled in the study if they have been on stable monthly (every 4 weeks) injections for at least 3 months before screening. Subjects would be required to synchronize their monthly somatostatin analog injection with the first and fifth injection of HM15136 (first dosing date = long-acting somatostatin injection date). Note: Lanreotide users not on every 4 weeks dosing regimen could be enrolled after discussion with the investigator and medical monitor
- HbA1c <7%
- Female subjects of childbearing potential must be nonpregnant and nonlactating. All females (regardless of age) of childbearing potential must have a negative urine/serum pregnancy test and must use highly effective methods of contraception throughout the duration of the study and until 60 days after final dose of HM15136 administration. Male subjects must agree to use condoms as a contraceptive measure throughout the duration of the study and until 60 days after final dose of HM15136 administration.
- Following receipt of oral and written information about the trial, the subject (children) (depending on local Institutional Review Board/Independent Ethics Committee requirements, or local regulatory requirements) must provide an assent and one or both parents (a) or guardians of the subject must provide signed informed consent before any trial-related activity is carried out. Adult subjects must provide signed informed consent. Adult subjects unable to provide informed consent and who require his/her legally authorized representative to provide informed consent will not be enrolled in the study. (a) If required by local regulations, both parents must give their permission unless one parent is deceased, unknown, incompetent, or not reasonably available, or when only one parent has legal responsibility for the care and custody of the child one parent has legal responsibility for the care and custody of the child
You likely can't join if
- With type 1 / type 2 diabetes mellitus
- History of any serious adverse reaction or hypersensitivity to study drug components
- Anemia findings of hemoglobin <10 g/dL in clinical laboratory results at sc
- Abnormal clinical laboratory results at sc: a) History of renal disease/ abnormal kidney function tests at sc: estimated glomerular filtration rate <60 mL/min/1.73m2 as estimated using pediatric formula (Schwartz formula) for subj. <18 y. and chronic kidney disease epidemiology collaboration formula for subj. ≥18 y. b) Clinically significant thyroid function abnormality in opinion of investigator. If sc thyroid stimulating hormone is >1.5 x ULN or <0.4 mIU/L, reflex laboratory testing for T3 and free T4 will be performed c) Clinically significant abnormal hepatic function tests suggestive of hepatic impairment: alanine aminotransferase and/or aspartate aminotransferase >3 x ULN or total bilirubin >1.5 x ULN (unless due to Gilbert's syndrome, total bilirubin >3.0 x ULN and direct bilirubin >1.5 x ULN).
- History of any clinically significant hepatic findings including gallstones (including incidental finding by ultrasonography or other imaging modalities)
- Hypertension or hypotension not on stable dose of supportive medication for at least 3 M prior to Scr
See the full eligibility criteria
Who can join
- Male and female subjects with CHI and persistent hypoglycemia defined as experiencing ≥3 level 1 or level 2 hypoglycemia events per week (blood glucose <70 mg/dL [<3.9 mmol/L]) despite current SoC treatment according to the investigator's evaluation or documentation
- Stable therapy with SoC medications or documented to be nonresponsive to SoC medications with or without nutritional supplementation (nutritional supplementation includes enteral feeding such as gastric carbohydrates [administered orally, via nasogastric tube, or gastrostomy]) as described below: a) Subjects aged ≥12 years on stable therapy with diazoxide or somatostatin analog (ie, octreotide and lanreotide) for at least 3 months prior to screening Note: Subjects treated with octreotide infusion could be included after review by the investigator and medical monitor. b) Children aged 2 to 11 years on stable therapy with diazoxide or octreotide for at least 1 month prior to screening; if on lanreotide, stability should be for at least 3 months prior to screening c) Subjects receiving other medications such as sirolimus or nifedipine could be included after review by the investigator and medical monitor
- Subjects on long-acting somatostatin analog may be enrolled in the study if they have been on stable monthly (every 4 weeks) injections for at least 3 months before screening. Subjects would be required to synchronize their monthly somatostatin analog injection with the first and fifth injection of HM15136 (first dosing date = long-acting somatostatin injection date). Note: Lanreotide users not on every 4 weeks dosing regimen could be enrolled after discussion with the investigator and medical monitor
- HbA1c <7%
- Female subjects of childbearing potential must be nonpregnant and nonlactating. All females (regardless of age) of childbearing potential must have a negative urine/serum pregnancy test and must use highly effective methods of contraception throughout the duration of the study and until 60 days after final dose of HM15136 administration. Male subjects must agree to use condoms as a contraceptive measure throughout the duration of the study and until 60 days after final dose of HM15136 administration.
- Following receipt of oral and written information about the trial, the subject (children) (depending on local Institutional Review Board/Independent Ethics Committee requirements, or local regulatory requirements) must provide an assent and one or both parents (a) or guardians of the subject must provide signed informed consent before any trial-related activity is carried out. Adult subjects must provide signed informed consent. Adult subjects unable to provide informed consent and who require his/her legally authorized representative to provide informed consent will not be enrolled in the study. (a) If required by local regulations, both parents must give their permission unless one parent is deceased, unknown, incompetent, or not reasonably available, or when only one parent has legal responsibility for the care and custody of the child one parent has legal responsibility for the care and custody of the child
- Criteria for Optional Extension Treatment Period: If the subject has not experienced any clinically significant AEs in the opinion of the investigator and sponsor during the 8-week core treatment period, the subject will be given an opportunity to participate in the optional extension treatment period.
What rules you out
- With type 1 / type 2 diabetes mellitus
- History of any serious adverse reaction or hypersensitivity to study drug components
- Anemia findings of hemoglobin <10 g/dL in clinical laboratory results at sc
- Abnormal clinical laboratory results at sc: a) History of renal disease/ abnormal kidney function tests at sc: estimated glomerular filtration rate <60 mL/min/1.73m2 as estimated using pediatric formula (Schwartz formula) for subj. <18 y. and chronic kidney disease epidemiology collaboration formula for subj. ≥18 y. b) Clinically significant thyroid function abnormality in opinion of investigator. If sc thyroid stimulating hormone is >1.5 x ULN or <0.4 mIU/L, reflex laboratory testing for T3 and free T4 will be performed c) Clinically significant abnormal hepatic function tests suggestive of hepatic impairment: alanine aminotransferase and/or aspartate aminotransferase >3 x ULN or total bilirubin >1.5 x ULN (unless due to Gilbert's syndrome, total bilirubin >3.0 x ULN and direct bilirubin >1.5 x ULN).
- History of any clinically significant hepatic findings including gallstones (including incidental finding by ultrasonography or other imaging modalities)
- Hypertension or hypotension not on stable dose of supportive medication for at least 3 M prior to Scr
- Any clinically significant abnormality at sc identified on ECG that in opinion of investigator would affect subject's ability to participate in trial or cardiac arrhythmia requiring medical or surgical treatment within 6 M prior to sc
- History of any active infection, other than mild viral illness within 30D prior to dosing as judged by investigator
- History of/ positive test for hepatitis B surface antigen, hepatitis C virus antibody, or HIV type 1 or type 2 antibody at sc
- Any anticipated procedures (eg surgery) that might interfere with compliance or completion of trial
- Presence of clinically significant physical examination, ECG, or clinical laboratory finding at sc that in opinion of investigator, may interfere with any aspect of study conduct / interpretation of results
- Other reasons for hypoglycemia, including but not limited to druginduced hyperinsulinemic hypoglycemia, exogenous insulin-induced hypoglycemia, insulinoma, insulin resistance syndrome, nonislet cells tumor hypoglycemia, postgastric bypass, postfundoplication for gastroesophageal reflux, or dumping syndrome with postprandial hypoglycemia, or hypoglycemia due to other endocrine or inherited metabolic diseases
- Pregnant female subjects based on sc and baseline pregnancy tests
- With history of major depression, anxiety, or other psychiatric disorders (within last 6 M), requiring active and ongoing medication regimen adjustments including selective serotonin reuptake inhibitors, serotonin norepinephrine reuptake inhibitors, antipsychotics, or lithium.
- Baseline period exclusion criteria include following: - Use of glucagon within 24 hours before 1st HM15136 administration - Significant changes to CHI medications during sc - Less than 3 hypoglycemia events/ W before baseline period
- Criteria for Optional Extension Treatment Period: if subject experiences clinically significant AEs with HM15136 treatment where the risks outweigh expected benefits or has other new complications that in the opinion of the investigator and/or sponsor would preclude continued participation, not be allowed to participate in optional extension treatment period.
- Treatment of CHI with continuous intravenous glucose or glucagon infusion (requiring hospitalization of >1 W) within 1 M prior to screening (sc) (subj. treated with a transient intravenous glucose or glucagon infusion could be included after review by investigator & medical monitor)
- History of or current active disease of any clinically-significant surgical, medical, or psychiatric condition that, in judgment of investigator, might interfere with absorption, distribution/ metabolism of study drug, interpretation of study assessments/ pose an additional safety risk in administering study drug to subj.
- Unable to tolerate adhesive tape or has any unresolved adverse skin reaction in area of CGMS sensor placement
- With current use of any drugs known to interfere with study drug, glucose metabolism/ study procedures (eg, use of systemic glucocorticoids [exclude. topical, intra-articular or ophthalmic application, nasal spray, or inhaled forms] for >10 consecutive days within 3 M prior to Sc / insulin)
- Consumption of alcohol in subj. <18 to 21 y. and heavy drinking in older adults
- Has participated in an interventional clinical trial (investigational or marketed product) within1 M of Sc or 5 half-lives of drug under investigation (whichever comes first)/ plans to participate in another interventional CT
- History of any major surgery within 6 M prior to sc (except for pancreatectomy)
The study team makes the final eligibility decision.
Where it's taking place
- Israel
- United States
- United Kingdom
- Korea, Republic of
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Israel; United States; United Kingdom; Korea, Republic of. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.