An extension study of Dasiglucagon in children with CHI

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Zealand Pharma A/S

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

8 Mar 2020 → 6 Dec 2024

Decision date (initial)

2024-08-09

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Zealand Pharma A/S

External identifiers

EU CT number

2024-514007-34-00

EudraCT number

2017-004546-15

ClinicalTrials.gov

NCT03941236

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Dose response, Pharmacokinetic, Therapy, Pharmacodynamic, Efficacy, Safety

To evaluate the long-term safety of dasiglucagon administered as SC infusion in children with CHI.

Secondary objectives 4

1. To evaluate the long-term efficacy of dasiglucagon in reducing hypoglycemia
2. To evaluate the long-term efficacy of dasiglucagon in reducing glucose requirements
3. To evaluate the long-term tolerability of dasiglucagon administered as SC infusion in children with CHI
4. To investigate quality of life (QoL) and resource utilization

Conditions and MedDRA coding

Children with congenital hyperinsulinism

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Completed treatment in either Trial ZP4207-17103 or ZP4207-17109
2. Expected to continue to have a positive benefit-risk assessment for treatment with dasiglucagon (based on considerations of glycemic effect, tolerability, and nature and frequency of AEs experienced in the lead-in trial), with signed investigator statement documenting the positive benefit-risk assessment made
3. Has a negative serum pregnancy test at baseline (only for females of child-bearing potential)
4. Sexually active female patients and their partners must use acceptable contraception or refrain from sexual activity from baseline until 30 days after the last dose of trial drug. Females must abstain from sexual activity that could result in pregnancy or agree to use acceptable methods of contraception. Abstinence can only be accepted if this is true abstinence in line with the preferred and usual lifestyle of the patient. Acceptable methods of contraception are: a) Hormonal contraceptives (e.g., oral contraceptive pill, depot, patch, intramuscular implant or injection, sponge, or vaginal ring), stabilized for at least 30 days if first use or b) Barrier method, e.g., (i) condom (male or female) and (ii) diaphragm with spermicide. Only highly effective methods of birth control are accepted (i.e., one that results in less than 1% per year failure rate when used consistently and correctly, such as implants, injectables, combined oral contraceptives, some intrauterine devices), or sexual abstinence.Only highly effective methods of birth control are accepted (i.e., one that results in less than 1% per year failure rate when used consistently and correctly, such as implants, injectables, combined oral contraceptives, some intrauterine devices), or sexual abstinence
5. Able and willing to comply with trial procedures
6. Following receipt of oral and written information about the trial, the patient (depending on local institutional review board [IRB]/independent ethics committee [IEC] requirements) must provide assent and one or both parents* or guardian of the patient must provide signed informed consent before any trial-related activity is carried out. * 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.

Exclusion criteria 2

1. The patient developed any conditions prohibited by the lead-in trial, requires medication prohibited by the lead-in trial, or has other new complications that preclude participation in the investigator's opinion.
2. Has participated in an interventional clinical trial (investigational or marketed product) within 3 months before baseline or 5 half-lives of the drug under investigation (whichever comes first) or plans to participate in another clinical trial. Excluded from this is participation in Trial ZP4207-17103, Trial ZP4207-17109, and/or 18F-Dopa positron emission tomography computed tomography/magnetic resonance imaging investigation (when performed as a part of a clinical trial) for diagnosis of focal CHI.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Adverse events

Secondary endpoints 6

1. Total amount of gastric carbohydrates administered (nasogastric [NG] tube or gastrostomy) to treat hypoglycemia
2. Time to removal of NG tube or gastrostomy
3. Time to pancreatic surgery (sub-total or total pancreatectomy)
4. CGM percent time <70 mg/dL (3.9 mmol/L)
5. Rate of CGM-detected hypoglycemia episodes <70 mg/dL (3.9 mmol/L) for 15 minutes or more
6. Rate of clinically significant CGM-detected hypoglycemia episodes <54 mg/dL (3.0 mmol/L) for 15 minutes or more

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Zealand Pharma A/S

Sponsor organisation

Zealand Pharma A/S

Address

Sydmarken 11

City

Soeborg

Postcode

2860

Country

Denmark

Scientific contact point

Organisation

Zealand Pharma A/S

Contact name

Clinical Trial Information desk

Public contact point

Organisation

Zealand Pharma A/S

Contact name

Clinical Trial Information desk

Third parties 5

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 8 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications