A Multi-center, Double-Blind, Randomized, Two-Arm, Parallel-Group, Placebo Controlled Study to Assess the Safety of ELGN-2112 in Populations of Interest

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Elgan Pharma Ltd.

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

19 Nov 2025 → ongoing

Decision date (initial)

2024-11-05

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Elgan Pharma

External identifiers

EU CT number

2024-517102-29-00

EudraCT number

2022-004195-42

ClinicalTrials.gov

NCT05904626

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety

To compare the safety of ELGN-2112 to placebo in preterm infants born less than 26 weeks GA and IUGR infants<3rd percentile* born at 26-31+6 weeks GA up to 3 months CA (Part A).
* According to Fenton preterm growth chart

Secondary objectives 14

1. To assess the efficacy of treatment with ELGN-2112 as compared to placebo on intestinal malabsorption in preterm infants as measured by number of days to achieve full enteral nutrition*. *Defined as first day of reaching three consecutive days of EN feeds ≥150 ml/kg/day.
2. To assess effect of ELGN-2112 compared to placebo on the number of days to wean off parenteral nutrition (PN).
3. To assess the effect of ELGN-2112 compared to placebo on the incidence of severe NEC (modified Bell’s stage ≥2a)
4. To assess the effect of ELGN-2112 compared to placebo on the distribution of severity of NEC according to modified Bell’s staging.
5. To assess the effect of ELGN-2112 compared to placebo on the number of days to achieve 120 ml/kg/day of enteral feeds.
6. To assess the effect of ELGN-2112 compared to placebo on the probability of reaching full enteral feeding at different time points
7. To assess the effect of ELGN-2112 compared to placebo on the probability of weaning off PN at different time points.
8. To assess the effect of ELGN-2112 compared to placebo on proportional contribution of EN and PN to total nutrition.
9. To assess the effect of ELGN-2112 compared to placebo on the incidence of late-onset sepsis.
10. To assess the effect of ELGN-2112 compared to placebo on growth of infants (Anthropometrics).
11. To assess the effect of ELGN-2112 compared to placebo on the incidence of any of the adverse events of relevance (NEC, late onset sepsis, death).
12. To assess the effect of ELGN-2112 compared to placebo on the length of stay in the primary hospital.
13. To assess the effect of ELGN-2112 compared to placebo on the number of days to discharge to home.
14. To assess the effect of ELGN-2112 compared to placebo on the incidence and severity of retinopathy of prematurity (ROP).

Conditions and MedDRA coding

Intestinal Malabsorption

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-002116-PIP01-17

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 11

1. Male or female preterm infants born at a gestational age below 26 weeks GA (<26+0) or IUGR infants (below 3rd percentile), born between 26+0 to 31+6 GA*. * Gestational age matching (±2 weeks) between maternal dates and/or early antenatal ultrasound. If both exist and differencediffer from each other > 2 weeks, determination will be based on early antenatal ultrasound
2. Birth weight ≥ 450g
3. Singleton or twin birth
4. Postnatal age up through and including Day 5 (up to 120 hours post birth)
5. Fraction of inspired oxygen ≤ 0.60 at enrolment
6. Infant is cardiovascularly stable at time of enrolment and would be considered unstable if they require inotropic support
7. Infant is able to tolerate enteral feeds (defined as minimum of 10 ml/kg/day)
8. Infant is expected to wean off parenteral nutrition (PN) at the primary hospital
9. Informed consent form signed by parent(s) or legal guardian** ** One or both parents/ legal guardians as required by local regulations
10. In the Investigator’s opinion, the infant is sufficiently stable to partake in the trial to completion
11. (France only) – only participants benefiting from a health insurance plan can participate in research.

Exclusion criteria 14

1. Infant is consuming more than 80 ml/kg /day enterally at study entry
2. Infant is receiving pharmacological treatment for a hemodynamically significant PDA at the time of randomization
3. Heart and chest compression or any resuscitation drugs given to the infant during delivery
4. Infant is not dependent on any parenteral amino acids/lipids as nutrition
5. Major congenital malformation (e.g., infants with genetic, metabolic, and/or endocrine disorder diagnosed before enrolment)
6. For infants born under 26 weeks GA, IUGR is defined as weight for gestational age less than the third percentile according to Fenton preterm growth chart (see Appendix D)
7. Confirmed NEC
8. Maternal diabetes (Type I/II or gestational) requiring insulin during pregnancy or in mothers past medical history
9. a. Confirmed hyperinsulinemia OR b. Suspected hyperinsulinemia requiring glucose administration of more than 12 mg/kg/min at randomization.**.** When calculating the glucose infusion rate include all intravenous glucose sources (i.e. parenteral nutrition and glucose/ dextrose infusions).
10. Any systemic insulin administration at randomization
11. Nothing per os (NPO) at study entry and enteral/oral supplements are not allowed
12. Subjects at risk for significant GI complications such as twin-to-twin transfusion syndrome (TTTS) or monochorionic monoamniotic twins
13. Participation in another interventional clinical study that may interfere with the results of this trial* * Participation in another interventional clinical study that may interfere with the results of this trial is not allowed until discharge from the primary hospital
14. Hypersensitivity to any of the drug components- Recombinant Human Insulin (rh-Insulin), Maltodextrin, Sodium Chloride

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. • Adverse Events (AEs) and Adverse Drug Reactions (ADRs) • Blood glucose • Blood Chemistry and haematology • Vital signs

Secondary endpoints 14

1. The number of days to achieve full enteral feeding, defined as the number of days from treatment initiation to achieve enteral feeding* of at least 150 ml/kg/day for three consecutive days during the treatment period. *Defined as first day of reaching three consecutive days of EN feeds ≥150 ml/kg/day.
2. The number of days from treatment initiation to PN wean-off during the treatment period.
3. Incidence of severe NEC (modified Bell’s stage ≥2a) by 40 weeks post-menstrual age (PMA) or discharge home, whichever is earlier, according to modified Bell’s staging as assessed by central blinded reviewers and classified according to the “Modified Bell’s Staging Criteria for NEC” (Walsh-1986).
4. Distribution of severity of NEC by 40 weeks PMA or discharge home, whichever is earlier, according to modified Bell’s staging as assessed by central blinded reviewers and classified according to the “Modified Bell’s Staging Criteria for NEC” (Walsh MC 1986).
5. The number of days from treatment initiation to the first day the infant achieves EN volume of 120 ml/kg/day for three consecutive days during the treatment period.
6. Percentage of infants reaching FEF for three consecutive days within 9, 12, and 16 days from treatment initiation.
7. Percentage of infants weaned off PN within 7, 10, and 14 days from initiation of treatment.
8. Percentage of EN and PN over total nutrition during the treatment period.
9. Incidence of late onset sepsis (onset ≥72 hours after birth) by 40 weeks PMA or discharge home, whichever is earlier. Will be defined* as either: a) culture proven late onset sepsis OR b) clinically suspected culture negative late onset sepsis * According to EMA Report EMA/477725/2010 on the Expert Meeting on Neonatal and Paediatric Sepsis (8 June 2010)
10. Neonatal anthropometrics measurements during the treatment period and up to 3 months CA.
11. Incidence of at least one of the adverse events of relevance by 40 weeks PMA or discharge home, whichever is earlier: • severe NEC (modified Bell’s stage ≥2a); • late onset sepsis; death
12. The number of days from treatment initiation to discharge to home.
13. Incidence and severity of ROP up to 3 months CA according to the International Classification of Retinopathy of Prematurity (ICROP3)
14. The number of days from treatment initiation to discharge from primary hospital.

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Elgan Pharma Ltd.

Sponsor organisation

Elgan Pharma Ltd.

Address

13 Wadi El Haj

City

Nazareth

Postcode

1603100

Country

Israel

Scientific contact point

Organisation

Elgan Pharma Ltd.

Contact name

Regulatory Department

Public contact point

Organisation

Elgan Pharma Ltd.

Contact name

Regulatory Department

Third parties 9

Locations

6 EU/EEA countries · 17 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

Documents 46 files

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

Application history

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