Study to Assess the Long-term Safety of Dupilumab Administered in Participants ≥6 Months to <18 Years of Age With Atopic Dermatitis (AD).

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Regeneron Pharmaceuticals Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Skin and Connective Tissue Diseases [C17]

Trial duration

9 Nov 2015 → ongoing

Decision date (initial)

2024-10-01

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Regeneron Pharmaceuticals Inc.

External identifiers

EU CT number

2023-509425-53-00

EudraCT number

2015-001396-40

ClinicalTrials.gov

NCT02612454

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

To assess the long-term safety of dupilumab in pediatric patients with Atopic Dermatitis (AD).
Optional Pre-filled Pen (PFP) Sub Study in pediatric patients ≥2 to <12 years of age with AD:
Co-Primary Objectives are:
- To evaluate the pharmacokinetic (PK) of dupilumab PFPs
- To evaluate the safety of dupilumab PFPs

Secondary objectives 3

1. To assess the long-term efficacy of dupilumab in pediatric patients with AD.
2. To assess the trough concentrations of functional dupilumab in serum and immunogenicity in pediatric patients with AD after re-treatment with dupilumab.
3. OPTIONAL SUB-STUDY: To evaluate the immunogenicity of dupilumab PFPs

Conditions and MedDRA coding

Atopic Dermatitis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Male or female, ≥6 months to <18 years of age at the time of screening
2. Participated in a prior dupilumab study in pediatric participants with AD and adequately completed the visits and assessments required for both the treatment and follow-up periods, as defined in the prior study protocol
3. PFP Sub-study Only: Age ≥2 to <12 years at time of screening
4. PFP Sub-study only: Body weight ≥5 kg and <60 kg at time of screening
5. PFP Sub-study only: Must have received the same dupilumab dose regimen to be used in the PFP sub-study during the previous 12 weeks in the main OLE study using the prefilled syringe, as defined in the protocol
6. Note: Other Protocol Defined Inclusion Criteria Apply

Exclusion criteria 11

1. Participants who, during their participation in a prior dupilumab study developed an adverse event (AE) or serious adverse event (SAE) deemed related to study drug which could indicate that continued treatment with study drug may present an unreasonable risk for the patient
2. Participants, who during the participation in a prior Dupilumab study, developed an AE that was deemed related to study drug and led to study treatment discontinuation, which in the opinion of the investigator or medical monitor could indicate that continued treatment with study drug may present an unreasonable risk for the patient
3. Treatment with an investigational drug, other than dupilumab, within 8 weeks or within 5 half-lives (if known), whichever is longer, before the baseline visit
4. Having used immunosuppressive/immunomodulating drugs within 4 weeks before the baseline visit
5. Treatment with a live (attenuated) vaccine within 4 weeks before the baseline visit
6. Diagnosed active endoparasitic infections or at high risk of these infections
7. Severe concomitant illness(es) that, in the investigator's judgment, would adversely affect the participant's participation in the study
8. PFP Sub-study Only: Poor compliance as defined by having missed 1 or more of the planned last 3 injections in the main OLE study prior to entering the sub-study
9. PFP Sub-study Only: Switched dupilumab doses within the past 12 weeks
10. PFP Sub-study Only: Meet criteria for temporary/permanent discontinuation of study drug at time of screening into PFP sub-study, as defined in the protocol.
11. Note: Other Protocol Defined Exclusion Criteria Apply

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 6

1. Rate of treatment-emergent adverse events (TEAEs) per participant year from baseline through the last study visit
2. Number of participants with at least one TEAE per participant year from baseline through the last study visit
3. OPTIONAL SUB-STUDY: Pharmacokinetic (PK) of dupilumab: Peak concentration (Cmax)
4. OPTIONAL SUB-STUDY: PK of dupilumab: Trough concentration (Ctrough)
5. OPTIONAL SUB-STUDY: Incidence of TEAEs during the 12-week PFP treatment period and during entire sub-study
6. OPTIONAL SUB-STUDY: Incidence of SAEs during the 12-week PFP treatment period and during entire sub-study

Secondary endpoints 17

1. Number of treatment-emergent serious adverse events (SAEs) from baseline through the last study visit
2. Incidence of TEAEs of special interest from baseline through the last study visit
3. Proportion of participants with an Investigator Global Assessment (IGA) score of 0 or 1 (clear or almost clear) at all in-clinic visits post-baseline
4. Proportion of participants with Eczema Area and Severity Index (EASI)-75 (≥75% reduction in EASI from baseline of parent study) response at all in-clinic visits post-baseline
5. Change from baseline in EASI score at all in-clinic visits post-baseline
6. Percent change from baseline in EASI at all in-clinic visits post-baseline
7. Change from baseline in Body Surface Area (BSA) affected by AD (BSA) at all in-clinic visits post-baseline
8. Percent change from baseline in SCORing Atopic Dermatitis (SCORAD) at all in-clinic visits post-baseline
9. Change from baseline in Children’s Dermatology Life Quality Index (CDLQI) for participants ≥4 years of age at all in-clinic visits post-baseline in which the assessments are planned to be performed
10. Change from baseline in Infants’ Dermatology Quality of Life Index (IDQOL) for participants <4 years of age at all in-clinic visits post-baseline in which the assessments are planned to be performed
11. Proportion of responders (defined as participants with IGA 0 or 1) who maintain IGA 0 or 1 during at least 75% of the subsequent visits during the treatment period
12. For responders (defined as participants with IGA 0 or 1), median percentage of subsequent visits during the treatment period, at which IGA 0 or 1 is maintained
13. Number of AD flares during the study
14. Annualize event rate of AD flares during the study
15. Proportion of participants with at least one flare during the study
16. Proportion of well-controlled weeks
17. OPTIONAL SUB-STUDY: Incidence and titer of treatment-emergent anti-drug antibodies (ADA) (PFP Sub-Study)

Investigational products

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

Test 4

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Regeneron Pharmaceuticals Inc.

Sponsor organisation

Regeneron Pharmaceuticals Inc.

Address

777 Old Saw Mill River Road

City

Tarrytown

Postcode

10591-6717

Country

United States

Scientific contact point

Organisation

Regeneron Pharmaceuticals Inc.

Contact name

Medical Affairs

Public contact point

Organisation

Regeneron Pharmaceuticals Inc.

Contact name

Medical Affairs

Third parties 6

Locations

3 EU/EEA countries · 18 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 39 files

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

Application history

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