Evaluating Long-term Safety of Efgartigimod Administered Intravenously and Subcutaneously in Children With Generalized Myasthenia Gravis (ADAPT Jr+)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Argenx

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

18 Aug 2022 → ongoing

Decision date (initial)

2024-02-27

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

argenx BV

External identifiers

EU CT number

2023-507379-23-00

EudraCT number

2021-002460-46

ClinicalTrials.gov

NCT05374590

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy, Safety, Others

To evaluate the safety and tolerability of efgartigimod IV and efgartigimod PH20 SC

Secondary objectives 1

1. To evaluate the immunogenicity of efgartigimod IV and efgartigimod PH20 SC

Conditions and MedDRA coding

Generalized Myasthenia Gravis

Study design 2 periods

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. The participant completed ARGX-113-2006 or ARGX-113-2207, defined as: a. The participant reached the end of the study in ARGX-113-2006 or ARGX-113-2207 and agreed to participate in the ARGX-113-2008 study. b. The participant qualifies for retreatment in ARGX-113-2006, but cannot complete a Treatment Period (TP) and the required IP visits within the ARGX-113-2006 study's timeframe."
2. The participant and/or the participant's legally authorized representative (LAR) can understand the requirements of the study and provide written informed consent/assent, and demonstrates a willingness and ability to comply with the study protocol procedures.
3. Contraceptive use for sexually active participants of childbearing potential should be consistent with local regulations for those participating in clinical studies. A participant is of childbearing potential if, in the opinion of the investigator, he/she is biologically capable of having children (ie, female participants have started their menses, and male participants have reached the middle of puberty). a. Male participants: contraceptive requirements for male participants are presented in Section 10.4.2.2. b. Female adolescents of childbearing potential (FAOCBP; defined in Section 10.4.1.1) must have a negative urine pregnancy test at study entry (TP1V1 or IP0V1). The contraceptive requirements for FAOCBP are described in Section 10.4.2.1."

Exclusion criteria 4

1. FAOCBP: Pregnancy or lactation, or the participant intends to become pregnant during their participation in the study
2. Discontinued early from ARGX-113-2006 or ARGX-113-2207 treatment
3. A known hypersensitivity reaction to efgartigimod or any of its excipients
4. Any of the following medical conditions: a. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at study entry not sufficiently resolved in the investigtor's opinion. b. Known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of gMG or put the participant at undue risk"

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 4

1. Incidence and severity of adverse events (AEs)
2. Incidence and severity of serious adverse events (SAEs)
3. Incidence and severity of adverse events of special interest (AESIs)
4. Changes in laboratory test results, vital signs, height and weight, and electrocardiogram (ECG) results

Secondary endpoints 1

1. Incidence and prevalence of antidrug antibodies (ADAs) against efgartigimod and antibodies against recombinant human hyaluronidase PH20 (rHuPH20).

Investigational products

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

Test 3

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Argenx

Sponsor organisation

Argenx

Address

Industriepark-Zwijnaarde 7

City

Gent

Postcode

9052

Country

Belgium

Scientific contact point

Organisation

Argenx

Contact name

Chief Scientific Officer

Public contact point

Organisation

Argenx

Contact name

Vice President Clinical Development

Third parties 11

Locations

9 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 117 files

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

Application history

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