An Open-label Study to evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in children with gMG

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Amgen Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

19 May 2026 → ongoing

Decision date (initial)

2026-02-10

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Amgen Inc.

External identifiers

EU CT number

2025-520993-20-00

WHO UTN

U1111-1324-6197

ClinicalTrials.gov

NCT06987539

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Others, Safety, Pharmacokinetic

To characterize the pharmacokinetics (PK) of inebilizumab administered in pediatric participants with generalized Myasthenia Gravis (gMG).
To characterize the pharmacodynamics (PD) of inebilizumab administered in pediatric participants with gMG.
To assess the safety and tolerability of inebilizumab administered in pediatric participants with gMG.

Secondary objectives 3

1. To assess disease activity when inebilizumab is administered in pediatric participants with gMG.
2. To assess gMG-related quality of life/disability when inebilizumab is administered in pediatric participants with gMG.
3. To characterize the immunogenicity of inebilizumab administered in pediatric participants with gMG.

Conditions and MedDRA coding

Generalized Myasthenia Gravis

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-001911-PIP02-22

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Age ≥ 2 to < 18 years of age on the day of enrollment.
2. "Diagnosis of gMG defined as: Positive serologic test for anti-AChR or anti-MuSK Ab titers as confirmed at screening (1 retest allowed), and; At least 1 of the following: History of abnormal neuromuscular transmission test results demonstrated by single-fiber electromyography or repetitive nerve stimulation; or; History of positive anticholinesterase test (eg, edrophonium chloride test); or Participant demonstrated improvement in gMG signs on oral cholinesterase inhibitors, as assessed by the treating physician; or Clinical syndrome consistent with a diagnosis of gMG, and not otherwise explained by another condition."
3. Myasthenia Gravis Foundation of America Clinical Classification Class II, III, or IV at the time of screening.
4. Quantitative Myasthenia Gravis score of 11 or greater at screening.
5. "Participants may enter the study on: (1) Corticosteroids only, with no dose increase within 4 weeks prior to screening, or (2) One allowed non-steroidal immunosuppressive therapies (IST) (azathioprine, mycophenolate mofetil, or mycophenolic acid) with continuous use for at least 6 months prior to screening and no dose increase within 4 months prior to screening, or Combination of (1) corticosteroids with no dose increase within 4 weeks prior to screening and (2) one allowed non-steroidal IST with continuous use for at least 6 months prior to screening and no dose increase within 4 months prior to screening."
6. "Participants may enter the study on a stable dose of acetylcholinesterase inhibitors (pyridostigmine dose). The acetylcholinesterase inhibitor dose must have been stable for at least 2 weeks prior to enrollment."

Exclusion criteria 9

1. Thymectomy within 12 months prior to baseline (day 1) visit or planned thymectomy during the duration of the treatment period.
2. "Unresected thymoma. Note: Participants with benign thymoma resected > 12 months prior to screening may enroll. Benign is defined as no known metastases and no extension into or beyond the capsule on pathological examination. Imaging to evaluate for thymoma must have been performed prior to screening per standard of care."
3. Hospitalization for any reason < 30 days prior to screening.
4. Current or recent gMG exacerbation that has not returned to baseline/resolved within at least 30 days prior to screening.
5. History of recurrent significant infections (eg, requiring hospitalization or IV antibiotics).
6. "Receipt of any biologic B-cell-depleting therapy (eg, rituximab, ocrelizumab, obinutuzumab, ofatumumab, inebilizumab) or any experimental B-cell-depleting agent in the 6 months prior to screening."
7. "Receipt of any other mAb or large molecule biologic, including but not limited to FcRn inhibitors, anti-TNF mAbs, anti-JAK Stat mAbs, and complement inhibitors within 6 months prior to screening."
8. Receipt within the 4 weeks prior to screening: Live attenuated vaccine (administration of inactivated [killed] vaccine is acceptable); Blood transfusion
9. "Participants of childbearing potential unwilling to use protocol-specified method of contraception see (Section 11.5) during treatment and for an additional 6 months after the last dose of investigational product."

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 5

1. "Pharmacokinetic parameters, including maximum observed concentration (Cmax), area under the concentration-time curve (AUC), halflife (t1/2), clearance (CL) and volume of distribution at steady state (Vss)"
2. Change from baseline in cluster of differentiation 20 (CD20)+ B-cell counts
3. "Incidence of treatment-emergent adverse events, treatment-emergent serious adverse events, adverse events of interest"
4. Changes in laboratory parameters
5. Changes in vital signs

Secondary endpoints 3

1. Change in Quantitative Myasthenia Gravis (QMG) score
2. Change in Myasthenia Gravis Activities of Daily Living (MG-ADL) score
3. Presence of anti-drug antibodies (ADAs)

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Amgen Inc.

Sponsor organisation

Amgen Inc.

Address

1 Amgen Center Drive

City

Thousand Oaks

Postcode

91320-1730

Country

United States

Scientific contact point

Organisation

Amgen Inc.

Contact name

Medical Information

Public contact point

Organisation

Amgen Inc.

Contact name

Medical Information

Third parties 8

Locations

4 EU/EEA countries · 7 investigational sites

By country

Investigational sites

Country notifications

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

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Corrective measures 2 · Art. 77 CTR

Results and documents

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

Documents 54 files

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

Application history

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