A study to learn if rozanolixizumab is safe, how it moves through the body, and if it works in children with myasthenia gravis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

UCB Biopharma

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

14 Jun 2024 → ongoing

Decision date (initial)

2024-02-01

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

UCB Biopharma SRL

External identifiers

EU CT number

2022-502074-16-00

WHO UTN

U1111-1285-0787

ClinicalTrials.gov

NCT06149559

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Safety, Pharmacokinetic

Assess the safety and tolerability of subcutaneous (sc) administration of rozanolixizumab in pediatric participants aged ≥2 to <18 years with generalized Myasthenia Gravis (gMG)

Secondary objectives 1

1. - Evaluate the activity of rozanolixizumab - Evaluate the safety and tolerability of sc administration of rozanolixizumab in pediatric participants aged ≥2 to <18 years with gMG - Evaluate the Pharmacokinetic (PK) of rozanolixizumab - Evaluate the immunogenicity of rozanolixizumab

Conditions and MedDRA coding

Generalized Myasthenia Gravis

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-002681-PIP01-02

Plan to share IPD

Yes

IPD plan description

Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. - Study participant must be ≥2 to <18 years of age inclusive, at the time of signing the informed consent/assent according to local regulation - Study participant must have a documented diagnosis of generalized Myasthenia Gravis (gMG) at Screening that includes a record confirming the presence of MG specific autoantibodies to acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) prior to Screening - Study participant has Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IVa at Screening - Study participant has received existing conventional treatment(s) for gMG (eg, pyridostigmine, corticosteroids, and/or immune suppressants) prior to Screening - Study participant has had an unsatisfactory clinical response or worsening of gMG symptoms and is in need of additional therapy (for example, plasma exchange (PEX) or treatment with intravenous immunoglobulin g (IVIg))

Exclusion criteria 1

1. - Study participant with severe weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis at Screening or Baseline - Study participant has a known hypersensitivity to any components of the Investigational Medicinal Product (IMP) or other anti-FcRn medications - Study participant with any active or untreated thymoma - Study participant has a history of thymectomy within 6 months prior to Screening - Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the Investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of IMP - Study participant has received a live vaccination within 4 weeks prior to Baseline or intends to have a live vaccination during the course of the study

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. - Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit (up to 18 weeks) - Occurrence of TEAEs leading to permanent withdrawal of Investigational Medicinal Product (IMP) up to the EOS Visit (up to 18 weeks) - Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit (up to 18 weeks)

Secondary endpoints 10

1. Percent change in total Immunoglobulin G (IgG) from Baseline at the end of Week 6
2. Absolute change in total IgG from Baseline at the end of Week 6
3. Percent change from Baseline in MG-specific autoantibodies (anti-AChR or anti-MuSK) levels at the end of Week 6
4. Absolute change from Baseline in MG-specific autoantibodies (anti-AChR or anti-MuSK) levels at the end of Week 6
5. Change from Baseline in myasthenia gravis-activities of daily living (MG-ADL) total score at the end of Week 6
6. Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6
7. Occurrence of other TEAEs (including headache, nausea, and infusion site reactions) during Treatment Period 1 (TP1) and Observation Period 1(OP1)
8. Evaluation of local tolerability at each scheduled assessment during TP1
9. Plasma concentration of rozanolixizumab at the 6-week treatment cycle
10. Incidence of antidrug antibodies (ADAs) at the end of Week 6

Investigational products

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

Test 1

Auxiliary 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

UCB Biopharma

Sponsor organisation

UCB Biopharma

Address

Researchdreef 60

City

Anderlecht

Postcode

1070

Country

Belgium

Scientific contact point

Organisation

UCB Biopharma

Contact name

UCB Cares

Public contact point

Organisation

UCB Biopharma

Contact name

UCB Cares

Third parties 12

Locations

2 EU/EEA countries · 5 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 41 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