Study to test the safety, tolerability and efficacy of UCB7665 in subjects with moderate to severe myasthenia gravis.

• Register: EUCTR
• Last refreshed on: 20 August 2018
• Main ID: EUCTR2016-002698-36-BE
• Date of registration: 29/11/2016
• Prospective Registration: Yes
• Primary sponsor: UCB Biopharma SPRL
• Public title: Study to test the safety, tolerability and efficacy of UCB7665 in subjects with moderate to severe myasthenia gravis.
• Scientific title: A Multicenter, Randomized, Investigator- and Subject-Blind, Placebo-Controlled, Treatment Sequence Study Evaluating the Safety, Tolerability, and Efficacy of UCB7665 in Subjects with Moderate to Severe Myasthenia Gravis.
• Date of first enrolment: 30/01/2017
• Target sample size: 42
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-002698-36
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: yes; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Belgium, Canada, Czech Republic, Denmark, Germany, Spain, United States

Contacts

• Name: Clin Trial Reg & Results Disclosure
• Affiliation: UCB Biosciences GmbH

• Name: Clin Trial Reg & Results Disclosure
• Affiliation: UCB Biosciences GmbH

Key inclusion & exclusion criteria

Inclusion criteria:
- Subject has a well-documented diagnosis of myasthenia gravis (MG) at Visit 1 (Screening), based on subject history and supported by previous evaluations
- Subject would currently be considered for treatment with immunological therapy (immunoglobulin/plasma exchange
(IVIG/PLEX)) by the investigator
-Subject has well-documented record of autoantibodies against antiacetylcholine receptor (Anti-AChR) or anti-muscle specific kinase (Anti- MuSK) prior to Screening
- Female subjects must either be: postmenopausal, permanently sterilized or if childbearing potential applicable will use a highly effective method of birth control
- Male subjects must be willing to use a method of contraception

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 37
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion criteria:
- Subject has previously received treatment in this study or subject has previously been exposed to UCB7665
- Subject has participated in another study of an investigational medicinal product (IMP;or a medical device) within the previous 30 days of Screening or is currently participating in another study of an
investigational medicinal product (IMP; or a medical device)
- Subject has a known hypersensitivity to any components of the IMP
- Subject has a history of hyperprolinemia, since L-proline is a constituent of the UCB7665 IMP
- Subjects with Myasthenia Gravis (MG) only affecting the ocular muscles
- Subjects with severe weakness affecting oropharyngeal or respiratory muscles, or who have myasthenic crisis at Screening or impending crisis
- Subject has quantitative myasthenia gravis (QMG) score of <11 at Baseline
- Subject has a serum total immunoglobulin G (IgG) level <= 6g/L at Screening
- Absolute neutrophil count <1500 cells/mm3
- Subject has any medical condition (acute or chronic illness) or psychiatric condition that, in the opinion of the investigator, could jeopardize or would compromise the subject’s ability to participate in this study
- Subject has any laboratory abnormality that, in the opinion of the investigator, is clinically significant, has not resolved at randomization, and could jeopardize or would compromise the subject’s ability to participate in this study
- Subject has received a live vaccination within 8 weeks prior to the Baseline Visit; or intends to have a live vaccination during the course of the study or within 7 weeks following the final dose of IMP
- Subject has received any experimental biological agent within or outside of a clinical study in the past 3 months or within 5 half-lives prior to Baseline (whichever is longer)



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Moderate to severe myasthenia gravis
MedDRA version: 20.0 Level: PT Classification code 10028417 Term: Myasthenia gravis System Organ Class: 10029205 - Nervous system disorders

Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Intervention(s)

Product Name: UCB7665
Product Code: UCB7665
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: rozanolixizumab
CAS Number: 1584645-37-3
Current Sponsor code: UCB7665
Other descriptive name: UCB7665
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Solution for infusion
Route of administration of the placebo: Subcutaneous use

Primary Outcome(s)

Main Objective: Evaluate the clinical efficacy of UCB7665 as a chronic-intermittent treatment in subjects with generalized myasthenia gravis (MG) who are classified as moderate to severe

Primary end point(s): Change from Baseline in Quantitative Myasthenia Gravis (QMG)
score to Visit 9

Secondary Objective: - Gather data for future study planning, whether for chronic-intermittent treatment or a longer therapy option by evaluating the general concept that UCB7665 has a clinical effect in patients with generalized myasthenia gravis (MG)
- Evaluate the safety and tolerability of UCB7665 administered by subcutaneous (sc) infusion in subjects with MG
- Assess the effect of UCB7665 as measured by total immunoglobulin G (IgG) concentrations in serum

Timepoint(s) of evaluation of this end point: Baseline and Visit 9 (Day 29)

Secondary Outcome(s)

Secondary end point(s): Change from Baseline in Myasthenia Gravis-Composite score to
Visit 9
Change from Baseline in Myasthenia Gravis-Activities of Daily
Living (MGADL) score to Visit 9

Timepoint(s) of evaluation of this end point: Baseline and Visit 9 (Day 29)

Secondary ID(s)

MG0002

2016-002698-36-DE

Source(s) of Monetary Support

UCB Biopharma SPRL