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RecruitingNCT07463521MyVisionUpdated Sep 25, 2026

A Study to Evaluate the Efficacy and Safety of Rozanolixizumab in Adult Participants With Ocular Myasthenia Gravis

A Phase 3 interventional study of Rozanolixizumab and Placebo in Ocular Myasthenia Gravis, sponsored by UCB Biopharma SRL. Recruiting at 14 sites in 5 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-09-25.

Sponsored by UCB Biopharma SRL · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
120
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study summary

The purpose of the study is to demonstrate the efficacy, safety and tolerability of rozanolixizumab compared with placebo in the treatment of adult study participants with Ocular Myasthenia Gravis.

02

Conditions studied

  • Ocular Myasthenia Gravis

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Keywords

  • Ocular Myasthenia Gravis
  • Phase 3
  • rozanolixizumab
03

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Participant must be a minimum of 18 years of age inclusive at the time of signing the informed consent form (ICF)
  • Participant has Myasthenia Gravis Foundation of America (MGFA) Class I with any ocular weakness at Screening through Baseline. The participant may have weakness in muscles of eye (ie, extraocular muscles that move the eyeball, including the medial rectus, lateral rectus, superior rectus, inferior rectus, superior oblique, and inferior oblique, orbicularis oculi muscles, and levator palpebrae superioris) but must have normal strength in all other facial, bulbar, and limb muscles.
  • Study participant has been diagnosed with Ocular Myasthenia Gravis (oMG) with consistent ocular clinical features at Screening and supported by:
  • Documented presence of autoantibodies against acetylcholine receptor (AChR) or muscle-specific kinase (MuSK), OR
  • Documented absence of autoantibodies against AChR or MuSK; in this case, documented abnormal repetitive nerve stimulation (RNS) or single fiber electromyography (SFEMG) (as defined in the adjudication manual) and at least 1 of the following should be met:

    • Documented positive ice test (Ptosis recovers with the ice test [applied 2 minutes (min) to the ptotic lid] with >2mm improvement)
    • History of positive edrophonium chloride (Tensilon) test (or equivalent tests used to establish oMG diagnostic as per current practice)
    • Demonstrated objective improvement in oMG signs with acetylcholinesterase inhibitor (AChEIs), plasma exchange (PLEX), intravenous immunoglobulin (IVIg), subcutaneous immunoglobulin (SCIg) or corticosteroids (CSs)
  • Participant has an Myasthenia Gravis Impairment Index (MGII) ocular score (Patient-Reported Outcome (PRO) part) ≥6 with at least 2 ocular items with a score of ≥2 at both Screening and Baseline visits.
  • Participant reported ocular symptom(s) onset \<3 years before Screening or ≥3 years provided they have demonstrated response (ie, improvement in ptosis or diploplia) to treatment (IVIg, PLEX, SCIg, pyridostigmine, and/or CSs) in the past year.
  • Participant has no pupillary abnormality except those resulting from prior localized eye disease or surgical intervention.
  • Participant who:

    • is currently receiving background treatment for oMG symptoms at the time of Screening and has been receiving treatment for oMG with a stable dose for at least 30 days prior to Screening, OR
    • is not receiving any background treatment at the time of Screening
  • Participant has a body weight ≥35kg at Baseline.

Exclusion criteria

Exclusion Criteria:

  • Participant has any clinically significant medical or psychiatric condition (including an alcohol or drug use disorder), recent major surgery (including thymectomy [within 3 months of Screening], solid organ, stem cell or marrow transplant), planned major surgery (including thymectomy) during study participation, and/or significant laboratory abnormality that, in the opinion of the investigator, could jeopardize or would compromise the study participant's ability to participate in this study.
  • Participant has been diagnosed with other diseases that lead to eyelid dropping, peripheral muscle weakness, or diplopia, including other autoimmune diseases that would interfere with an accurate assessment of the oMG clinical symptoms or other neurological diseases, such as congenital myasthenic syndromes, mitochondrial diseases, and muscular dystrophies.
  • Participant has a known hypersensitivity to other anti-Fc receptor (FcRn) medications, to any components of the study medication (including the excipient polysorbate 80) or has a known history of hyperprolinemia, since L-proline is a constituent of the rozanolixizumab formulation.
  • Participant has active neoplastic disease or has received treatment for neoplastic disease within 5 years of study entry (except for basal or squamous cell carcinoma of the skin, carcinoma in situ of the uterine cervix, carcinoma in situ of the breast, or incidental histological findings of prostate cancer [Tumor, Node, Metastasis (TNM) stage T1a or T1b] that has been definitely treated with standard of care approaches).
  • Participant has a clinically relevant active infection (eg, tuberculosis (TB) infection) or a history of serious infection (resulting in hospitalization or requiring intravenous (iv) antibiotic treatment) within 6 weeks before the Baseline Visit.
  • Participant has renal impairment, defined as glomerular filtration rate less than 30milliliter/min/1.73m2 at Screening.
  • Participant has been previously treated with FcRn inhibitors.
  • Participant has been treated with any of the immunosuppressive medications, biologics, or other therapies, in the specified prohibitive timeframe.
04

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Triple (Participant, Care provider, Investigator)
Enrollment
120 participants (estimated)

Study arms

  • Experimental
    Rozanolixizumab

    After the Screening Period, participants will enter the study Intervention Period and will be administered study drug for up to 6 weeks. Prior to entering the Intervention Period, study participants will be randomized at a 1:1 ratio and will receive rozanolixizumab. At the End of Treatment Visit, participants will enter an Observation Period of 4 to 7 weeks with an opportunity to transition to an open-label extension study.

    Drug: Rozanolixizumab

  • Placebo comparator
    Placebo

    After the Screening Period, participants will enter the study Intervention Period and will be administered study drug for up to 6 weeks. Prior to entering the Intervention Period, study participants will be randomized at a 1:1 ratio and will receive placebo. At the End of Treatment Visit, participants will enter an Observation Period of 4 to 7 weeks with an opportunity to transition to an open-label extension study.

    Drug: Placebo

Interventions

  • DrugRozanolixizumab

    Rozanolixizumab will be administered by subcutaneous infusion.

    Also known as: UCB7665

  • DrugPlacebo

    Placebo will be administered by subcutaneous infusion.

05

What researchers measure

Primary outcomes

  1. Change from Baseline at Day 43 in (Myasthenia Gravis Impairment Index) MGII ocular score (Patient-Reported Outcome (PRO) part)

    MGII is a measure of disease severity based on the signs and symptoms of MG patients. The MGII has 22 patient-reported items and 6 examination items and scores are presented as a sum of all items for a total score but also as an ocular and generalized sub-score. The scoring range is 0 to 84, 0-23 for the ocular score, and 0-61 for the generalized score, where higher scores are indicative of more severe symptoms. The recall period is "the past week" ie, the last 7 days.

    Time frame: At Day 43

Secondary outcomes

  1. Change from Baseline at Day 43 in Myasthenia Gravis Symptoms Patient-Reported Outcome (MGSPRO) ocular muscle weakness scale score

    The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (items 1-5); bulbar muscle weakness (items 6-15); respiratory muscle weakness (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42). Study participants will be asked to choose response option that best describes how frequently they experienced muscle weakness fatigability (items 34-42) over the past 7 days using a 5-point Likert scale (1="none of the time" to 5="all of the time") for each item.

    Time frame: At Day 43

  2. Change from Baseline at Day 43 in Myasthenia Gravis Quality of Life 15-item Scale (revised version) (MG-QoL15r) total score

    The MG-QoL15r is a brief survey, completed by the study participant, that is designed to assess some aspects of "quality of life" related to MG. The recall period that will be used is "past 7 days". When completing the 15-item MG-QoL15r, MG study participant should consider only how their MG affects these items.

    Time frame: At Day 43

  3. Change from Baseline at Day 43 in Myasthenia Gravis Activities of Daily Living (MG-ADL) ocular subdomain score

    The total MG-ADL score is obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3). The score ranges from 0 to 24, with a higher score indicating more disability. A positive change indicates worsening and a negative change indicates improvement.

    Time frame: At Day 43

  4. Change from Baseline at Day 43 in Myasthenia Gravis Impairment Index (MGII) ocular score (Physical Examination part)

    MGII is a measure of disease severity based on the signs and symptoms of MG patients. The MGII has 22 patient-reported items and 6 examination items and scores are presented as a sum of all items for a total score but also as an ocular and generalized sub-score. The scoring range is 0 to 84, 0-23 for the ocular score, and 0-61 for the generalized score, where higher scores are indicative of more severe symptoms. The recall period is "the past week" ie, the last 7 days.

    Time frame: At Day 43

  5. Incidence of treatment-emergent adverse events (TEAEs)

    Treatment Emergent Adverse Events (TEAEs) are any untoward medical incidence in a participant after the administration of study treatment, whether or not these events are related to study treatment.

    Time frame: Up to Week 13

  6. Incidence of treatment-emergent serious adverse events (TESAEs)

    An SAE is defined as any untoward medical occurrence that, at any dose, meets 1 or more of the criteria listed: * Results in death * Is life-threatening * Requires inpatient hospitalization or prolongation of existing hospitalization * Results in persistent or significant disability/incapacity * Is a congenital anomaly/birth defect * Other important medical events which based on medical or scientific judgement may jeopardize the patients or may require medical or surgical intervention to prevent any of the above.

    Time frame: Up to Week 13

  7. Incidence of TEAEs leading to permanent withdrawal of study treatment

    Treatment Emergent Adverse Events (TEAEs) are any untoward medical incidence in a participant after the administration of study treatment, whether or not these events are related to study treatment. This measure considers any TEAE leading to permanent withdrawal from study.

    Time frame: Up to Week 13

06

Study locations

14 of 14 sites recruiting
  • Mg0038 10104
    Scottsdale, Arizona 85251, United States
    Recruiting
  • Mg0038 10105
    O'Fallon, Illinois 62269, United States
    Recruiting
  • Mg0038 10106
    Amherst, New York 14226, United States
    Recruiting
  • Mg0038 10103
    Columbus, Ohio 43210, United States
    Recruiting
  • Mg0038 10108
    Greenfield, Wisconsin 53228, United States
    Recruiting
  • Mg0038 10201
    Montreal, Canada
    Recruiting
  • Mg0038 31901
    Beijing, China
    Recruiting
  • Mg0038 31906
    Chengdu, China
    Recruiting
  • Mg0038 31907
    Nanchang, China
    Recruiting
  • Mg0038 31902
    Suzhou, China
    Recruiting
  • Mg0038 31802
    Kobe, Japan
    Recruiting
  • Mg0038 20605
    Alicante, Spain
    Recruiting
  • Mg0038 20604
    Barcelona, Spain
    Recruiting
  • Mg0038 20611
    Madrid, Spain
    Recruiting
07

References and documents

Individual participant data

Plan to share: Yes — 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 pre-specified 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.

Supporting information: Study protocol, Sap, Csr

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT07463521
Lead sponsor
UCB Biopharma SRL
Responsible party
Sponsor
First posted
Mar 11, 2026
Start date
May 27, 2026
Primary completion
Dec 1, 2028 (estimated)
Completion
Jan 22, 2029 (estimated)
Last update
Sep 25, 2026

Study contacts

UCB Cares
Contact
ucbcares@ucb.com
+18445992273
UCB Cares
Contact
ucbcares@ucb.com
00184459922733 (UCB)

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
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