A Proof-of-concept Study to Evaluate the Efficacy and Safety of Rozanolixizumab to Treat Adult Study Participants With Severe Fibromyalgia Syndrome

• ClinicalTrials.gov Identifier: NCT05643794
• Recruitment Status: Active, not recruiting
• First Posted: December 9, 2022
• Last Update Posted: December 18, 2023
• Sponsor: UCB Biopharma SRL
• Information provided by (Responsible Party): UCB Pharma ( UCB Biopharma SRL )

Study Description

Brief Summary:

The purpose of the study is to evaluate efficacy and safety of rozanolixizumab to treat adult study participants with severe fibromyalgia syndrome (FMS).

Condition or disease	Intervention/treatment	Phase
Fibromyalgia	Drug: rozanolixizumab; Other: Placebo	Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 63 participants
• Allocation: Randomized
• Intervention Model: Crossover Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Phase 2A, Proof-Of-Concept Study to Evaluate the Efficacy and Safety of Rozanolixizumab to Treat Adult Study Participants With Severe Fibromyalgia Syndrome
• Actual Study Start Date: December 21, 2022
• Estimated Primary Completion Date: May 28, 2024
• Estimated Study Completion Date: July 17, 2024

Arms and Interventions

Arm	Intervention/treatment
Experimental: Treatment sequence 1; Study participants on Treatment sequence 1 will receive rozanolixizumab and Placebo during the dosing period at pre-specified timepoints.	Drug: rozanolixizumab; Study participants will receive rozanolixizumab during the dosing periods as pre-defined.; Other Name: UCB7665; Other: Placebo; Study participants will receive Placebo during the dosing periods as pre-defined.; Other Name: PBO
Experimental: Treatment sequence 2; Study participants on Treatment sequence 2 will receive rozanolixizumab and Placebo during the dosing period at pre-specified timepoints.	Drug: rozanolixizumab; Study participants will receive rozanolixizumab during the dosing periods as pre-defined.; Other Name: UCB7665; Other: Placebo; Study participants will receive Placebo during the dosing periods as pre-defined.; Other Name: PBO
Placebo Comparator: Treatment sequence 3; Study participants on Treatment sequence 3 will receive Placebo during the dosing period at pre-specified timepoints.	Other: Placebo; Study participants will receive Placebo during the dosing periods as pre-defined.; Other Name: PBO

Outcome Measures

Primary Outcome Measures :

1. Brief Pain Inventory short form (BPI-SF) average interference score after 12 weeks of treatment [ Time Frame: After 12 weeks of treatment ]
   The short form of the BPI is a self-administered questionnaire used to evaluate the severity of a study participant's pain and the impact of this pain on the study participant's daily functioning. The BPI-SF assesses for the location of pain, pain intensity and functional interference from pain. The 7 BPI-SF interference items include: general activity, mood, walking ability, normal work (including housework), relations with other people, sleep, and enjoyment of life. Each item is rated on a 0 (does not interfere) to 10 (completely interferes) scale with a recall period of 24 hours. The arithmetic mean of the 7 interference items can be used as a measure of pain interference.

Secondary Outcome Measures :

1. Percentage of participants with treatment-emergent adverse events (TEAEs) during the study [ Time Frame: From Baseline till end of Safety Follow-up (up to 32 Weeks) ]
   An Adverse Event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication.
2. Percentage of participants with TEAEs leading to withdrawal of investigational medicinal product (IMP) [ Time Frame: From Baseline till end of Safety Follow-up (up to 32 Weeks) ]
   An Adverse Event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication.
3. Brief Pain Inventory short form (BPI-SF) average interference score after 24 weeks of treatment [ Time Frame: After 24 weeks of treatment ]
   The short form of the BPI is a self-administered questionnaire used to evaluate the severity of a study participant's pain and the impact of this pain on the study participant's daily functioning. The BPI-SF assesses for the location of pain, pain intensity and functional interference from pain. The 7 BPI-SF interference items include: general activity, mood, walking ability, normal work (including housework), relations with other people, sleep, and enjoyment of life. Each item is rated on a 0 (does not interfere) to 10 (completely interferes) scale with a recall period of 24 hours. The arithmetic mean of the 7 interference items can be used as a measure of pain interference.
4. Revised Fibromyalgia Impact Questionnaire (FIQR) score after 10 weeks of treatment [ Time Frame: After 10 weeks of treatment ]
   The Revised Fibromyalgia Impact Questionnaire (FIQR) is a 21-item questionnaire with a recall period of 7 days. The FIQR includes 3 domains: function, overall impact, and symptoms. Each item is based on an 11-point numeric rating scale. The total score ranges from 0 to 100, with 0 denoting the best possible condition and 100 denoting the worst possible condition.
5. Mean 7-day average daily pain score assessed with Pain Numeric Rating Scale after 10 weeks of treatment [ Time Frame: After 10 weeks of treatment ]
   The Pain Numeric Rating Scale (NRS) is a numeric version of the Visual Analogue Scale (VAS) in which a respondent selects a whole number that best describes "How much pain have you experienced on average over the past 24 hours?" The 11-point Pain NRS ranges from 0 (no pain) to 10 (pain as bad as you can imagine).
6. Mean 7-day fatigue score assessed with Fatigue Numeric Rating Scale after 10 weeks of treatment [ Time Frame: After 10 weeks of treatment ]
   The Fatigue Numeric Rating Scale (NRS) is a numeric version of the Visual Analogue Scale (VAS) in which a respondent selects a whole number that best describes "How much fatigue have you experienced on average over the past 24 hours?" The 11-point Fatigue NRS ranges from 0 (no fatigue) to 10 (fatigue as bad as you can imagine).

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 70 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• Study participant must be ≥18 years and ≤70 years of age at the time of signing the informed consent form (ICF)
• Study participant with a diagnosis of fibromyalgia as defined by the 2016 Revisions to the 2010/2011 fibromyalgia diagnostic criteria (American College of Rheumatology Preliminary

Diagnostic Criteria) plus the following characteristics during the Screening Period:

1. Brief Pain Inventory-short form (BPI-SF) interference score ≥6.
2. Study participant has been diagnosed with fibromyalgia syndrome (FMS) for at least 6 months.
3. Study participant has been having FMS symptomatology for at least 2 years before enrollment - Capable of giving signed informed consent as described in the Protocol which includes compliance with the requirements and restrictions listed in the ICF and in the Study Protocol

Exclusion Criteria:

• Study participant has been diagnosed with fibromyalgia syndrome (FMS) for >15 years
• Study participant has any systemic autoimmune inflammatory disease
• Study participant has any medical or psychiatric or separate chronic pain condition that, in the opinion of the investigator, could jeopardize or would compromise the study participant's ability to participate in this study or the ability to assess FMS-related pain
• Study participant has severe renal impairment, defined as estimated glomerular filtration rate <30 mL/min/1.73 m^2, (calculated using Modification of Diet in Renal Disease [MDRD] study equation), at Screening visit
• Study participant has a clinically important active infection (including unresolved or not adequately treated infection) as assessed by the investigator
• Study participant has chronic inflammatory demyelinating polyneuropathy
• Study participant has a current or medical history of primary immunodeficiency
• Study participant is pregnant or lactating

• Study participant

  • Has suicide attempt in the past 2 years (including an active attempt, interrupted attempt, or aborted attempt),
  • OR had suicidal ideation with at least some intent to act in the past 6 months as indicated by a positive response (Yes) to either Question 4 or Question 5 of the Columbia Suicide Severity Rating Scale (C-SSRS) at Screening or Baseline (Visit 3);
  • OR is otherwise judged clinically to be at a serious suicidal risk based on the investigator's judgment

Contacts and Locations

Locations

United Kingdom

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Blackpool, United Kingdom

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Cannock, United Kingdom

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Leeds, United Kingdom

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Liverpool, United Kingdom

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Manchester, United Kingdom

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Stockton-on-tees, United Kingdom

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Tankersley, United Kingdom

Sponsors and Collaborators

UCB Biopharma SRL

Investigators

• Study Director: UCB Cares; 001 844 599 2273 (UCB)

More Information

• Responsible Party: UCB Biopharma SRL
• ClinicalTrials.gov Identifier: NCT05643794
• Other Study ID Numbers: FM0001
• First Posted: December 9, 2022
• Last Update Posted: December 18, 2023
• Last Verified: December 2023

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• 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.
• Supporting Materials: Study Protocol; Statistical Analysis Plan (SAP); Clinical Study Report (CSR)
• Time Frame: 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.
• Access Criteria: Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, 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.
• URL: http://www.vivli.org/

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: No

Keywords provided by UCB Pharma ( UCB Biopharma SRL ):

Phase 2; Fibromyalgia; rozanolixizumab

Additional relevant MeSH terms:

Fibromyalgia; Myofascial Pain Syndromes; Muscular Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Neuromuscular Diseases; Nervous System Diseases; Rozanolixizumab; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs