Phase 2 Open-Label Extension Study to Evaluate the Safety and Efficacy of Emrusolmin in Adults with Multiple System Atrophy

Trial ID: 2025-521642-14-00
Protocol: TV56286-NDG-20041
Sponsor: Teva Branded Pharmaceutical Products R&D LLC

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Diseases & Conditions

Multiple system atrophy

Objectives

The primary objective is to evaluate the long-term safety and tolerability of oral emrusolmin in adult patients diagnosed with multiple system atrophy. This assessment is critical for determining the clinical profile of the investigational product during extended administration. The study also evaluates the efficacy, pharmacokinetics, and pharmacodynamics of the intervention.

Participants

This study involves 51 participants diagnosed with multiple system atrophy. The study population consists of adults, including both males and females. Participants are selected based on the completion of a previous treatment period and the week 48 visit of a double-blind trial while maintaining compliance with study requirements. A required dysphagia evaluation must confirm the ability to swallow the investigational medicinal product. Females of childbearing potential are eligible provided they have a negative pregnancy test at the baseline visit. Additionally, both fertile males and their female partners are required to utilize highly effective contraception for the duration of the trial and for 28 days following the final dose.

Plans and Procedures

This open-label extension, multi-centered, Phase 2 clinical trial is designed to evaluate the long-term safety and tolerability of emrusolmin in adults diagnosed with multiple system atrophy. The administration of the investigational medicinal product occurs via oral capsules at a dose of 300 mg. The study procedures include a baseline visit following the completion of the double-blind treatment period and the week 48 visit. Eligibility for the extension requires a successful dysphagia evaluation to ensure the ability to swallow the medication whole. Participants must comply with pregnancy testing requirements and utilize highly effective contraception if applicable. The primary endpoints focus on the incidence of adverse events and the frequency of withdrawal due to such events. The trial is estimated to conclude by September 2029.

Treatment

The experimental medication consists of emrusolmin, an orphan drug. This substance is administered in the form of a capsule at a dosage of 300 mg via the oral route.

Efficacy

The assessment of efficacy in this phase 2 clinical trial involves the evaluation of multiple system atrophy. The primary objectives focus on characterizing the long-term safety and tolerability of emrusolmin administered via oral use.

Inclusion and Exclusion Criteria

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Inclusion Criteria

Completion of the treatment period and the week 48 (V9) visit of the DB trial whilst remaining compliant with trial requirements.
Is able to swallow the IMP, as determined by a dysphagia evaluation during the OLE baseline visit; and is willing to swallow the IMP capsules whole, and as scheduled, throughout the duration of the OLE trial
In the investigator’s opinion, the participant and where applicable, participant’s caregiver(s), has (have) the ability to understand the nature of the OLE trial and its procedures, and is (are) able and willing to comply with the requirements of the OLE trial
Participant must provide consent, either by signature, spoken word, or gesture. If participant is physically unable to provide written informed consent, the completed ICF must be signed by one of the following in accordance with local regulation: (1) legally acceptable representative (LAR) or (2) acknowledgement of participant’s consent by an impartial witness. The process for consent should follow the local regulatory requirements. Caregiver consent will also be obtained in the circumstances where caregiver participation is applicable.
Females of childbearing potential (CBP) may be included only if they have a negative pregnancy test at the OLE baseline visit
Females of CBP whose male partners are potentially fertile (ie, no vasectomy) must use highly effective birth control methods for the duration of the OLE trial and for 28 days after the last dose of IMP
Males who are potentially fertile/reproductively competent (not surgically [eg, vasectomy] or congenitally sterile) and their female partners who are of CBP must use, together with their female partners, highly effective birth control methods for the duration of the OLE trial and for 28 days after the last dose of IMP

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Exclusion Criteria

Has any clinically significant uncontrolled medical or psychiatric condition (treated or untreated), or any findings from vital signs, imaging, physical examination, electrocardiogram (ECG), or clinical laboratory, other than MSA related that could jeopardize or compromise the participant’s ability to participate in the OLE trial. The investigator should consult with the medical monitor or trial physician as needed.
Is a female participant who is pregnant, plans to become pregnant, or is breastfeeding during the OLE trial.
Has moderate to severe renal impairment as assessed by estimated glomerular filtration rate <60 mL/min/1.73m2. Minor deviations or borderline results (up to 10 mL/minute/1.73m2) may be acceptable for eligibility at the discretion of the Principal Investigator.
Current or history of chronic liver or biliary disease, >2.5x the upper limit of normal (ULN) for alanine aminotransferase (ALT) or aspartate aminotransferase (AST), or 1.5x ULN total bilirubin (isolated bilirubin >1.5x ULN is acceptable if bilirubin is fractionated and direct bilirubin <35%).
Has any clinically significant disorder that may interfere with absorption, distribution, metabolism, or excretion of IMP (including relevant gastrointestinal surgery, malabsorption syndrome) that makes the participant unsuitable for the trial.
Is of a vulnerable population (eg, people kept in detention or jail).
Is regularly using or consuming any prohibited concomitant medications within the specified exclusionary windows of this trial