A Study to Evaluate the Safety, Tolerability, Efficacy, and Drug Levels of CC-97540 in Participants With Relapsing Forms of Multiple Sclerosis, Progressive Forms of Multiple Sclerosis or Refractory Myasthenia Gravis (MG) (Breakfree-2)
Juno Therapeutics, Inc., a Bristol-Myers Squibb Company
Phase
Phase 1
Started
2024-03-28
Last updated
2026-08-03
Condition(s) studied
Multiple SclerosisMyasthenia Gravis
Investigational drug(s) / intervention(s)
CC-97540FludarabineCyclophosphamide
CC-97540: Specified dose on specified days
Fludarabine: Specified dose on specified days
Cyclophosphamide: Specified dose on specified days
Study summary
The purpose of this study is to evaluate the safety, tolerability, efficacy, and drug levels of CC-97540 in participants with Relapsing Forms of Multiple Sclerosis (RMS), Progressive Forms of Multiple Sclerosis (PMS) or Refractory Myasthenia Gravis (MG).
Eligibility
Sex
ALL
Min age
18 Years
Max age
60 Years
Healthy volunteers
No
Inclusion Criteria
\- Relapsing forms of Multiple Sclerosis (RMS) - Cohort 1.
i) Participants must have an Expanded Disability Status Scale (EDSS) of ≥ 3.0 and ≤ 5.5.
ii) Participants must have a diagnosis of Multiple Sclerosis (MS) with relapsed/refractory MS or conversion to active secondary progressive multiple sclerosis (aSPMS), and worsening of disease within 12 months prior to Screening and while on treatment with a high-efficacy DMT for at least 6 months.
\- Progressive forms of MS - Cohort 2.
i) Participants must have an EDSS ≥ 3.0 and ≤ 6.0.
ii) Participants must have a diagnosis of primary progressive multiple sclerosis (PPMS) that is treatment-resistant or diagnosis of inactive secondary progressive multiple sclerosis (iSPMS).
\- Myasthenia Gravis - Cohort 3
i)MGFA classification of II-IV at screening
ii) Documentation of autoantibodies against AChR or MuSK (historical or at Screening)
iii) Refractory disease defined as disease activity on at least 2 immunosuppressants, including steroids, NSIs, or biologics.
iv) Has had thymectomy, only if indicated according to current guidelines.
Exclusion Criteria
* Cohorts 1 and 2: Participants that cannot complete the 9-Hole Peg Test (9-HPT) in at least 1 hand in \<240 seconds unless extenuating medical conditions unrelated to MS prohibit this.
* Participants that cannot perform a Timed 25-Foot Walk Test (T25FWT) in \< 150 seconds.
* Presence of other confounding peripheral nervous system disorders or other disorders that may impact muscle strength (eg, myositis) or cause weakness, stroke, chronic inflammatory demyelinating polyradiculoneuropathy, Lambert-Eaton myasthenic syndrome.
* Other protocol-defined Inclusion/Exclusion criteria apply.
Primary outcome measure(s)
Number of participants with adverse events (AEs) — Up to week 104
Number of participants with serious adverse events (SAEs) — Up to week 104
Number of participants with adverse events of special interest (AESIs) — Up to week 104
Number of participants with laboratory test result abnormalities — Up to week 104
Number of participants with imaging abnormalities — Up to week 104 For Cohorts 1 and 2
Number of participants with dose-limiting toxicities (DLTs) — Up to week 104
Recommended Phase 2 dose (RP2D) based on the incidence of DLTs that occur during the DLT evaluation period — Up to week 104
Trial sites (34)
Facility
City
Region
Status
Local Institution - 0011
Birmingham
Alabama
University of California, Irvine
Irvine
California
University of Colorado Anschutz Medical Campus
Aurora
Colorado
Colorado Blood Cancer Institute
Denver
Colorado
Yale-New Haven Hospital
North Haven
Connecticut
University of Kansas Medical Center
Kansas City
Kansas
Local Institution - 0005
Boston
Massachusetts
Washington University School of Medicine
St Louis
Missouri
Hackensack University Medical Center
Hackensack
New Jersey
Icahn School of Medicine at Mount Sinai
New York
New York
Neurological Institute of New York
New York
New York
University of Cincinnati Medical Center
Cincinnati
Ohio
Cleveland Clinic Mellen Center
Cleveland
Ohio
Oregon Health and Science University
Portland
Oregon
Swedish Medical Center
Seattle
Washington
Medical College of Wisconsin
Milwaukee
Wisconsin
Antwerp University Hospital
Edegem
Antwerpen
UZ Gent
Ghent
Oost-Vlaanderen
Hopital Claude Huriez - CHU de Lille
Lille
Nord
Pitie Salpetriere University Hospital
Paris
Ville de Paris
Universitaetsklinikum Essen
Essen
North Rhine-Westphalia
Universitaetsklinikum Magdeburg
Magdeburg
Saxony-Anhalt
Local Institution - 0033
Düsseldorf
Germany
Universitaetsklinikum Erlangen
Erlangen
Germany
Klinikum der Universität München Großhadern
München
Germany
Hospital Universitari Vall d'Hebron
Barcelona
Barcelona [Barcelona]
Local Institution - 0016
Barcelona
Barcelona [Barcelona]
Hospital Clínic de Barcelona
Barcelona
Catalunya [Cataluña]
Hospital Universitario Ramón y Cajal
Madrid
Madrid, Comunidad de
Hospital Universitari i Politecnic La Fe
Valencia
Spain
Manchester Royal Infirmary
Manchester
Lancashire
University College London Hospital
London
London, City of
Salford Royal Hospital
Salford
Manchester
Barts Health NHS Trust
London
United Kingdom
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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