A Study of Luspatercept (ACE-536) to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes

Completed · Phase 3 · Has a placebo group

Conditions studied: Myelodysplastic Syndromes

In brief

The study will be conducted in compliance with the International Council on Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use/Good Clinical Practice (GCP) and applicable regulatory requirements. This is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) versus placebo in participants with anemia due to the Revised International Prognostic Scoring System (IPSS-R) very low, low, or intermediate MDS with ring sideroblasts who require red blood cell (RBC) transfusions.

Key facts

Study ID

NCT02631070

Run by

Celgene

People needed

229

Starts

2016-02-09

Expected to finish

2020-11-26

Last updated by the study team

2021-12-17

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Subjects must satisfy the following criteria to be enrolled in the study:
• Subject is ≥ 18 years of age the time of signing the informed consent form (ICF).
• Documented diagnosis of MDS according to World Health Organization (WHO)/French American British (FAB) classification that meets IPSS R classification of very low, low, or intermediate risk disease, and:
• Ring sideroblast ≥ 15% of erythroid precursors in bone marrow or ≥ 5% (but < 15%) if SF3B1 mutation is present.
• < 5% blasts in bone marrow
• Peripheral blood white blood cell (WBC) count < 13,000/µL 3. Requires red blood cell RBC transfusions 4. Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2 5. Subjects who are refractory/intolerant/ineligible to prior erythropoietin-stimulating agents (ESA) treatment, defined as:
• Refractory to prior - erythropoietin stimulating agents treatment: documentation of non-response or response that is no longer maintained to prior ESA-containing regimen, either as single agent or combination (eg, with granulocyte colony stimulating factor (G-CSF); ESA regimen must have been either recombinant human erythropoietin (rHu EPO) ≥ 40,000 IU/wk for at least 8 doses or equivalent OR darbepoetin alpha ≥ 500 μg Q3W for at least 4 doses or equivalent
• Intolerant to prior ESA treatment: documentation of discontinuation of prior ESA-containing regimen, either as single agent or combination (eg, with G-CSF), at any time after introduction due to intolerance or an adverse event
• ESA ineligible: low chance of response to ESA base on endogenous serum erythropoietin level > 200 U/L for subjects not previously treated with ESAs

You may not qualify if…

• The presence of any of the following will exclude a subject from enrollment:
• Prior therapy with disease modifying agents for underlying MDS disease.
• Previously treated with either luspatercept (ACE-536) or sotatercept (ACE-011)
• MDS associated with del 5q cytogenetic abnormality
• Secondary MDS, ie, MDS that is known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases.
• Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding
• iron deficiency to be determined by serum ferritin less than or equal to 15 ug/L and additional testing if clinically indicated (eg, calculated transferrin saturation [iron/total iron binding capacity less than or equal to 20%] or bone marrow aspirate stain for iron).
• Prior allogeneic or autologous stem cell transplant
• Known history of diagnosis of acute myeloid leukemia (AML)
• Use of any of the following within 5 weeks prior to randomization:
• anticancer cytotoxic chemotherapeutic agent or treatment
• corticosteroid, except for subjects on a stable or decreasing dose for ≥ 1 week prior to randomization for medical conditions other than MDS
• iron-chelating agents, except for subjects on a stable or decreasing dose for at least 8 weeks prior to randomization
• other RBC hematopoietic growth factors (eg, Interleukin-3)
• investigational drug or device, or approved therapy for investigational use. If the half-life of the previous investigational product is known, use within 5 times the half-life prior to randomization or within 5 weeks, whichever is longer is excluded.
• Prior history of malignancies, other than MDS, unless the subject has been free of the disease (including completion of any active or adjuvant treatment for prior malignancy) for ≥ 5 years. However, subjects with the following history/concurrent conditions are allowed:
• Basal or squamous cell carcinoma of the skin
• Carcinoma in situ of the cervix
• Carcinoma in situ of the breast
• Incidental histologic finding of prostate cancer (T1a or T1b using the tumor, nodes, metastasis [TNM] clinical staging system)
• Major surgery within 8 weeks prior to randomization. Subjects must have completely recovered from any previous surgery prior to randomization

Where it is running

• Yale University School of Medicine — New Haven, Connecticut, United States
• H Lee Moffitt Cancer Center and Research Institute — Tampa, Florida, United States
• Emory University Hospital — Atlanta, Georgia, United States
• Ochsner Medical Institutions — New Orleans, Louisiana, United States
• Johns Hopkins Sidney Kimmel Comprehensive Cancer Center — Baltimore, Maryland, United States
• Karmanos Cancer Institute — Detroit, Michigan, United States
• Columbia-Presbyterian Medical Center — New York, New York, United States
• Montefiore Medical Center Albert Einstein Cancer Center — The Bronx, New York, United States
• Gabrail Cancer Center — Canton, Ohio, United States
• Cleveland Clinic Taussig Cancer Institute — Cleveland, Ohio, United States
• Vanderbilt University Medical Center — Nashville, Tennessee, United States
• MD Anderson Cancer Center — Houston, Texas, United States
• Algemeen Ziekenhuis Klina — Brasschaat, Belgium
• AZ Sint-Jan AV Brugge — Bruges, Belgium
• UZ Brussels — Brussels, Belgium
• Grand Hopital de Charleroi — Charleroi, Belgium
• UZ Gent — Ghent, Belgium
• UZ Leuven — Leuven, Belgium
• Cliniques Universitaires UCL de Mont-Godine — Yvoir, Belgium
• Tom Baker Cancer Center — Calgary, Alberta, Canada
• Juravinski Cancer Centre — Hamilton, Ontario, Canada
• Sunnybrook Health Sciences Centre — Toronto, Ontario, Canada
• Princess Margaret Hospital — Toronto, Ontario, Canada
• CHU d'Angers — Angers, France
• Stanford Cancer Center — Stanford, California, United States

Full record on ClinicalTrials.gov

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