The study is a Phase 2, open-label, multicenter study to determine the efficacy and safety of Luspatercept (ACE-536) in adults with congenital dyserythropoietic anemia type II (CDA II).
- Conditions
- Congenital Dyserythropoietic Anemia type II (CDAII)MedDRA version: 21.1Level: PTClassification code 10081457Term: Congenital dyserythropoietic anaemiaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2020-005736-30-IT
- Lead Sponsor
- FOndazione per la Ricerca sulle ANemie ed EMoglobinopatie in ItaliA - For Anemia
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 40
- Males or female patients >= 18 years of age at the time of signing the informed consent document (ICF).
- Subject must understand and voluntarily sign an informed consent form (ICF) prior to any study-related assessments/procedures being conducted.
- Biallelic causative mutations in SEC23B gene alone or associated with other gene variants (e.g., globin gene defects).
- Monoallelic causative mutations in SEC23B gene in presence of hypoglycosylation of band 3 and/or associated with other gene variants (e.g., globin gene defects).
- Performance status: Eastern Cooperative Oncology Group (ECOG) score of 0 or 1
- Non-transfusion dependent (NTD) patients, defined as having received < 1/0 unit of RBCs within 8 weeks prior to Cycle 1 Day 1.
- Or transfusion dependent patients, defined as requiring ³ 3 units of RBCs every 8 weeks (confirmed over 6 months prior to Cycle 1 Day 1).
- Anemia, defined as: (i) mean Hb concentration < 10.9 g/dl of 2 measurements (one performed within one day prior to Cycle 1 Day 1 and the other performed during the screening period) in non-transfusion dependent patients, or (ii) transfusion dependent.
- Splenectomized or non-splenectomized patients are both eligible.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 35
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5
1. Any clinically significant pulmonary (including pulmonary hypertension), cardiovascular, endocrine, neurologic, hepatic (including cirrhosis and liver failure), gastrointestinal, infectious, immunological (including clinically significant allo- or auto-immunization) or genitourinary disease considered by the investigator as not adequately controlled prior to Cycle 1 Day 1.
2. Patients with history of cancer, or suffering from evolutive cancer, or in remission with treatment stopped less than 2 years before.
3. Anemia from other causes.
4. Symptomatic splenomegaly.
5. Thromboembolic events = grade 3 according to the National Cancer lnstitute-Common Terminology Criteria for Adverse Events (NCl-CTCAE) v.4 .0 (current active minor version) within 12 months prior to Cycle 1 Day 1.
6. Ejection fraction < 50% by echocardiogram, MUGA or cardiac MRI.
7. Uncontrolled hypertension defined as systolic blood pressure (BP) = 150 mm Hg or diastolic BP =95 mm Hg.
8. QTc > 450 msec on screening ECG.
9. Proteinuria = Grade 2.
10. Any active infection requiring parenteral antibiotic therapy within 28 days prior to Cycle 1 Day 1 or oral antibiotics within 14 days of Cycle 1 Day 1.
11. Treatment with another investigational drug or device, or approved therapy for investigational use 28 days prior to Cycle 1 Day 1, or if the half-life of the previous investigational product is known, within 5 times the half-life prior to Cycle 1 Day 1, whichever is longer.
12. Transfusion event within 7 days prior to Cycle 1 Day 1.
13. Splenectomy within 56 days prior to Cycle 1 Day 1.
14. Major surgery (except splenectomy) within 28 days prior to Cycle 1 Day 1. Patients must have completely recovered from any previous surgery prior to Cycle 1 Day 1.
15. lron chelation therapy initiated within 56 days prior to Cycle 1 Day 1.
16. Cytotoxic agents, systemic corticosteroids, immunosuppressants, or anticoagulant therapy such as warfarin or heparin within 28 days prior to Cycle 1 Day 1 (prophylactic aspirin up to 100 mg/d is permitted).
17. Evidence of active hepatitis C (HCV) infection, or active infectious hepatitis B, or known positive human immunodeficiency virus (HIV)
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method