IMPACT B-thalasemia (CA056-144)

Type:
Studiedocumentatie
Ziektebeelden:
Leeftijd:
≥ 18 jaar
Fase:
N.v.t.
Lijn:
N.v.t.
Status:
Open
Locatie:
Amsterdam UMC
Volledige titel Improvement of MRI assessed cerebral Perfusion and oxygenation by luspatercept-induced Anemia Correction in non-transfusion dependent Thalassemia
Documenten
Samenvatting met in- en exclusiecriteria

Inclusiecriteria:

  1. NTDT bèta-thalassemia or HbE/bèta-thalassemia
  2. Baseline Hb level of ≤ 6.2 mmol/L before initiation of luspatercept
  3. ≥ 18 years of age
  4. ≤ 5 RBC units transfused in the 24 weeks prior to inclusion
  5. Highly effective contraception from study start to 30 days after the last dose of study drug for fertile women and barrier methods of contraception for men

 

Exclusiecriteria:

  1. Contra-indication for MRI
  2. Female who is breast feeding or pregnant.
  3. Hepatic dysfunction characterized by alanine aminotransferase (ALT) > 4 × ULN.
  4. Severe renal dysfunction (estimated glomerular filtration rate <30mL/min).
  5. History of malignancy within the past 2 years prior to participation requiring chemotherapy and/or radiation (with the exception of local therapy for non-melanoma skin malignancy).
  6. History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent, including but not limited to the following: 1. Unstable angina pectoris or myocardial infarction or elective coronary intervention. 2. Congestive heart failure requiring hospitalization. 3. Uncontrolled clinically significant arrhythmias.

 

Primary objective:

To assess whether luspatercept can improve the cerebral oxygen metabolism (CMRO2) in patients with NTDT

 

Secondary objectives:

To assess the effect of luspatercept treatment on CBF in NTDT patients

To assess the effect of luspatercept treatment on neurocognitive impairment

To assess the effect of luspatercept treatment on cardiac parameters of cardiac stress (NTproBNP and TRV)

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Principal investigator Amsterdam UMC Bart Biemond
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