A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes

Официальное название

An Open-label, Dose-escalation, Dose-finding, and Proof-of-concept Trial of SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes

Обзор

The goal of this clinical trial is to learn about SP-420 ability to remove iron from organs in subjects with transfusion-dependent β-thalassemia or transfusion-dependent low-risk myelodysplastic syndrome. The main questions it aims to answer are: * How efficient is SP-420 in cleaning iron from the liver? * How is the safety and tolerability of ascending doses of SP-420? Participants will: * Take medication three times weekly * Attend up to 20 site visits * Undergo MRI scans

Вмешательства

• Препарат SP-420
  Capsules for oral intake

Первичные конечные точки

• To establish dose-response relationship of SP-420 for 24 weeks in the treatment of subjects with transfusion-dependent β-thalassemia [Срок оценки: 24 weeks]
• To assess the safety and tolerability of ascending doses of SP-420 after 12 weeks treatment of subjects with transfusion-dependent low-risk myelodysplastic syndrome [Срок оценки: 12 weeks]

Вторичные конечные точки (4)

• To assess the efficacy of SP-420 in clearing iron from the liver after 24 weeks treatment of subjects with transfusion-dependent β-thalassemia [Срок оценки: 24 weeks]
• To assess the efficacy of SP-420 in clearing iron from the liver after 12 and 48 weeks treatment of subjects with transfusion-dependent β-thalassemia [Срок оценки: 12 and 48 weeks]
• To assess the efficacy of SP-420 on serum (s-) ferritin [Срок оценки: up to 48 weeks]
• To assess the safety and tolerability of ascending doses of SP-420 [Срок оценки: 48 weeks]

Критерии участия

Thalassemia cohorts:

Критерии включения

• Women and men aged 18 years or older
• Transfusion-dependent β-thalassemia including HbE/β-thalassemia requiring iron chelation therapy (β-thalassemia with mutation and/or multiplication of α-globin is allowed)
• On a stable dose of iron chelation for at least 4 weeks prior to screening
• Weight ≥ 35kg at screening
• Transfusion iron overload
• Treated and followed for at least the past 6 months in a specialized centre

Критерии исключения

• β-thalassemia with the structural Hb variants HbS and HbC
• Current MDS
• Current biliary disorder
• Historic or ongoing clinically significant kidney disease
• Unable to undergo trial assessments including MRI e.g. due to claustrophobia in MRI scanner
• Pregnant or nursing women
• Men who do not agree to practice effective barrier contraception during the entire period

Myelodysplastic Syndromes Cohorts:

Критерии включения

• Women and men aged 18 years or older
• Very low, low, or intermediate risk Myelodysplastic Syndrome according to IPSS-R
• Weight ≥ 35kg at screening
• Transfusion iron overload
• Treated and followed for at least the past 6 months at medical facilities experienced with MDS

Критерии исключения

• Therapy-related MDS or MDS with a known bone marrow fibrosis
• Diagnosis of decompensated liver cirrhosis
• Clinically significant kidney disease, either historic or ongoing
• Uncontrolled ischemic heart disease or uncontrolled arrythmia
• Uncontrolled hypertension
• Uncontrolled dyslipidaemia
• Uncontrolled Diabetes
• Major surgery within 8 weeks prior to screening
• Pregnant or nursing women
• Men who do not agree to practice effective barrier contraception during the entire period

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Последовательный дизайн

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Дания · 1 центр

• Pharmacosmos Investigational Site — Copenhagen

Идентификаторы

NCT: NCT05693909 · P-SP420-THAL-01

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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