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

Pharmacosmos

Status and phase: Enrolling; Phase 2

Conditions: Beta Thalassemia Major Anemia; Myelodysplastic Syndrome

Treatments: Drug: SP-420

Study type: Interventional

Funder types: Industry

Identifiers

NCT05693909

P-SP420-THAL-01

Details and patient eligibility

About

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

Enrollment: 90 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Thalassemia cohorts:

Inclusion criteria:

• 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

Exclusion criteria:

• 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:

Inclusion criteria:

• 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

Exclusion criteria:

• 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

---

90 participants in 6 patient groups

Cohort 1a-1b

Experimental group

Description:

SP-420 for 48 weeks

Treatment:

Drug: SP-420

Cohort 2a-2b

Experimental group

Description:

SP-420 for 48 weeks

Treatment:

Drug: SP-420

Cohort 3a-3b

Experimental group

Description:

SP-420 for 48 weeks

Treatment:

Drug: SP-420

1c-1d

Experimental group

Description:

SP-420 for 48 weeks

Treatment:

Drug: SP-420

2c-2d

Experimental group

Description:

SP-420 for 48 weeks

Treatment:

Drug: SP-420

3c-3d

Experimental group

Description:

SP-420 for 48 weeks

Treatment:

Drug: SP-420