A study of a drug removing iron from the body of patients with transfusion-dependent β-thalassemia
Research type
Research Study
Full title
An open-label, dose-escalation, dose-finding, and proof-of-concept trial of SP-420 in subjects with transfusion-dependent β-thalassemia
IRAS ID
1008436
Contact name
MArtin Damm Olling
Contact email
Sponsor organisation
Pharmacosmos A/S
Eudract number
2022-002395-36
Research summary
This is a phase 2 open-label, dose escalation, dose-finding, and proof-of-concept multi-centre trial evaluating the iron clearing efficacy, safety, tolerability, and pharmacokinetic (PK) properties of SP-420 administered 3 times per week to participants with transfusion-dependant β-thalassemia for 48 weeks. Iron chelators are small molecules that form complexes with iron and promote iron excretion, these are used to treat β-thalassemia. Current licensed iron chelators are all associated with significant side effects and poor adherence in a considerable portion of patients. SP-420 is a novel tridentate iron chelator of the desferrithiocin class.
Approximately 90 participants will be enrolled in 3 dose cohorts of approximately 30 participants each. Dosing will start with 6 participants in each cohort (1a-3a) in a dose-escalating manner, and if no safety issues are identified, inclusion of additional 24 participants is allowed in each cohort (1b-3b). The study consists of 2 parts: part 1 with a fixed dosing regimen and part 2 with an adjustable dosing regimen. The duration of the trial will be approximately 52 weeks (including up to 4 weeks screening period and 48 weeks on treatment) and each participant in cohort 1a-3a will attend for a minimum of 20 visits and in cohort 1b-3b each participant will attend a minimum of 18 visits.
Participants will be assessed during the study using blood and urine samples as well as physical examinations, vital sign monitoring, MRIs to assess liver iron concentration (LIC) and cardiac iron content (CIC), electrocardiogram (ECG) and questionnaires.
REC name
East of England - Essex Research Ethics Committee
REC reference
24/EE/0022
Date of REC Opinion
12 Mar 2024
REC opinion
Further Information Unfavourable Opinion