Study of efficacy and safety of INC424 in regularly transfused patients with thalassemia.
- Conditions
- MedDRA version: 16.1Level: LLTClassification code 10054658Term: ThalassemiaSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Cancer [C04]Thalassemia
- Registration Number
- EUCTR2013-002812-28-IT
- Lead Sponsor
- OVARTIS FARMA S.p.A
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 30
1. Patients 18 years of age or older
2. Patients with thalassemia on a regular and stable transfusion regimen (at least 2 RBC units within every 4-week interval for 24 weeks prior to Screening) and anticipated to receive the same transfusion regimen during the study.
3. Patients with spleen enlargement at Screening, defined as
a. Spleen palpable below the costal margin, AND
b. Spleen volume of = 450 cm3 as confirmed by MRI (or CT scan in applicable patients)
4. Patients need to be on iron chelation treatment (deferoxamine or deferasirox) for at least four weeks prior to Screening.
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 25
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5
1. Splenectomy prior to or planned during the study
2. Active serious bacterial, mycobacterial, fungal, parasitic or viral infection which requires therapy (e.g., pneumonia, tuberculosis, systemic mycosis, herpes zoster)
3. Hemoglobin <65 g/L (<4.0 mmol/L) at Screening
4. Platelet count < 75 109/L, absolute neutrophils count < 1.5 109/L at Screening
5. Estimated MDRD < 30 mL/min/1.73 m2 at Screening
6. ALT (SGPT) levels >5 times ULN at Screening
7. Hepatocellular disease such as hepatitis B (presence of HBs antigen), hepatitis C (presence of HCV RNA), liver cirrhosis
8. HIV positivity
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To assess the effect of INC424 on transfusion requirement;Secondary Objective: Key secondary:<br>- to assess the effect of INC424 on spleen volume<br><br>Other secondary:<br>- to assess the effect of INC424 on pre transfusion hemoglobin levels<br>- to assess the effect of INC424 ib spleen lenght<br>- to assess pharmacokinetics of INC424<br>- to assess the safety of INC424;Primary end point(s): Percent change of RBC (Red Blood Cell) transfusion requirement between week 6 and week 30 compared to the baseline period between week -24 and the day before first study drug administration;Timepoint(s) of evaluation of this end point: week -24 to baseline period against week 6 to week 30
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Key secondary:<br>- change of spleen volume from baseline measured by MRI or CT at week 12 and week 30<br><br>Other secondary:<br>- change of pre-transfusion hemoglobin level from baseline at each post-baseline visit<br>- change of spleen length from baseline over time measured by palpation<br>- pharmacokinetic (PK) parameters of Cmin and Cmax (1h) of INC424 by actual dose administered<br>- number of participants with adverse events as measure of satefy and tolerability (adverse events, serious adverse events, lab results, ECGs, vital signs);Timepoint(s) of evaluation of this end point: for Key secondary:<br>- baseline, week 12 and week 30<br><br>for Other secondary:<br>- baseline; week 1, 2, 3, 4, 6, 12, 18, 24, 30<br>- baseline; week 1, 2, 3, 4, 6, 12, 18, 24, 30<br>- baseline, week 2 and week 12<br>- baseline; week 1, 2, 3, 4, 6, 12, 18, 24, 30