START Study

Tahapan Penelitian : Awal
Sponsor:
Mitra Pelaksana:
PT Quintiles Indonesia
No Registry
Tanggal Input Registry : 05-03-2019

11-03-2019
The percentage of patients who still have a serum ferritin level < 1000 μg/L at Month 12.
• The percentage of patients whose serum ferritin level is still less than 1000 μg/L at each visit • The percentage of patients whose LPI value is still less than 0.6 μM at each visit • The percentage of patients in each treatment arm whose TSAT value is still less than 60% at each visit • Time to reach a serum ferritin level ≥ 1000 μg/L • Time to reach an LPI value ≥ 0.6 μM • Time to reach a TSAT value ≥ 60% Safety: • Adverse events (AEs): Frequency, intensity, time to onset, duration, and relatedness to study drug • Serious adverse events (SAEs): Frequency, intensity, time to onset, duration, and relatedness to study drug • Number of discontinuations due to AEs • Growth parameters: Weight, height, and height velocity, as classified using the Z-score system • Change in prolactin level from baseline to Month 12
 
START Study
Safety and efficacy of early-start deferiprone treatment in infants and young children newly diagnosed with transfusion-dependent beta thalassemia
Observasional
Investigational product: - Product: Deferiprone oral solution 80 mg/mL - Dose: 25, 50, or 75 mg/kg/day, divided t.i.d. - Mode of administration: Oral Reference product: - Product: Placebo - Dose: Volume of solution matching that required for a 25, 50, or 75 mg/kg/day dose of active product, divided t.i.d. - Mode of administration: Oral
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Inclusion Criteria:

• Male or female aged ≥ 6 months to < 10 years • Confirmed diagnosis of beta-thalassemia, as determined by high performance liquid chromatography (HPLC) or DNA testing • Started on a regular RBC transfusion regimen, with a minimum of 2 transfusions already completed • Screening level of serum ferritin greater than >200 g/L but not more than 600 μg/L. Since SF level may be impacted by the presence of infection, it must additionally be verified that the child has had no signs of infection in the previous 7 days, including the day of screening, and that the level of C-reactive protein (CRP) is no greater than 20% higher than the normal range for the patient’s age. If there are signs of infection and/or the CRP level is above this threshold, the SF level must be checked again a minimum of one week later. (Note: If an investigator has valid reason to believe that an out-of-range SF level may be attributable to infection even if this is not indicated by the CRP result, SF may be rechecked once more a minimum of one week later.)

Exclusion Criteria:

• Prior use of iron chelation • Diagnosis of hepatitis B or C, or HIV infection • Evidence of abnormal liver or kidney function at screening (serum ALT level > 5 times upper limit of normal or creatinine levels >2 times upper limit of normal) • Disorders associated with neutropenia (ANC < 1.5 x 109/L) prior to the initiation of study medication Exception: Patients whose neutropenia is attributed by the treating physician to episodes of infection or to drugs associated with a decline in the neutrophil count and in whom the ANC has returned to a normal level at the screening visit.
 
Ethical approval from Ethics Committee Medical Faculty of University of Indonesia, date 09 Jul 2018, letter no. 0669/UN2.F1/ETIK/2018 and Ethical approval from Health Research Ethics Committee of Dr. Hasan Sadikin General Hospital Bandung, date 14 Sep 2018, letter no. LB.04.01/A05/EC/270/IX/2018
MTA Approval, date 04 Feb 2019, letter no. LB.02.01/I/619/2019
BPOM Approval, date 04 Jan 2019, letter no. B-PN.01.06.3.323.01.19.0040 (for site 006) and BPOM Approval, date 04 Jan 2019, letter no. B-PN.01.06.3.323.01.19.0039 (for site 007)
LA55-0417
Contact for Scientific Queries: PI at site 006: Dr. dr. Pustika Amalia Wahidiyat SpA(K); Address: Dr. Cipto Mangunkusumo Hospital – Faculty of Medicine University of Indonesia, Pediatric Department Jalan Pangeran Diponegoro No.71, RW.5, Kenari, Senen, Jakarta Pusat, Daerah Khusus Ibukota Jakarta 10430, Indonesia I PI at site 007: Dr.dr.Lelani Reniarti, SpA(K), M.Kes; Address: Department of Child Health Hasan Sadikin Hospital, Faculty of Medicine Universitas Padjadjaran, Jalan Pasteur No 38 Bandung Indonesia II Contact for Public Queries: Phone no.: 021-29537947; Email address: wike.veranike@iqvia.com; Office address: PT Quintiles Indonesia (IQVIA), Wisma Nugra Santana 11th Floor, Jl. Jend Sudirman Kav 7-8, Jakarta 10220 Indonesia