Evaluated across ages and genotypes1
41 patients aged 4 to 34 years were part of the safety and efficacy assessment of ZYNTEGLO®
- Both phase 3 studies were open-label, single-arm, 24-month studies
- After completing the parent study, patients were invited to enroll in Study 3 (LTF-303), a 13-year follow-up study in safety and efficacy
- Patients enrolled in all studies had diagnosis of beta-thalassemia and received regular RBC transfusions (a history of at least 100 mL/kg/year of RBCs or ≥8 transfusions of RBCs per year for the prior 2 years)
- All patients were administered ZYNTEGLO with a median (min, max) dose of 9.4 (5.0, 42.1) x 106 CD34+ cells/kg as an intravenous infusion (N=41)
The primary outcome for Study 1 and Study 2 was transfusion independence.
Transfusion independence: a weighted average Hb ≥9 g/dL without any packed red blood cell transfusions for a continuous period of ≥12 months at any time during the study after ZYNTEGLO infusion.1
The two phase 3 studies are ongoing. All data is as of March 2021.1,2
STUDY 1: HGB-207 | |
PATIENTS ENROLLED IN PARENT STUDY |
23 |
PATIENTS ENROLLED IN LTF-303 STUDY |
19 |
MEDIAN DURATIONt
OF FOLLOW-UP MONTHS (MIN, MAX) |
29.5
(13.0, 48.2) |
GENOTYPE | 23
non-β0/β0 |
MEDIAN AGE
(YEARS) (MIN, MAX) |
13
(4, 34) |
PRE-ENROLLMENTt TRANSFUSION VOLUME (mL/kg/YEAR) |
208
(142, 274) |
PRE-ENROLLMENTt NUMBER OF TRANSFUSIONS PER YEAR MEDIAN (MIN, MAX) |
16
(12, 37) |
STUDY 2: HGB-212 | |
PATIENTS ENROLLED IN PARENT STUDY |
18 |
PATIENTS ENROLLED IN LTF-303 STUDY |
10 |
MEDIAN DURATIONt
OF FOLLOW-UP MONTHS (MIN, MAX) |
24.6
(4.1, 35.5) |
GENOTYPE
(Non-β0/β0 patients |
12
β0/β0‡ |
6
non-β0/β0 |
|
MEDIAN AGE
(YEARS) (MIN, MAX) |
13
(4, 33) |
PRE-ENROLLMENTt TRANSFUSION VOLUME (mL/kg/YEAR) |
194
(75, 289) |
PRE-ENROLLMENTt NUMBER OF TRANSFUSIONS PER YEAR MEDIAN (MIN, MAX) |
17
(11, 40) |
*The phase 3 studies included 16 children (<12 years) and 11 adolescents (12 years to <18 years).
†Data beyond 24 months are from the long-term follow-up study, Study 3 (LTF-303).1
Characteristics of all study participants (N=41)
GENDER1
49%
FEMALE
51%
MALE
LOCATION AND ETHNICITY1,2
49%
ASIAN
44%
WHITE/
CAUCASIAN
FRANCE • GERMANY • GREECE • ITALY • THAILAND • UK • US
IRON LEVELS3,4
Cardiac T2* (msec)
median (min, max) was
37 (15, 75)
Liver iron concentration
(mg/g) median (min, max) was
4.9 (1.0–41.0)
Serum ferritin (pmol/L)
median (min, max) was
3671.9 (784, 22517)
Explore Next