Evaluated across ages and genotypes1
41 patients aged 4 to 34 years were part of the safety and efficacy assessment of ZYNTEGLO™ 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 |
STUDY 2:
HGB-212 |
|
|
ENROLLED IN PARENT STUDY |
23
PATIENTS |
18
PATIENTS |
|
ENROLLED IN LTF-303 STUDY |
19
PATIENTS |
10
PATIENTS |
|
MEDIAN DURATION† OF FOLLOW-UP |
29.5
(13.0, 48.2) |
24.6
(4.1, 35.5) |
| GENOTYPE |
non-β0/β0 23 |
β0/β0
12 non-β0/β0* 6 (IVS-I-110 homozygous or β0/IVS-I-110) |
|
MEDIAN AGE (MIN, MAX) |
15 YEARS (4, 34) 9 adult, 14 pediatric* |
13
YEARS (4, 33) 5 adult, 13 pediatric* |
|
PRE-ENROLLMENT TRANSFUSION VOLUME (mL/kg/YEAR) MEDIAN (MIN, MAX) |
208 (142, 274) |
194
(75, 289) |
|
PRE-ENROLLMENT NUMBER OF TRANSFUSIONS PER YEAR MEDIAN (MIN, MAX) |
16 (12,37) |
17
(11, 40) |
KEY CHARACTERISTICS OF ALL
STUDY PARTICIPANTS (N=41)1,2
DEMOGRAPHICS
FEMALE
MALE
ASIAN
CAUCASIAN
- France
- Germany
- Greece
- Italy
- Thailand
- UK
- US
BASELINE IRON LEVELS3,4
37 (15, 75)
Cardiac T2* (msec)
median (min, max)
4.9 (1.0–41.0)
Liver iron concentration (mg/g)
median (min, max)
3671.9 (784, 22517)
Serum ferritin (pmol/L)
median (min, max)
*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
KEY CHARACTERISTICS OF ALL
STUDY PARTICIPANTS (N=41)1,2
DEMOGRAPHICS
FEMALE
MALE
ASIAN
CAUCASIAN
- France
- Germany
- Greece
- Italy
- Thailand
- UK
- US
BASELINE IRON LEVELS3,4
37 (15, 75)
Cardiac T2* (msec)
median (min, max)
4.9 (1.0–41.0)
Liver iron concentration (mg/g)
median (min, max)
3671.9 (784, 22517)
Serum ferritin (pmol/L)
median (min, max)
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