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-β00

MEDIAN AGE

(YEARS) (MIN, MAX)

13

(4, 34)
9 adult, 14 pediatric*

PRE-ENROLLMENTt
TRANSFUSION VOLUME

(mL/kg/YEAR)
MEDIAN (MIN, MAX)

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-β00 patients
were IVS-I-110 homozygous
or β0/IVS-I-110)

12

β00

6

non-β00

MEDIAN AGE

(YEARS) (MIN, MAX)

13

(4, 33)
5 adult, 13 pediatric*

PRE-ENROLLMENTt
TRANSFUSION VOLUME

(mL/kg/YEAR)
MEDIAN (MIN, MAX)

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

As of March 2021, all patients who completed a study enrolled in a long-term study 3 (LTF-303).

Characteristics of all study participants (N=41)

GENDER1

Gender

49%

FEMALE

51%

MALE

LOCATION AND ETHNICITY1,2

Location and Ethnicity

49%

ASIAN

44%

WHITE/
CAUCASIAN

FRANCE • GERMANY • GREECE • ITALY • THAILAND • UK • US

IRON LEVELS3,4
Cardiac

Cardiac T2* (msec)
median (min, max) was
37 (15, 75)

Iron levels

Liver iron concentration
(mg/g) median (min, max) was
4.9 (1.0–41.0)

Serum ferritin

Serum ferritin (pmol/L)
median (min, max) was
3671.9 (784, 22517)

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