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Gene Therapy

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

Phase 3
Waitlist Available
Research Sponsored by bluebird bio
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at month 6, 9, 12, 18 and 24
Awards & highlights
Pivotal Trial
No Placebo-Only Group

Summary

This study is evaluating whether a drug can be used to treat a blood disorder.

Eligible Conditions
  • Beta Thalassemia

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at month 6, 9, 12, 18 and 24
This trial's timeline: 3 weeks for screening, Varies for treatment, and at month 6, 9, 12, 18 and 24 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Percentage of Participants Who Have Achieved Transfusion Independence (TI)
Secondary study objectives
Annualized Number of pRBC Transfusions
Annualized Phlebotomy Therapy Usage Following Drug Product Infusion
Annualized Volume of pRBC Transfusions
+22 more

Side effects data

From 2022 Phase 3 trial • 19 Patients • NCT03207009
89%
Thrombocytopenia
84%
Neutropenia
79%
Anaemia
63%
Alopecia
63%
Vomiting
58%
Febrile neutropenia
58%
Stomatitis
47%
Procedural pain
47%
Nausea
42%
Alanine aminotransferase increased
42%
Abdominal pain
42%
Hypocalcaemia
37%
Epistaxis
37%
Aspartate aminotransferase increased
37%
Pyrexia
37%
Mucosal inflammation
37%
Headache
32%
Leukopenia
26%
Bone pain
26%
Decreased appetite
26%
Diarrhoea
26%
Skin hyperpigmentation
21%
Pharyngeal inflammation
21%
Back pain
21%
Anxiety
21%
Cough
21%
Hypertension
21%
Catheter site pain
21%
Hypoxia
21%
Constipation
21%
Rash
21%
Nasopharyngitis
16%
Abdominal pain upper
16%
Fatigue
16%
Pain
16%
Blood bilirubin increased
16%
Dyspnoea
16%
Dizziness
16%
Dry skin
16%
Erythema
16%
Pruritus
16%
Pain in extremity
16%
COVID-19
16%
Oral herpes
11%
Transfusion reaction
11%
Muscular weakness
11%
Hypokalaemia
11%
Rhinorrhoea
11%
Hypophosphataemia
11%
Insomnia
11%
Non-cardiac chest pain
11%
Puncture site pain
11%
Transaminases increased
11%
Anal inflammation
11%
Dyspepsia
11%
Haematuria
11%
Rash maculo-papular
11%
Myalgia
11%
Viral infection
5%
Blood testosterone decreased
5%
Chalazion
5%
Metabolic acidosis
5%
Weight decreased
5%
Syncope
5%
Ear pain
5%
Hypogonadism
5%
Pulmonary mass
5%
Hyponatraemia
5%
Post procedural discomfort
5%
Conjunctival haemorrhage
5%
Dry eye
5%
Secondary hypogonadism
5%
Tachycardia
5%
Lymphopenia
5%
Incision site pain
5%
Hypomagnesaemia
5%
Blood creatinine increased
5%
Gamma-glutamyltransferase increased
5%
Pancytopenia
5%
Upper respiratory tract inflammation
5%
Neutrophilia
5%
Vitamin K deficiency
5%
C-reactive protein increased
5%
Flank pain
5%
Coombs direct test positive
5%
Immune thrombocytopenia
5%
Lip dry
5%
Arthralgia
5%
Vulvovaginal candidiasis
5%
Laryngeal pain
5%
Musculoskeletal pain
5%
Mouth haemorrhage
5%
Cardiac failure congestive
5%
Application site swelling
5%
Feeling cold
5%
Bartholin's cyst
5%
Hypotension
5%
Adverse drug reaction
5%
Gastric ulcer perforation
5%
Soft tissue infection
5%
Device related infection
5%
Focal nodular hyperplasia
5%
Hot flush
5%
Application site pain
5%
Injection site reaction
5%
Hypersensitivity
5%
Vaginal haemorrhage
5%
Oropharyngeal pain
5%
Pulmonary function test decreased
5%
Contusion
5%
Skin abrasion
5%
Ageusia
5%
Paraesthesia
5%
Gingival bleeding
5%
Dermatitis contact
5%
Ecchymosis
5%
Hidradenitis
5%
Urticaria
5%
Dysuria
5%
Pollakiuria
5%
Proteinuria
5%
Delayed puberty
5%
Oral pain
5%
Palatal ulcer
5%
Cholelithiasis
5%
Hepatic mass
5%
Hyperbilirubinaemia
5%
Jaundice
5%
Intertrigo
5%
Petechiae
5%
Rash macular
5%
Skin exfoliation
5%
Skin hypopigmentation
5%
Osteopenia
5%
Upper respiratory tract infection
5%
Varicella zoster virus infection
5%
Bronchitis
5%
Cystitis
5%
Herpes zoster
5%
Infection
5%
Lice infestation
5%
Mucosal infection
5%
Pyelonephritis
5%
Rhinovirus infection
5%
Sinusitis
5%
Staphylococcal infection
5%
Systemic infection
5%
Hyperchloraemia
5%
Hyperphosphataemia
100%
80%
60%
40%
20%
0%
Study treatment Arm
LentiGlobin BB305 Drug Product

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: LentiGlobin BB305 Drug ProductExperimental Treatment1 Intervention
LentiGlobin BB305 Drug Product (autologous CD34+ cell-enriched population that contains cells transduced with LentiGlobin BB305 lentiviral vector encoding human βA-T87Q-globin)
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
LentiGlobin BB305 Drug Product
2013
Completed Phase 3
~70

Find a Location

Who is running the clinical trial?

bluebird bioLead Sponsor
20 Previous Clinical Trials
2,028 Total Patients Enrolled
Richard ColvinStudy Directorbluebird bio
3 Previous Clinical Trials
175 Total Patients Enrolled
Himal L Thakar, MDStudy Directorbluebird bio
1 Previous Clinical Trials
66 Total Patients Enrolled
~2 spots leftby Dec 2025
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