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Gene Therapy

LentiGlobin BB305 Drug Product for Beta 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

Study Summary

This trial is testing a new treatment for transfusion-dependent β-thalassemia (TDT), which is a blood disorder. The treatment is autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product. The trial will enroll 18 participants who are less than or equal to 50 years of age and who have a β0/β0, β0/IVS-I-110, or IVS-I-110/IVS-I-110 genotype. The trial will evaluate the efficacy and safety of the treatment.

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

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

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
2,024 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia
2017. "A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03207009.
~2 spots leftby Apr 2025
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