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Gene Editing Therapy

ST-400 Investigational product for Beta Thalassemia

Phase 1 & 2
Waitlist Available
Research Sponsored by Sangamo Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be between 18 and 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from baseline (2 years prior to screening/consent), to st-400 infusion (day 0), after hematopoietic reconstitution and up to 156 weeks (post st-400 infusion)
Awards & highlights

Study Summary

This trial is testing a new gene therapy for people with transfusion-dependent beta thalassemia, a rare blood disorder. The therapy uses the patient's own blood stem cells, which are genetically modified in the laboratory to disrupt a sequence of the enhancer of the BCL11A gene. This is intended to boost fetal hemoglobin production. The therapy is then infused back into the patient. The primary objective is to understand safety and tolerability of the therapy, and secondary objectives are to assess the effects on hemoglobin levels and transfusion requirements.

Eligible Conditions
  • Beta Thalassemia

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from baseline (2 years prior to screening/consent), to st-400 infusion (day 0), after hematopoietic reconstitution and up to 156 weeks (post st-400 infusion)
This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline (2 years prior to screening/consent), to st-400 infusion (day 0), after hematopoietic reconstitution and up to 156 weeks (post st-400 infusion) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) up to 156 Weeks After the ST-400 Infusion
Secondary outcome measures
Annualized Frequency of Packed RBC Transfusions
Annualized Volume (mL) of Packed RBC Transfusions
Clinical Laboratory Measurement of Hemoglobin (Hb) Fractions (A and F in g/dL)
+1 more

Side effects data

From 2022 Phase 1 & 2 trial • 5 Patients • NCT03432364
100%
Insomnia
100%
Nausea
100%
Arthralgia
80%
Febrile neutropenia
80%
Anaemia
80%
Constipation
80%
Stomatitis
60%
Diarrhoea
60%
Alopecia
60%
Vascular access site pain
60%
Neutropenia
60%
Headache
60%
Procedural pain
60%
Thrombocytopenia
40%
Depression
40%
Sinus tachycardia
40%
Viral upper respiratory tract infection
40%
Epistaxis
40%
Anxiety
40%
Dehydration
40%
Hypophosphataemia
40%
Road traffic accident
40%
Bone pain
40%
Dysgeusia
40%
Hypokalaemia
40%
Iron overload
40%
Tachycardia
40%
COVID-19
40%
White blood cell count decreased
40%
Back pain
40%
Upper respiratory tract infection
40%
Hypoxia
40%
Platelet count decreased
40%
Leukocytosis
20%
Hypertension
20%
Dry eye
20%
Infusion related reaction
20%
Vomiting
20%
Hepatic enzyme increased
20%
Hypotension
20%
Neutrophil count decreased
20%
Influenza
20%
International normalised ratio increased
20%
Myalgia
20%
Vitamin D deficiency
20%
Abdominal pain upper
20%
Dyspepsia
20%
Vascular access site haemorrhage
20%
Hypomagnesaemia
20%
Haematuria
20%
Dysuria
20%
Bacterial sepsis
20%
Coxsackie viral infection
20%
Sinusitis
20%
Skin hyperpigmentation
20%
Hyperuricaemia
20%
Fatigue
20%
Hypersensitivity
20%
Decreased appetite
20%
Cholelithiasis
20%
Laryngeal inflammation
20%
Restless legs syndrome
20%
Incision site pain
20%
Migraine
20%
Lymphocyte count decreased
20%
Skin hypopigmentation
20%
Hyperglycaemia
20%
Palpitations
20%
Haemoperitoneum
20%
Peripheral swelling
20%
Fluid overload
20%
Hyperaesthesia
20%
Rash
20%
Leukopenia
20%
Flank pain
20%
Transfusion reaction
20%
Stasis dermatitis
20%
Abdominal pain
20%
Pneumonia
20%
Drug withdrawal syndrome
20%
Gastrooesophageal reflux disease
20%
Osteoporosis
20%
Non-cardiac chest pain
20%
Pain in extremity
20%
Respiratory syncytial virus infection
20%
Bradypnoea
20%
Vision blurred
20%
Post-traumatic pain
20%
Ovarian cyst ruptured
20%
Traumatic haematoma
20%
Vaccination complication
20%
Dry skin
20%
Cough
100%
80%
60%
40%
20%
0%
Study treatment Arm
ST-400 Investigational Product

Trial Design

1Treatment groups
Experimental Treatment
Group I: ST-400 Investigational productExperimental Treatment1 Intervention
ST-400 Investigational product is composed of autologous CD34+ hematopoietic stem/progenitor cells that are genetically modified ex vivo at the erythroid-specific enhancer of the BCL11A gene
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
ST-400 Investigational product
2018
Completed Phase 2
~10

Find a Location

Who is running the clinical trial?

Sangamo TherapeuticsLead Sponsor
28 Previous Clinical Trials
944 Total Patients Enrolled
SanofiIndustry Sponsor
2,164 Previous Clinical Trials
3,514,975 Total Patients Enrolled
Medical MonitorStudy DirectorSangamo Therapeutics, Inc.
1,649 Previous Clinical Trials
979,720 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
Progress in Molecular Biology and Translational ScienceJournal
Genome Editing Approaches to Β-hemoglobinopathies
Brusson, Mégane, and Annarita Miccio. 2021. “Genome Editing Approaches to Β-hemoglobinopathies”. Progress in Molecular Biology and Translational Science. Elsevier. doi:10.1016/bs.pmbts.2021.01.025.
clinicaltrials.govStudy
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
2018. "A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03432364.
~1 spots leftby Apr 2025
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