The BENeFiTS Trial in Beta Thalassemia Intermedia

Enrolling by invitation · Phase 1/Phase 2

Conditions studied: Beta Thalassemia Intermedia, Sickle Cell Disease

In brief

Beta-thalassemias and hemoglobinopathies are serious inherited blood diseases caused by abnormal or deficiency of beta A chains of hemoglobin, the protein in red blood cells which delivers oxygen throughout the body.The diseases are characterized by hemolytic anemia, organ damage, and early mortality without treatment. Increases in another type of (normal) hemoglobin, fetal globin (HbF), which is normally silenced in infancy, reduces anemia and morbidity. Even incremental augmentation of fetal globin is established to reduce red blood cell pathology, anemia, certain complications, and to improve survival. This trial will evaluate an oral drug discovered in a high throughput screen, which increases fetal globin protein (HbF and red blood cells expressing HbF)and messenger ribonucleic acid (mRNA) to high levels in anemic nonhuman primates and in transgenic mice. The study drug acts by suppressing 4 repressors of the fetal globin gene promoter in progenitor cells from patients. The drug has been used for 50 years in a combination product for different actions - to enhance half-life and reduce side effects of a different active drug- and is considered safe for long-term use. This trial will first evaluate 3 dose levels in small cohorts of nontransfused patients with beta thalassemia intermedia. The most active dose will then be evaluated in larger subject groups with beta thalassemia and other hemoglobinopathies, such as sickle cell disease.

Key facts

Study ID

NCT04432623

Run by

Phoenicia BioScience

People needed

36

Starts

2020-10-05

Expected to finish

2026-02-28

Last updated by the study team

2025-08-15

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Beta thalassemia intermedia (BTI) or (NTDT, Non-Transfusion Dependent Thalassemia) with at least one documented beta thalassemia mutation, including HbE beta thalassemia or an established diagnosis of sickle cell disease
• >18 years of age at time of consent
• Average of 2 total hemoglobin (Hgb) levels between 6.0 and 10.0 g/dL in the preceding 6 months
• Able and willing to give consent and comply with all study procedures
• If female and of childbearing potential, must have a documented negative pregnancy test prior to entry and agree to use one or more locally medically accepted methods of contraception

You may not qualify if…

• Red blood cell (RBC) transfusion within 2 months prior to administration of study medication
• Participating in a chronic transfusion program
• Pulmonary hypertension requiring oxygen therapy
• Use of erythropoiesis stimulating agents within 90 days of first dose
• Transaminases > 3 times upper limit of institution normal (ULN)
• Total and direct bilirubin > 3 times institution ULN unless due solely to hemolysis
• Known infection with HIV or hepatitis C (untreated)
• Fever > 38.5°C in the week prior to first administration of study medication
• History of osteoporosis or osteomalacia with a fragility fracture
• Received other investigational systemic therapy within 30 days prior to first dose
• Narrow angle glaucoma
• Currently pregnant or breast feeding a child
• Known current drug or alcohol abuse
• Taking monoamine oxidase inhibitors
• Other co-morbidity that substantially increases subject risk for the study per Investigator discretion

Where it is running

• UCSF Benioff Children's Hospital at Oakland — Oakland, California, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Susan Perrine — Weston, Massachusetts, United States
• Weil Cornell Medicine — New York, New York, United States
• University Health Network and Toronto General Hospital — Toronto, Ontario, Canada

Full record on ClinicalTrials.gov

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