The BENeFiTS Trial in Beta Thalassemia Intermedia

Part of paid clinical trials in Oakland, California.

Sponsor
Phoenicia BioScience
Study ID
NCT04432623
Phase
PHASE1/PHASE2
Status
Enrolling By Invitation

Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Benserazide Only Product — DRUG
    Investigational drug

Study Details

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 Dates

Start date
Oct 5, 2020
Status verified
Aug 2025
Primary completion
Feb 28, 2026
Completion
Feb 28, 2026

Study Design

Enrollment
36 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Low dose
    A low dose, by mouth, once per day, on Monday, Wednesday, and Friday for 12 weeks
  • Experimental: Middle dose
    A middle dose, by mouth, once per day, on Monday, Wednesday, and Friday, for 12 weeks
  • Experimental: High dose 3 days per week
    Highest dose, by mouth, once per day, on Monday, Wednesday, and Friday, for 12 to 24 weeks
  • Experimental: High dose 5 days per week
    The highest dose, by mouth once per day on 5 days per week for 24 weeks
  • Experimental: Sickle Cell Disease Arm
    The most active dose given once per day on the most active regimen for up 24 weeks

Primary Outcome Measure

Safety and Tolerability [ Time Frame: 12 to 24 weeks ]

Locations (4)

FacilityCityStateZIPSite coordinators
UCSF Benioff Children's Hospital at OaklandOaklandCalifornia94609-
Massachusetts General HospitalBostonMassachusetts02114-
Susan PerrineWestonMassachusetts02493-
Weil Cornell MedicineNew YorkNew York10065-

Find similar trials in Oakland, CA

By condition
Sickle cell disease
By research site
UCSF Benioff Children's Hospital at Oakland· Oakland, CAMassachusetts General Hospital· Boston, MASusan Perrine· Weston, MAWeil Cornell Medicine· New York, NY

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