NCT04432623Enrolling by invitationPhase 1/2
The BENeFiTS Trial in Beta Thalassemia Intermedia
A Phase 1b Sequential Open Label Dose-Ranging Study of Safety, Pharmacokinetics, and Preliminary Activity of Benserazide in Subjects With Beta Thalassemia Intermedia and Sickle Cell Disease
Source: ClinicalTrials.gov · Last updated 15 Aug 2025 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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.
- Status (registry)
- Enrolling by invitation
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 36 (estimated)
- First submitted
- 9 Jun 2020
- Start date
- 5 Oct 2020
- Primary completion date
- 28 Feb 2026
- Lead sponsor
- Phoenicia BioScience
- Diseases
- Sickle cell disease, Thalassaemia
- NCT number
- NCT04432623
What you commit to
- Study type: Interventional · Phase 1/2
- Planned participants: 36
- From 5 Oct 2020 to the primary completion date, 28 Feb 2026
- 5 sites in 2 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Red blood cell (RBC) transfusion within 2 months prior to administration of study medication
- Fever \> 38.5°C in the week prior to first administration of study medication
- Received other investigational systemic therapy within 30 days prior to first dose
- Known current drug or alcohol abuse
- Taking monoamine oxidase inhibitors
Other illness
- Pulmonary hypertension requiring oxygen therapy
- Known infection with HIV or hepatitis C (untreated)
- History of osteoporosis or osteomalacia with a fragility fracture
Lab values
- Transaminases \> 3 times upper limit of institution normal (ULN)
- Total and direct bilirubin \> 3 times institution ULN unless due solely to hemolysis
Other
- Participating in a chronic transfusion program
- Use of erythropoiesis stimulating agents within 90 days of first dose
- Narrow angle glaucoma
- Currently pregnant or breast feeding a child
- Other co-morbidity that substantially increases subject risk for the study per Investigator discretion
Who the study is looking for
- 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
Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers
Sites (5)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| University Health Network and Toronto General Hospital Coordinator of this site? Confirm its status | Toronto | Canada | Unknown status |
| Massachusetts General Hospital Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| Weil Cornell Medicine Coordinator of this site? Confirm its status | New York | United States | Unknown status |
| UCSF Benioff Children's Hospital at Oakland Coordinator of this site? Confirm its status | Oakland | United States | Unknown status |
| Susan Perrine Coordinator of this site? Confirm its status | Weston | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.