Status:
ENROLLING_BY_INVITATION
The BENeFiTS Trial in Beta Thalassemia Intermedia
Lead Sponsor:
Phoenicia BioScience
Collaborating Sponsors:
National Heart, Lung, and Blood Institute (NHLBI)
Conditions:
Beta Thalassemia Intermedia
Sickle Cell Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief Summary
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 thro...
Detailed Description
The study will first evaluate 3 doses of the investigational drug which are considered safe with chronic use in a combination therapeutic used widely for a different disease in Europe and Canada. The ...
Eligibility Criteria
Inclusion
- 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
Exclusion
- 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
Key Trial Info
Start Date :
October 5 2020
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
February 28 2026
Estimated Enrollment :
36 Patients enrolled
Trial Details
Trial ID
NCT04432623
Start Date
October 5 2020
End Date
February 28 2026
Last Update
August 15 2025
Active Locations (5)
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1
UCSF Benioff Children's Hospital at Oakland
Oakland, California, United States, 94609
2
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
3
Susan Perrine
Weston, Massachusetts, United States, 02493
4
Weil Cornell Medicine
New York, New York, United States, 10065