Oral drug treatment for beta thalassemia intermedia and sickle cell disease
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
This study is testing a new oral medication to see if it can help adults with beta thalassemia intermedia and sickle cell disease by increasing their fetal globin levels and improving their anemia.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 36 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Phoenicia BioScience Academic / other |
| Locations | 5 sites (Oakland, California and 4 other locations) |
| Trial ID | NCT04432623 on ClinicalTrials.gov |
What this trial studies
This trial evaluates an oral drug designed to increase fetal globin levels in patients with beta thalassemia intermedia and sickle cell disease. The study will assess the safety and efficacy of the drug through a phased approach, starting with three different doses administered to adult participants. Patients will take the drug daily for 12 weeks, with regular monitoring of their health and blood parameters. The goal is to identify a dose that significantly raises fetal globin levels, potentially improving anemia and related complications.
Who should consider this trial
Good fit: Ideal candidates are adults over 18 with beta thalassemia intermedia or sickle cell disease and specific hemoglobin levels.
Not a fit: Patients who have received recent blood transfusions or are on chronic transfusion programs may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly reduce anemia and improve the quality of life for patients with beta thalassemia intermedia and sickle cell disease.
How similar studies have performed: Other studies have shown promise in increasing fetal globin levels, but this specific approach is novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 Criteria: * 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 this trial is running
Oakland, California and 4 other locations
- UCSF Benioff Children's Hospital at Oakland — Oakland, California, United States (Completed)
- Massachusetts General Hospital — Boston, Massachusetts, United States (Completed)
- Susan Perrine — Weston, Massachusetts, United States (Active_not_recruiting)
- Weil Cornell Medicine — New York, New York, United States (Completed)
- University Health Network and Toronto General Hospital — Toronto, Ontario, Canada (Recruiting)
Study contacts
- Principal investigator: Kevin Kuo, MD — University Health Network, Toronto General Hospital
- Study coordinator: Susan Perrine, MD
- Email: sperrine@bu.edu
- Phone: 617 335-7002
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.