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Benserazide for Beta Thalassemia (PB04-001 Trial)

Phase 1 & 2
Recruiting
Led By Sylvia Singer, MD
Research Sponsored by Phoenicia BioScience
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 16 to 24 weeks
Awards & highlights

PB04-001 Trial Summary

This trial is testing a drug that could improve the lives of people with beta thalassemia or other hemoglobinopathies by increasing the levels of fetal globin protein.

Who is the study for?
The trial is for adults with Beta thalassemia intermedia or Non-Transfusion Dependent Thalassemia who have a specific mutation, with hemoglobin levels between 6.0 and 10.0 g/dL. Participants must not be on oxygen therapy for pulmonary hypertension, have certain liver conditions, untreated HIV or hepatitis C, recent fever, other recent treatments or transfusions, or be pregnant/breastfeeding.Check my eligibility
What is being tested?
An oral drug known as Benserazide Only Product is being tested to increase fetal globin in red blood cells which could benefit those with beta thalassemia and related blood disorders. The study starts by testing three dose levels in nontransfused patients before moving to larger groups.See study design
What are the potential side effects?
While the trial description does not specify side effects of Benserazide Only Product directly since it's considered safe for long-term use from past applications, potential risks may include typical drug reactions such as digestive issues, allergic responses or fatigue.

PB04-001 Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 18 years old or older.
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My average hemoglobin levels have been between 6.0 and 10.0 g/dL in the last 6 months.
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I am not pregnant and agree to use birth control during the study.
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I have a type of thalassemia that doesn't always require blood transfusions.

PB04-001 Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~16 to 24 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 16 to 24 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Maximum plasma concentration (Cmax)
Plasma drug concentration over time
Safety and Tolerability
Secondary outcome measures
F-cells
Fetal hemoglobin (HbF)
HbF protein per cell
+1 more

PB04-001 Trial Design

5Treatment groups
Experimental Treatment
Group I: Sickle Cell Disease ArmExperimental Treatment1 Intervention
The most active dose given once per day on the most active regimen for up 24 weeks
Group II: Middle doseExperimental Treatment1 Intervention
A middle dose, by mouth, once per day, on Monday, Wednesday, and Friday, for 12 weeks
Group III: Low doseExperimental Treatment1 Intervention
A low dose, by mouth, once per day, on Monday, Wednesday, and Friday for 12 weeks
Group IV: High dose 5 days per weekExperimental Treatment1 Intervention
The highest dose, by mouth once per day on 5 days per week for 24 weeks
Group V: High dose 3 days per weekExperimental Treatment1 Intervention
Highest dose, by mouth, once per day, on Monday, Wednesday, and Friday, for 12 to 24 weeks

Find a Location

Who is running the clinical trial?

Phoenicia BioScienceLead Sponsor
National Heart, Lung, and Blood Institute (NHLBI)NIH
3,814 Previous Clinical Trials
47,290,839 Total Patients Enrolled
Sylvia Singer, MDPrincipal InvestigatorUCSF Benioff Children's Hospital at Oakland

Media Library

Benserazide Only Product (Other) Clinical Trial Eligibility Overview. Trial Name: NCT04432623 — Phase 1 & 2
Beta Thalassemia Research Study Groups: Low dose, Middle dose, High dose 3 days per week, High dose 5 days per week, Sickle Cell Disease Arm
Beta Thalassemia Clinical Trial 2023: Benserazide Only Product Highlights & Side Effects. Trial Name: NCT04432623 — Phase 1 & 2
Benserazide Only Product (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04432623 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What outcomes is this research endeavor hoping to accomplish?

"Over a 4 week period, the main goal of this research is to assess Safety and Tolerability. Secondary endpoints being measured are F-cells (percentage of red blood cells containing HbF), Soluble transferrin receptors (ng/ml) and HbF protein per cell (Mean fluorescent intensity [MFI])."

Answered by AI

How many participants are involved in this trial at present?

"For its successful completion, the trial necessitates 36 qualified patients. Those interested can register at clinics like Weil Cornell Medicine in New york City and University Health Network/Toronto General Hospital of Toronto, Canada."

Answered by AI

Are there any vacancies for participation in this experiment?

"As per clinicaltrials.gov, this medical experiment is currently in the process of recruiting volunteers and has been since October 5th 2020. The trial's details were last updated on April 9th 2022."

Answered by AI

What are the geographical points of reference for this trial?

"Presently, the trial is taking place at 5 different sites situated in New york, Toronto and Oakland as well as other cities. To reduce travel costs associated with enrollment, it may be wise to select a clinic located near you."

Answered by AI

Is Benserazide Only Product being tested in any other clinical trials?

"Currently, a single medical trial is currently underway to study the efficacy of Benserazide Only Product. This Phase 3 clinical trial is based in Boston, Massachusetts and there are 5 other locations that have been recruited for this research."

Answered by AI
~8 spots leftby Mar 2025