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Benserazide for Beta Thalassemia (PB04-001 Trial)

Phase 1 & 2
Recruiting
Led By Hanny D Al-Samkari, 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
Must not have
Pulmonary hypertension requiring oxygen therapy
Known infection with HIV or hepatitis C (untreated)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 16 to 24 weeks
Awards & highlights
No Placebo-Only Group

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.
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.
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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I need oxygen therapy for my high blood pressure in the lungs.
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I have an untreated HIV or hepatitis C infection.
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I have been diagnosed with narrow angle glaucoma.
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I am part of a program that requires regular blood transfusions.
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I haven't used any drugs to increase red blood cells in the last 90 days.
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I have not had a fever over 38.5°C in the last week.
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I am currently taking medication for depression or Parkinson's disease.
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I have had a fracture due to weak bones.

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

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

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

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
3,944 Previous Clinical Trials
47,799,793 Total Patients Enrolled
Hanny D Al-Samkari, MDPrincipal InvestigatorMassachusetts General Hospital
Sylvia Singer, MDPrincipal InvestigatorUCSF Benioff Children's Hospital at Oakland
Sujit Sheth, MD MSPrincipal InvestigatorWeill Medical College of Cornell University
Susan Perrine, MDStudy DirectorPhoenicia BioScience
Kevin Kuo, MDPrincipal InvestigatorUniversity Health Network, Toronto General Hospital

Media Library

Benserazide Only Product (Other) Clinical Trial Eligibility Overview. Trial Name: NCT04432623 — Phase 1 & 2
Sickle Cell Disease Research Study Groups: Low dose, Middle dose, High dose 3 days per week, High dose 5 days per week, Sickle Cell Disease Arm
Sickle Cell Disease 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
~0 spots leftby Dec 2024
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