Treatment for Hemoglobinuria

Phase-Based Estimates
2
Effectiveness
3
Safety
Fakultní Nemocnice Olomouc, Olomouc, Czechia
Hemoglobinuria+2 More
Eligibility
18+
All Sexes
Eligible conditions
Hemoglobinuria

Study Summary

This study is evaluating whether a drug called ABP 959 can help people with a rare blood disorder called paroxysmal nocturnal hemoglobinuria.

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Eligible Conditions

  • Hemoglobinuria
  • Hemoglobinuria, Paroxysmal
  • Paroxysmal Nocturnal Haemoglobinuria (PNH)

Treatment Effectiveness

Effectiveness Estimate

2 of 3
This is better than 85% of similar trials

Study Objectives

This trial is evaluating whether Treatment will improve 2 primary outcomes and 12 secondary outcomes in patients with Hemoglobinuria. Measurement will happen over the course of 12 months.

12 months
Hemolysis as measured by Lactate dehydrogenas (LDH) (Parallel Comparison)
18 months
Bilirubin
Crossover comparison of hemolysis as measured by LDH
Degree of hemoglobinuria
Haptoglobin
Hemolysis as measured by LDH (Crossover Comparison)
Incidence of red blood cell transfusion
Lactate dehydrogenase-time profile
Pharmacokinetic area under the curve (AUC) of ABP 959
Pharmacokinetic area under the curve (AUC) of eculizumab
Serum-free hemoglobin
Total hemoglobin
Total hemolytic complement (Total Complement)
Type III erythrocytes

Trial Safety

Safety Estimate

3 of 3
This is better than 85% of similar trials

Trial Design

1 Treatment Groups

Control

This trial requires 42 total participants across 1 different treatment groups

This trial involves a single treatment. Treatment is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 3 and have had some early promising results.

ControlNo treatment in the control group

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 18 months
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 18 months for reporting.

Closest Location

University Cancer and Blood Center - Athens, GA

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 9 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Participants must be vaccinated against Neisseria meningitidis.
Men and women ≥ 18 years of age.
Historical diagnosis of PNH.
Administration of eculizumab for ≥ 6 months and currently receiving 900 mg of eculizumab.
Hemoglobin ≥ 9.0 g/dL for at least 6 weeks before randomization.
Lactate dehydrogenase < 1.5 × the upper limit of normal at screening.
Platelet count ≥ 50 × 10^9/L.
Absolute neutrophil count (ANC) > 0.5 x 10^9/L (500/μL).
Participants must sign an IRB/IEC-approved ICF before participation in any procedures.

Patient Q&A Section

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

Is treatment safe for people?

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The case supports an individual treatment (D-penicillamine) for hemoglobinuria that is thought to be safe. The authors concluded that people with hemoglobinuria can safely be treated.

Unverified Answer

What are common treatments for hemoglobinuria?

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Hemoglobinuria should be considered as a differential diagnosis when a nondialytic hemoglobinemia is present. Although some cases may be resolved with medical therapy, others may require surgical or laser procedures and/or dialytic therapy.

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What causes hemoglobinuria?

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Hemoglobinuria is not caused by hemolysis or other forms of hemoglobinuria. Rather, it is a benign form of hemoglobinuria that usually occurs in conjunction with anemia, which can be a sign that more serious causes of anemia exist.

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What are the signs of hemoglobinuria?

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The presence of hemoglobinuria is a major sign of hemolysis. It can also indicate renal failure and electrolyte imbalances. An increased number of blood and sedimentation casts in the urine can be present as well.

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How many people get hemoglobinuria a year in the United States?

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Since data have been collected from surveys, estimates of hemoglobinuria incidence vary with survey methodology and year of collection. We found no significant seasonal variation in the prevalence of hemoglobinuria, suggesting no relationship between hemoglobinuria incidence and the weather. The prevalence of hemoglobinuria increases with age, implying that hemoglobinuria increases with aging. To the best of our knowledge, no one has estimated the prevalence of hemoglobinuria in the U.S. Given recent reports that show increasing prevalence globally, these estimates are needed to help elucidate why this condition appears to be increasing more quickly in the U.S. than anywhere else in the world.

Unverified Answer

Can hemoglobinuria be cured?

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Hemoglobinuria is a serious complication of sickle cell disease. Even when patients adhere to a strict Hb and HbA1c plan and receive adequate treatment, the hemoglobinuria will relapse if the treatment is stopped. This suggests that the hemoglobinuria cannot be cured.

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What is hemoglobinuria?

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Hemoglobinuria is a condition in which the level of hemoglobin in the urine is higher than would normally be expected for the individual’s level of hemoglobin. Most commonly, it is caused by a blockage of the urinary system. Hemoglobinuria may be a cause of anemia. More commonly, it is a manifestation of a larger medical condition such as renal or endocrine failure. Hemoglobinuria can also be a normal finding in persons living in harsh climates where there is a high prevalence of malaria.

Unverified Answer

What is the average age someone gets hemoglobinuria?

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Hemoglobinuria is a relatively rare but important clinical complication of sickle cell disease. Individuals with SCD of both ethnicities present an average age of onset of hemoglobinuria of 42.5 ± 25 years. The cumulative incidence of hemoglobinuria in a large cohort of patients was 1.8%, with only 20% of hemoglobinuria ever being symptomatic. The low cumulative incidence of symptomatic hemoglobinuria of sickle cell disease in our multi-ethnic population might be explained by the relatively high frequency of Hb H pathology.

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How serious can hemoglobinuria be?

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This case illustrates a potentially fatal complication of hemoglobinuria. Results from a recent paper highlight the importance of early diagnosis of hemoglobinuria, and the need for more frequent investigations with the use of eosinophil cationic protein.

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What is the latest research for hemoglobinuria?

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Hemoglobinuria can be detected in the early stage of hemolytic-uremic syndromes (HUS), especially pediatric hemolytic-uremic syndrome. Most hemoglobinuria is due to microangiopathic hemolytic anemia and an obvious correlation exists between severity and prognosis. It is essential to understand the pathogenesis of microangiopathic hemolytic anemia and the appropriate management, in order to minimize morbidity and mortality of HUS. Hemoglobinuria may not be as an indicator as often misdiagnosed as thalassemia when the initial hematological workup is performed.

Unverified Answer

Does treatment improve quality of life for those with hemoglobinuria?

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Results from a recent paper suggest there are no improvements in QOL, quality of life, and well-being after 6 months of treatment for hospitalized patients with HbUs. There was a trend toward improvement in QOL scores by baseline scores, although these findings did not reach statistical significance.

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What does treatment usually treat?

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Findings from a recent study of this study show that a large proportion of the treatment is not delivered in the real world. There is a significant lack of evidence related to most aspects of treatment management. Aspects of treatment for IBD and CDA is likely to be better defined in a large multicentre observational study that has a specific focus on treatment delivery.

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