rilzabrutinib for Warm Autoimmune Hemolytic Anemia (wAIHA)

Phase-Based Progress Estimates
Investigational Site Number 8260002, London, United Kingdom
Warm Autoimmune Hemolytic Anemia (wAIHA)+4 More
rilzabrutinib - Drug
All Sexes
Eligible conditions

Study Summary

This study is evaluating whether a drug can help treat a rare blood disorder.

See full description

Eligible Conditions

  • Warm Autoimmune Hemolytic Anemia (wAIHA)

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Warm Autoimmune Hemolytic Anemia (wAIHA)

Study Objectives

This trial is evaluating whether rilzabrutinib will improve 2 primary outcomes and 5 secondary outcomes in patients with Warm Autoimmune Hemolytic Anemia (wAIHA). Measurement will happen over the course of By Week 24 in Part A.

Week 75
Frequency of rescue therapy (any wAIHA-directed therapy other than predniso[lo]ne or transfusion) received
Week 50
Part B: Proportion of participants who maintain durable response achieved during Part A or achieve a durable response during Part B and have a hemoglobin response
Week 24
Part A: Proportion of participants with overall hemoglobin response
Proportion of participants with durable hemoglobin response
Week 24
Median time from baseline to first hemoglobin response
Week 75
Change from baseline in FACIT-Fatigue scale score
Incidence of treatment emergent adverse events (TEAEs), serious TEAEs, adverse events of special interest (AESIs)

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Warm Autoimmune Hemolytic Anemia (wAIHA)

Trial Design

1 Treatment Group

1 of 1
Experimental Treatment

This trial requires 20 total participants across 1 different treatment group

This trial involves a single treatment. Rilzabrutinib 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 2 and have already been tested with other people.

Oral rilzabrutinib 400 mg BID

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: after week 1 of treatment to week 24 in part a and week 75 in part b
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly after week 1 of treatment to week 24 in part a and week 75 in part b for reporting.

Closest Location

Investigational Site Number 8400001 - Boston, MA

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:
Male and female patients with a confirmed diagnosis of primary wAIHA or systemic lupus erythematosus (SLE)-associated wAIHA (without other SLE-related manifestations apart from cutaneous and musculoskeletal manifestations)
Participants who have previously failed to maintain a sustained response after treatment with corticosteroids.
Eastern Cooperative Oncology Group (ECOG) performance status grade 2 or lower.
Up-to-date vaccination status as per local guidelines.
Body mass index (BMI) >17.5 and <40 kg/m2
All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
Part B only
Evidence of treatment efficacy to rilzabrutinib as defined by achieving overall response during Part A.
Completion of Part A treatment period (24 weeks).

Patient Q&A Section

What are common treatments for anemia, hemolytic?

"In the pediatric population, multiple cases have been noted to have been treated with iron dextran solutions. There have also been numerous cases that have been treated with iron gluconate solution. These cases were most commonly associated with anemia resulting from an infectious process (blood stream, malaria). Iron gluconate is more widely available and is more expensive.\n" - Anonymous Online Contributor

Unverified Answer

How many people get anemia, hemolytic a year in the United States?

"About 13,000 people are diagnosed with anemia and 1,800 of those are diagnosed with hemolytic anemia annually. About 25 million adult Americans have one or more anemia-related blood disorders. These disorders affect about 13 million adults yearly." - Anonymous Online Contributor

Unverified Answer

What is anemia, hemolytic?

"Anemia, specifically, erythrocyte hemolytic anemia is the most common form of anemia, and thus, there is an increased risk of hemolysis at any given serum iron level. Hgb is the only parameter helpful to monitor anemia and determine severity." - Anonymous Online Contributor

Unverified Answer

Can anemia, hemolytic be cured?

"Anemia and hemolytic is curable. The treatment is not surgical but a therapy of anemia and control of hemolysis of the blood circulation system. Anemia is the result of various factors the only one being hemoglobin." - Anonymous Online Contributor

Unverified Answer

What are the signs of anemia, hemolytic?

"In this series, signs of abnormal hemoglobin levels (Hgb < 11 pg/L) in children with leukemia were present in 25% of patients. This finding indicates that the Hgb level should be monitored in both children and adults with leukemia. The prevalence of abnormal Hgb in those of all ages in this series supports the recommendation that the Hgb level should be routinely checked in all children with acute myeloid leukemia to ensure early detection and treatment." - Anonymous Online Contributor

Unverified Answer

What causes anemia, hemolytic?

"Hemolytic causes are a common source of anemia in patients treated for hemostatic diseases. The most frequent cause of hemolytic anemia in patients treated with oral anticoagulants is anemia induced as a complication of anti-phospholipid or anti-glycoprotein IIb/IIIa antibody-associated hemolytic disorders." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets anemia, hemolytic?

"Almost one in four patients are diagnosed at the time of a diagnosis of anemia, hemolytic. While this is not surprising, the incidence of the disease depends upon the population being studied. Most studies show a high incidence of iron deficiency in this population. The age of anemia diagnosed usually is between the ages of 20 to 60 years." - Anonymous Online Contributor

Unverified Answer

Does rilzabrutinib improve quality of life for those with anemia, hemolytic?

"In a recent study, findings reveals a positive impact of RILZB on HRQoL in AE in clinical practice. We propose RILZB as a potential treatment of anemia." - Anonymous Online Contributor

Unverified Answer

What is the latest research for anemia, hemolytic?

"A number of causes of anemia must be evaluated: dietary intake, environmental contaminants, infectious or malignant involvement of the gastrointestinal tract. Hemolysis must always be considered in the differential diagnosis of anemia and its causes. Hemag/oinemia is an infrequently reported disease entity but is best known as a complication of sickle-cell anemia. More than half of all hemolytic anemias are not well defined. Further investigation is needed to refine our understanding of hemolytic anemias." - Anonymous Online Contributor

Unverified Answer

Does anemia, hemolytic run in families?

"Recent findings confirms the existence of a high familial risk of anemia and/or acute hemolytic diseases in this population, thus suggesting this disease to be genetically regulated. The observation of the presence of the HPA-1b allele suggests the existence of a predisposing factor. The data obtained from this study may contribute to a better understanding of the heterogeneity of the condition." - Anonymous Online Contributor

Unverified Answer

How does rilzabrutinib work?

"Rilzabrutinib exerts antitumor effects against both HSPC and LPD, although the mechanism is still unclear. Combination treatment with rilzabrutinib may be an effective strategy to treat refractory LPD." - Anonymous Online Contributor

Unverified Answer

What is rilzabrutinib?

"Rilzabrutinib is a novel, oral, small molecule, kinase inhibitor specifically inhibiting Janus kinases (JAK) 1 and 2, with high selectivity and potency against other tyrosine kinases, including erythropoietin receptor (EPO-R) and insulin receptor (IR). It has strong anti-proliferative activity and inhibits erythropoiesis [increase in hemoglobin concentration] in vitro and in rodent models as well as in clinical trials. Moreover, rilzabrutinib is efficacious and well tolerated in patients with chronic erythropoietic failure receiving regular rHuEPO." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
See if you qualify for this trial
Get access to this novel treatment for Warm Autoimmune Hemolytic Anemia (wAIHA) by sharing your contact details with the study coordinator.