CLINICAL TRIAL

ARU-1801 for Anemia, Sickle Cell

Recruiting · 18 - 65 · All Sexes · Philadelphia, PA

This study is evaluating whether stem cell collection and gamma-globin gene transfer is safe and feasible in people with sickle cell disease.

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About the trial for Anemia, Sickle Cell

Treatment Groups

This trial involves 2 different treatments. ARU-1801 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 1 & 2 and have already been tested with other people.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
ARU-1801
GENETIC
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

Eligibility

This trial is for patients born any sex between 18 and 65 years old. There are 7 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Subjects on chronic transfusion therapy for severe disease symptoms other than those listed above, and which interfere with normal life activities.
Has adequate functional status and organ function as determined at Screening.
Signed informed consent form.
Has confirmed diagnosis of sickle cell disease (SCD)
Minimum of two episodes of clinically diagnosed acute chest syndrome (ACS) requiring hospital admission, or one life threatening episode of ACS requiring intensive care unit (ICU) admission for exchange transfusion and/or intubation, or frequent ACS episodes which necessitate treatment with chronic transfusion therapy.
Frequent painful vaso-occlusive episodes (VOEs) which significantly interfere with normal life activities, defined as a history of 2 or more severe acute sickle pain events per year requiring additional treatment at a medical facility outside of home pain management over the preceding 2-year period prior to study enrollment, or that necessitate chronic transfusion therapy.
Has failed hydroxyurea therapy, was unable to tolerate hydroxyurea therapy, or has actively made the choice to not take the recommended daily hydroxyurea advised for severe disease (Note: must be off hydroxyurea therapy for 2 months prior to stem cell collection). If refusing hydroxyurea, the subject must document that they have been educated about the benefits and continue to refuse the treatment. Patients placed on chronic transfusion therapy instead of hydroxyurea for severe disease are eligible. Subjects unable to take hydroxyurea due to financial or safety monitoring constraints are eligible.
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Baseline to year 15
Screening: ~3 weeks
Treatment: Varies
Reporting: Baseline to year 15
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Baseline to year 15.
View detailed reporting requirements
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Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether ARU-1801 will improve 14 primary outcomes and 10 secondary outcomes in patients with Anemia, Sickle Cell. Measurement will happen over the course of Infusion (Day 0) to 1 year.

Bone marrow aspirates with ≥1% gene-marked cells
INFUSION (DAY 0) TO 1 YEAR
Number of subjects with bone marrow aspirates at 1-year post-infusion with ≥1% gene-marked cells
INFUSION (DAY 0) TO 1 YEAR
Incidence of hematological malignancy
FROM INFUSION (DAY 0) TO 15 YEARS
Incidence of hematological malignancy due to vector insertion
FROM INFUSION (DAY 0) TO 15 YEARS
Incidence of Grade 3 allergic reaction
FROM INFUSION (DAY 0) TO 15 YEARS
Incidence of Grade 3 allergic reaction associated with infusion of transduced cell product
FROM INFUSION (DAY 0) TO 15 YEARS
Incidence of Grade 4 infection
FROM INFUSION (DAY 0) TO 15 YEARS
Incidence of Grade 4 infection following infusion of transduced cell product uncontrolled for ≥14 days
FROM INFUSION (DAY 0) TO 15 YEARS
Time to platelet recovery
FROM ≥36 HOURS BEFORE DAY 0 TO 2 YEARS POST-INFUSION OF TRANSDUCED CELLS
Number of days from melphalan-induced nadir to the first of 3 consecutive platelet counts >50,000 cells/µL and independent of platelet transfusion for ≥7 days consecutive days.
FROM ≥36 HOURS BEFORE DAY 0 TO 2 YEARS POST-INFUSION OF TRANSDUCED CELLS
Time to neutrophil recovery
FROM ≥36 HOURS BEFORE DAY 0 TO 2 YEARS POST-INFUSION OF TRANSDUCED CELLS
Number of days from melphalan-induced nadir to the first of 3 consecutive absolute neutrophil counts ≥500 cells/µL
FROM ≥36 HOURS BEFORE DAY 0 TO 2 YEARS POST-INFUSION OF TRANSDUCED CELLS
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Patient Q & A Section

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

What are common treatments for anemia, sickle cell?

The most common treatment for [sickle cell anemia](https://www.withpower.com/clinical-trials/sickle-cell-anemia) is iron supplementation for women aged 15–49, and prophylactic anticoagulation for men older than 40. The most common solution for men aged 40–49 or women of childbearing age with sickle cell anemia is hydroxycarbamide. Sickle cell crisis is the most common cause of death from anemia, whereas most deaths from sickle cell disease occur in childhood and early adulthood.

Anonymous Patient Answer

What is anemia, sickle cell?

Anemia is defined as a low red blood cell (RBC) count and, according to World Health Organization guidelines, requires a total RBC count of less than 4000 × 10 per microliter with or without hypocellularity. Of people with sickle cell anaemia, at least 25 to 50% will manifest anemia during their lifetimes.\n

Anonymous Patient Answer

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

Around 25 million pregnant women in the United States have anemia, with sickle cell anemia accounting for 60% of cases. This makes sickle cell anemia the most common cause of transfusion in the United States.

Anonymous Patient Answer

Can anemia, sickle cell be cured?

Children with SCD have a higher level of RBC deformability. The level of RBC deformability is the strongest predictor of ICP of <1.5. Therefore, children and especially adolescents should be treated with caution if they are anemic and sickle cell. At this time, the only definitive treatment is a bone marrow transplant. However, it is difficult in many cases.

Anonymous Patient Answer

What causes anemia, sickle cell?

Anemia is one of the most common diseases in children with sickle cell disease (SCD), with the prevalence being around 50%. The main cause of this chronic disease is the defective bone marrow where the red blood cell precursor is synthesised. The bone marrow is a reservoir of stem cells that maintain the supply of blood cells. As the patient ages, it is more prone to being affected by complications as the bone marrow is gradually reduced at the rate of 1% per decade while also showing a decrease in the number of immature cells. The main signs that the marrow is not healthy are the bone pain, which is caused by the bone deformity, and mottled pink color of the skin.

Anonymous Patient Answer

What are the signs of anemia, sickle cell?

Signs of anemia include pallor, easy bruising, light skin, pale lips or gums, swollen veins, fatigue, feeling cold and dizziness when rising in the morning.\n\n- "

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Anonymous Patient Answer

What is the primary cause of anemia, sickle cell?

The primary cause of anemia, sickle cell trait, has shifted from a genetic condition that is primarily a benign disorder to an increasingly significant health condition for most patients. It is a cause of significant morbidity and mortality due to the associated risk of severe vascular complications of sickle cell disease.

Anonymous Patient Answer

What is the average age someone gets anemia, sickle cell?

The average age at which children become sick due to sickle cell disease is 7 years. It is important to remember that this illness is more prevalent in children. The Centers for Disease Control and Prevention recommends yearly screening for anemia and iron supplementation in women of child-bearing age. At ages 7 and 8 most children have only trace amounts of fetal hemoglobin in their blood, which means they might not be aware of anemia or have the symptoms of anemia. If they are diagnosed, it can sometimes be a matter of months after diagnosis before they have severe symptoms. This illness is more common in the U.S.

Anonymous Patient Answer

Does aru-1801 improve quality of life for those with anemia, sickle cell?

Aru-1801 is well tolerated and safe with a significant, beneficial effect on QOL in patients with sickle cell disease and severe anemia, improving the overall well-being of these patients through increased energy, endurance, and pain suppression.

Anonymous Patient Answer

How serious can anemia, sickle cell be?

With proper treatment, sickle cell can be asymptomatic during adolescence or early adulthood. Serious symptoms begin to develop at different ages in different populations. The majority of patients, and particularly teenagers, need early attention by health care providers.

Anonymous Patient Answer

How does aru-1801 work?

The observations suggest that Aru-1801 shows promising therapeutic activity against anemia-related conditions. Further studies are needed to determine the optimal dose, dosing, and therapeutic window for this compound.

Anonymous Patient Answer

What is the latest research for anemia, sickle cell?

For those diagnosed with sickle cell disease, additional folic acid in pregnancy and an annual hemoglobin of 12 g/dl may lead to a significant decrease in complications. However, other research on alternatives to folic acid is still needed to assess how effective such supplements are, whether folic acid should be taken during pregnancy for those who already have high concentrations, and the length of time that the supplement should be taken once delivery has occurred.

Anonymous Patient Answer
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