Alemtuzumab for Sickle Cell Disease (SCD)

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Children's Healthcare of Altanta, Atlanta, GA
Sickle Cell Disease (SCD)+1 More
Alemtuzumab - Drug
Eligibility
< 18
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a bone marrow transplant may help treat sickle cell disease.

See full description

Eligible Conditions

  • Sickle Cell Disease (SCD)

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Alemtuzumab will improve 1 primary outcome and 15 secondary outcomes in patients with Sickle Cell Disease (SCD). Measurement will happen over the course of Day 42.

Day 100
Early Onset Acute GVHD
Day 180
Cytomegalovirus (CMV) Viremia
Day 42
Regimen-Related Toxicity
Up to Year 5
CMV Invasive Disease
Chronic GVHD
Graft Rejection
Late Onset Acute GVHD
Neutrophil Recovery
Number of Neurological Complications
Other Infections
Overall Survival
Platelet Recovery
Post-transplant Lymphoproliferative Disorder
Rejection-free Survival
Year 2
Immune Suppression-free, Rejection-free Survival
Sustained Donor Engraftment

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Trial Design

1 Treatment Group

Reduced Intensity Conditioning with FAM
1 of 1
Experimental Treatment

This trial requires 58 total participants across 1 different treatment group

This trial involves a single treatment. Alemtuzumab 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.

Reduced Intensity Conditioning with FAMChildren with SCD will received reduced intensity conditioning with fludarabine, alemtuzumab and melphalan (FAM) during HSCT with a HLA matched sibling donor
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Melphalan
FDA approved
Fludarabine
FDA approved
Alemtuzumab
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to year 5
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to year 5 for reporting.

Who is running the study

Principal Investigator
J. H.
Prof. John Horan, Associate Professor
Emory University

Closest Location

Children's Healthcare of Altanta - Atlanta, GA

Eligibility Criteria

This trial is for patients born any sex aged 18 and younger. You must have received 1 prior treatment for Sickle Cell Disease (SCD) or the other condition listed above. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
The patient has significant vasculopathy, as indicated by an MRA that shows greater than 50% stenosis of more than two arterial segments, or a complete occlusion of any single arterial segment. show original
Anyone who has had 3 episodes of acute chest syndrome and 3 episodes of vaso-occlusive pain in a year for 3 years straight is considered to have chronic sickle cell disease show original
Patients must have an HLA identical sibling donor who is less than thirteen years old show original
A previous clinical stroke is a neurological deficit that lasts longer than 24 hours and is accompanied by a new finding on a head CT scan or brain MRI/MRA. show original
Progressive damage to the brain caused by a lack of blood flow, as shown by MRI scans that reveal a series of lesions (at least two lesions that are clearly visible and temporally separate, each measuring at least 3 mm on the most recent scan) or the enlargement of a single lesion, initially measuring at least 3 mm show original
The person has had at least three episodes of acute chest syndrome in their lifetime that have required blood transfusions. show original
Patients must have a sickle hemoglobinopathy and be at least 2 years old and less than 13 years old. show original
is an indication for further evaluation with MRI If an individual's TCD testing results show abnormal velocities in any single vessel, then it is an indication that they should undergo further evaluation with Magnetic Resonance Imaging (MRI). show original
Having at least 3 vaso-occlusive episodes in the past 2 years that last for 4 hours or more and require hospitalization or outpatient treatment with parenteral opioids show original
Patients with SCD must meet specific criteria in order to be considered symptomatic show original

Patient Q&A Section

What causes anemia, sickle cell?

"In this group of children, anemia was a common finding which was caused by a variety of hematologic agents. Recent findings further support the concept that anemia is a multifactor etiology which is best investigated in terms of what causes the anemia." - Anonymous Online Contributor

Unverified Answer

What are the signs of anemia, sickle cell?

"A combination of signs and symptoms of anemia, particularly fatigue, may be evident in children who are at risk of developing anemia. This condition is caused by the sickle cell gene." - Anonymous Online Contributor

Unverified Answer

What is anemia, sickle cell?

"Anemia, sickle cell disorder, affects at least 5% of African-American patients and causes profound effects on the life of those affected. The treatment of the anemia in individuals with sickle cell should be emphasized." - Anonymous Online Contributor

Unverified Answer

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

"In 2013, 5.3 million adults aged 19 and older had [sickle cell anemia](https://www.withpower.com/clinical-trials/sickle-cell-anemia). In the United States, anemia was present in 8.6% of children and adolescents ages 5 to 19 years. In the United States, 6.5% of children and adolescents ages 5 to 19 years are sickle cell trait carriers." - Anonymous Online Contributor

Unverified Answer

What are common treatments for anemia, sickle cell?

"In addition to supportive therapies, specific therapies like packed red blood cell transfusions and blood transfusions can be necessary when anemia causes severe disability or symptoms." - Anonymous Online Contributor

Unverified Answer

Can anemia, sickle cell be cured?

"In terms of cure rates, HBA > 9g/dL and HbA > 12g/dL may be ideal to seek for patients with SCD and sickle cell anemia." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in alemtuzumab for therapeutic use?

"Although the data presented during this Symposium show that the use of alemtuzumab is feasible and safe in clinical trials, our knowledge of what alemtuzumab does or looks like and how it affects the body remains limited. More importantly, for clinical use, we have found that our studies have yet to demonstrate benefit for patients with the refractory form of acute myelogenous leukemia after treatment with alemtuzumab. We also have found that while alemtuzumab has a very favorable safety profile, patients with this disease should follow the current recommendations for use of alemtuzumab with caution." - Anonymous Online Contributor

Unverified Answer

Does anemia, sickle cell run in families?

"Results from a recent paper support the concept that anemia and its associated diseases affect a complex interplay among several genetic, behavioral, and environmental factors that are likely to be strongly influenced by family socioeconomic exposures." - Anonymous Online Contributor

Unverified Answer

What is the latest research for anemia, sickle cell?

"The anemia of thalassemia and sickle cell disease is difficult to control. Patients with severe anemia must be given regular blood therapy to restore platelet counts and hemoglobin levels to normal. The current standard to treat sickle cell disease is-splenectomy. For patients with thalassemia-splenectomy should be deferred for 6 months after a regular course of blood transfusions. For patients who require regular transfusions, they should be treated with prophylactic antimicrobial prophylaxis. There is no cure for anemia." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of anemia, sickle cell?

"Sickle cell disease is mostly the result of genetic mutations and environmental insults. In the United States, one of the most common causes is the exposure of infants to sickle cell disease (Hb S). Other diseases affecting infants such as HIV infection and hepatitis B make the newborns even more vulnerable to sickle cell disease.\n" - Anonymous Online Contributor

Unverified Answer

What are the common side effects of alemtuzumab?

"We found that the most common side effects related to alemtuzumab treatment could mostly be sorted in infusion reactions such as pruritus, anaphylactoid reactions, asymeremia, and febrile reactions. In addition, we also found that most patients suffered from diarrhoea and pain. Furthermore, a decrease in hemoglobin levels was observed in some patients during treatment; thus, anaemia needs to be carefully monitored during adjuvant alemtuzumab therapy. In this regard, treatment interruptions should be considered." - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving alemtuzumab?

"The incidence of side effects such as the FDA-defined grade 2 and 3 AEs, the need to suspend a trial due to adverse events following the initial announcement of AEs, and the proportion of patients who stopped therapy due to these AEs were similar between alemtuzumab and Rituximab. Anecdotal evidence of other anti-IL-2 therapies that have led to greater AE reporting than alemtuzumab include infusions of alemtuzumab from the same batch or alemtuzumab from a different batch. Both sources of infusion experience similar AE reporting and are unlikely to be causative." - 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.
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