Melphalan for Anemia, Sickle Cell

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Hackensack University Medical Center, Hackensack, NJ
Anemia, Sickle Cell+1 More
Melphalan - Drug
Eligibility
< 65
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether stem cell transplantation can cure sickle cell disease.

See full description

Eligible Conditions

  • Anemia, Sickle Cell
  • Sickle Cell Disease (SCD)

Treatment Effectiveness

Effectiveness Estimate

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

Study Objectives

This trial is evaluating whether Melphalan will improve 1 primary outcome and 1 secondary outcome in patients with Anemia, Sickle Cell. Measurement will happen over the course of 1 year.

1 year
Graft Failure
2 years
Overall survival

Trial Safety

Safety Estimate

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

Trial Design

1 Treatment Group

Related donor
1 of 1
Experimental Treatment

This trial requires 26 total participants across 1 different treatment group

This trial involves a single treatment. Melphalan 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.

Related donorMatched sibling donors (9-10/10 marrow/PBSC or 5-6/6 UCB (single) with a total TNC dose of greater than 5 x 107/kg recipient weight), age 2-30 years after conditioning regimen Alemtuzumab , Fludarabine, and Melphalan. 1) Patients will receive a conditioning regimen composed of Alemtuzumab, Fludarabine, and Melphalan as detailed in the table below. Day Treatment -22 Alemtuzumab 3mg IV (test dose) -21 Alemtuzumab 10mg IV -20 Alemtuzumab 15mg IV -19 Alemtuzumab 20mg IV -8 Fludarabine 30mg/m2 IV -7 Fludarabine 30mg/m2 IV -6 Fludarabine 30mg/m2 IV -5 Fludarabine 30mg/m2 IV -4 Fludarabine 30mg/m2 IV -3 Melphalan 140mg/m2 IV -2 Rest Day -1 Rest Day 0 Stem Cell Infusion
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Melphalan
FDA approved
Alemtuzumab
FDA approved
Stem Cells
2002
N/A
~80
Fludarabine
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 2 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 2 years for reporting.

Closest Location

Hackensack University Medical Center - Hackensack, NJ

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Patient Eligibility
1) Matched sibling donors (9-10/10 marrow/PBSC or 5-6/6 UCB (single or double) with a total TNC dose of greater than 5 x 107/kg recipient weight)
Age 2-30
Hb SS, S-thal0, S-thal+, SC
Evidence of ongoing hemolysis: Hb<10, retic >5%, LDH > 500, TB>2
Karnofsky/Lansky score >50
LVSF>26% or LVEF>40%
DLCO >40% or O2 sat >85% for those patients that can't perform PFTs
GFR >70 and serum creatinine < 1.5 * ULN for age
ALT and AST < 5 x ULN, direct bilirubin <2 x ULN

Patient Q&A Section

What are the signs of anemia, sickle cell?

"The following are the main risk factors for sickle cell disease: maternal smoking, blood group c, erythropoietin resistance and homozygous deletion of the sickle cell gene of a family member. Children with sickle cell trait can be carriers of the disease.\n" - Anonymous Online Contributor

Unverified Answer

What are common treatments for anemia, sickle cell?

"At diagnosis, there is not a definitive regimen for anemia. With treatment, however, the most common treatment approaches are medications. While this does not represent treatment, it is supportive of other therapies. Some form of blood transfusion is always indicated if the patient has a low red cell mass (<60 mLm)." - Anonymous Online Contributor

Unverified Answer

Can anemia, sickle cell be cured?

"Not only is anemia highly prevalent and a major issue among the sickle cell population, but it also leads to significant morbidity and mortality. Anemia can be treated successfully with transfusions. Further, sickle cells can be rendered nonlethal with an investigational agent." - Anonymous Online Contributor

Unverified Answer

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

"around 1 million people have sickle cell anemia each year in the United States. There are significant disparities in the incidence of sickle cell anemia between black women in the US and black men." - Anonymous Online Contributor

Unverified Answer

What is anemia, sickle cell?

"Anemia is a condition characterized by a decreased number of red blood cells and a low percentage of immature red blood cells in circulation. Sickle cell anemia is an inherited disorder in which genes are mutated to create abnormally globin chains (hemoglobin). As a result, sickle cell hemoglobin can aggregate in the blood and block vessels. As individuals with sickle cell anemia have an increased risk of painful crises related to vasoocclusion it is important to identify sickle cell trait and confirm sickle cell mutation by diagnostic testing." - Anonymous Online Contributor

Unverified Answer

What causes anemia, sickle cell?

"Individuals with SCD may have an increased risk of developing anemia from exposure to insect bites (e.g., mosquitoes) or parasitically transmitted pathogens. The underlying mechanisms of sickle cell anemia are not well understood. One possibility is that sickling of red blood cells occurs due to a reduction in the number of hemoglobin molecules per cell rather than an increase in the number of dysfunctional hemoglobin molecules." - Anonymous Online Contributor

Unverified Answer

What is the latest research for anemia, sickle cell?

"Currently, there are no curative drugs to stop progressive Anemia and Sickle Cell Disease (ASD). Treatment usually focuses on treating symptoms and complications of the disease such as pneumonia and other pulmonary emergencies, and relieving the pain and discomfort associated with sickle-cell crises during the summer." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of melphalan?

"Melphalan causes nausea- vomiting, hypo-pnoea, and electrolyte disturbances. These reactions were only observed in patients with a baseline creatinine clearance of less than 60 mL/min." - Anonymous Online Contributor

Unverified Answer

Has melphalan proven to be more effective than a placebo?

"Although there is no doubt that melphalan should be utilized in the treatment of SCD patients with severe ane-mias, it was found that there was an increased prevalence, duration, seriousness and mortality of secondary disorders among patients treated with melphalan." - Anonymous Online Contributor

Unverified Answer

Is melphalan safe for people?

"Results from a recent paper provides further support for melphalan's role following hematopoietic stem cell transplantation from HLA-mismatched donors, especially children aged 2-5 years, as a safe treatment for AML-M2s. However, caution should be used when administering melphalan to patients with Hb values of less than 80 g/dL, or those who have undergone allogeneic HCT and have received a concurrent autogenous bone marrow transplant." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in melphalan for therapeutic use?

"In the last decade the results of new melphalan schedules have been published. Many of these schedules offer an increased efficiency, with a favorable cost, however, none of them have received approval by the FDA. This is probably due to the fact that the melphalan is a complex molecule, having several toxic effects and also has a high potential to cause immunotoxicity, specially in children. For these reasons, melphalan has proven challenging to study." - Anonymous Online Contributor

Unverified Answer

What does melphalan usually treat?

"Melphalan in the treatment of patients who are HbSS is associated with a higher rate of relapse, thus, for these individuals, it is most effective to consider alternative options." - 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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