Melphalan for Amyloidosis

Phase-Based Estimates
1
Effectiveness
1
Safety
Memorial Sloan Kettering Cancer Center, New York, NY
Amyloidosis+2 More
Melphalan - Device
Eligibility
18+
All Sexes
Eligible conditions
Amyloidosis

Study Summary

This study is evaluating whether a new formulation of melphalan can be used to treat multiple myeloma and AL amyloidosis.

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Eligible Conditions

  • Amyloidosis
  • Multiple Myeloma
  • Neoplasms, Plasma Cell

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Melphalan will improve 1 primary outcome in patients with Amyloidosis. Measurement will happen over the course of 1 day.

1 day
Overall Response Rate (ORR)

Trial Safety

Safety Estimate

1 of 3

Trial Design

2 Treatment Groups

Control
Melphalan

This trial requires 46 total participants across 2 different treatment groups

This trial involves 2 different treatments. 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 1 and are in the first stage of evaluation with people.

MelphalanTest Dose CE Melphalan 10mg/m2 with PK studies Day -12 to Day-3, CE Melphalan Dose of Target AUC 13 mg/L/h infused with PK studies Day -2, 3-10 x 10^6 CD 34+ cells/kg reinfused Day 0, Pegfilgrastim 6mg injection Day +1
ControlNo treatment in the control group
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Melphalan
2008
Completed Phase 3
~2250
Melphalan
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 1 day
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 1 day for reporting.

Closest Location

Memorial Sloan Kettering Cancer Center - New York, NY

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. You must have received newly diagnosed for Amyloidosis or one of the other 2 conditions 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's AST, ALT and alkaline phosphatase are all less than three times the upper limit of what is considered normal in a laboratory setting. show original
Patients must have a minimum of 3 million CD34+ cells per kilogram of frozen material. show original
This patient has a creatinine clearance level of ≥40 ml/min, which is considered normal. show original
The patient's left ventricular ejection fraction is ≥45% as determined by MUGA or rest ECHO. show original
Age between 18 and 75 years old. show original
Patients who have responded favorably to previous induction or salvage chemotherapy treatments (i.e show original
Patients who are receiving high-dose melphalan and AHCT as part of salvage therapy require a minor response to their last line of therapy in order to document chemosensitive disease show original
People with AL amyloidosis, who may have been recently diagnosed or who have been treated in the past, may need to take different steps to stay healthy. show original
After reviewing the histologic and serologic findings, it has been confirmed that the patient has multiple myeloma or AL amyloidosis show original
This means that the serum bilirubin level is 2.0 mg/dl or less. show original

Patient Q&A Section

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

Can amyloidosis be cured?

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In severe cases of amyloidosis, there is still a small but definite survival benefit for early diagnosis. However, in milder or early stages of the disease, there is little to no survival benefit.

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What is amyloidosis?

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Amyloidosis is a group of blood and bone marrow disorders in which abnormal, abnormal blood-derived materials accumulate in various tissues and organs of the human body. A protein called amyloid is involved in causing amyloidosis.\n

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

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The American Cancer Society estimates there will be 910 new cases of AL amyloidosis, 910 cases of LG-AL amyloidosis, 630 cases of AG-AL amyloidosis and 15,090 new cases of other AL amyloidosis and 4,100 new cases of other LG-AL amyloidosis. In 2017, 26,000 Americans are expected to have AL amyloidosis with a prevalence of 7 per 100,000. For LG-AL, the average age in 2017 is around 70. For AG-AL it is around 50. For other amyloidosis it is around 65.

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What are common treatments for amyloidosis?

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About one third of all patients die from amyloidosis and one third succumb to amyloidosis related illnesses; 20 times out of 25 is usually due to kidney failure. Treatment of amyloidosis has long been controversial, for various reasons including lack of strong scientific evidence that amyloidosis is the result of an underlying disease and that amyloidosis treatment is effective in preventing progression of the disease and preventing mortality, as well as being harmful. More aggressive treatment that slows the progression of symptoms will improve outcomes in patients with early-stage amyloidosis. Treatment options that improve quality of life or prolong life in patients with late-stage amyloidosis are less clear.

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What are the signs of amyloidosis?

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There is a range of non-specific signs and symptoms of amyloidosis. These signs may include fatigue, unexplained weight loss and anemia. In addition, amyloidosis has many clinical manifestations of which heart palpitations are a prominent one. A specific test for the disease can be done when there are no other specific signs of the syndrome.

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What causes amyloidosis?

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Amyloidosis is caused by a primary systemic amyloid precursor protein disease, where both amyloid A and amyloid B are involved in the etiology of the disease. Amyloid is also found, but less commonly, in renal disease in general. These conditions are related, which is reflected in the fact that amyloidosis can cause some renal manifestations of the disease.

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Is melphalan safe for people?

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Melphalan is a highly effective treatment for amyloidosis in most countries. However, for people who have kidney failure, this substance should be avoided, as there is evidence that it may increase risk of kidney failure. In particular, the use of any chemotherapeutic agent, with or without haematological impairment, including melphalan for treatment of amyloidosis may increase risk of kidney failure.

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What is melphalan?

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Melphalan is a drug that can produce skin lesions that look similar to myeloma. Myeloma patients have low levels of serum protein, low levels of serum albumin, low calcium levels, high levels of Bence Jones protein and low levels of other proteins. In myeloma patients, high levels of serum calcium, increased albumin and increased total protein level in serum seem to exist, which is the indication of the use of melphalan among myeloma patients in the treatment of low body weight. Also, this method could help treat amyloidosis patients by improving nutritional status for patients. But the result will be the same as Bence Jones protein.

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Who should consider clinical trials for amyloidosis?

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Clinicians should be aware of the limitations and pitfalls of clinical trial evidence for systemic therapies for amyloidosis beyond current treatments. Importantly, patients with advanced disease or in whom clinical trials evaluating targeted therapies may help direct therapy warrant careful consideration and a rigorous discussion of the potential clinical benefits and the potential risks of such trials in patients with advanced amyloidosis.

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What is the primary cause of amyloidosis?

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Patients with AA amyloidosis tend to come from a family of end stage renal disease regardless of renal function and are usually of a young age at the time of diagnosis. The most frequent type of amyloid protein is a monoclonal form of plasma cell dyscrasias. Therefore, in patients of AA amyloidosis, renal failure is due to glomerular amyloid deposition rather than to amyloid accumulation in the kidney itself. In a subset of patients with renal failure of unknown origin, it is found to be secondary to amyloid deposition in the kidneys.

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Is melphalan typically used in combination with any other treatments?

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Melphalan may be particularly useful in combination with other, more novel agents for aggressive amyloidoses of the AL and AL wild-type with a high amyloid constituent: CHOP, in the case of amyloidosis with AL wild-type with a low amyloid content (sporadic, or AL with no detectable amyloid in routine pathology). Such combinations may be particularly useful for patients with no detectable amyloid components, although these patients carry a real risk of relapse and should be treated accordingly. This may be particularly urgent for the treatment of AL amyloidosis with a high amyloid constituent.

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What are the common side effects of melphalan?

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The most common side effects of melphalan are nausea/vomiting and vomiting. Skin reactions (e.g. erythema multiforme, local or generalized dermatitis, pruritus) and general weakness are also common. Hemorrhagic complications (due to blood transfusion) are rarely seen.

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