Mycophenolate mofetil (MMF) for Syndrome

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
National Institutes of Health Clinical Center, Bethesda, MD
Syndrome+2 More
Mycophenolate mofetil (MMF) - Drug
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a stem cell transplant can be used to treat VEXAS Syndrome.

See full description

Eligible Conditions

  • Syndrome
  • Immunodeficiencies
  • Hematopoietic Stem Cell Transplantation (SCT)

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

This trial is evaluating whether Mycophenolate mofetil (MMF) will improve 2 primary outcomes and 3 secondary outcomes in patients with Syndrome. Measurement will happen over the course of +1, +2 and +3 years post HSCT.

Year 2
Reversal of clinical phenotype of VEXAS
incidence of grade III-IV acute GVHD and moderate to severe chronic GVHD
Year 3
Overall survival and event free survival
Safety of allo HSCT
Year 1
Sustained donor engraftment

Trial Safety

Safety Progress

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

Trial Design

2 Treatment Groups

Arm A
1 of 2
Arm B
1 of 2
Experimental Treatment

This trial requires 37 total participants across 2 different treatment groups

This trial involves 2 different treatments. Mycophenolate Mofetil (MMF) is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Arm AReduced intensity regimen (Fludarabine, busulfan)+HSCT+GVHD prophylaxis
Arm BReduced intensity regimen (Fludarabine, low dose cyclophosphamide, 200cGY TBI, busulfan)+HSCT+GVHD prophylaxis
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Mycophenolate mofetil
FDA approved
Cyclophosphamide
FDA approved
Fludarabine
FDA approved
Busulfan
FDA approved
Allogeneic HSCT
2015
Completed Phase 2
~60
Sirolimus
FDA approved
Total Body Irradiation (TBI)
2016
Completed Phase 3
~1040

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: day +100 and +1 year post hsct
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly day +100 and +1 year post hsct for reporting.

Closest Location

National Institutes of Health Clinical Center - Bethesda, MD

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. You must have received 1 prior treatment for Syndrome 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:
Age >= 18-year-old and <= 75-year-old
Availability of an 8/8 or 7/8 HLA-matched related or unrelated donor, or a haploidentical related donor Karnofsky performance status of >= 40%
Pulmonary function tests: FEV1 and DLCO >30%
Ability of subject to understand and the willingness to sign a written informed consent document.
As therapeutic agents used in this trial may be harmful to a fetus, women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) at the study entry and for at least one-year post-allo HCT. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in the study, she should inform her treating physician immediately.
Non-disease related
Left ventricular ejection fraction > 35%, preferably by 2-D echocardiogram (ECHO) obtained within 60 days prior to treatment initiation.
Creatinine <= 2.0 mg/dl and creatinine clearance >= 30 ml/min;
Serum conjugated bilirubin < 3.0 mg/dl; serum ALT and AST <= 5 times upper limit of normal.
Willingness to remain in the NIH hospital or, if discharged, live within 2 hours drive from the NIH, for a minimum of 100 days after transplant or longer, if there are complications. If outpatient in the first 100 days after transplant, participant must commit to having an adult caregiver with them at all times.

Patient Q&A Section

What causes syndrome?

"The concept of syndromes is an important one and is crucial for understanding how different medical conditions can be associated. This article provides a definition of the term syndrome and discusses how the term relates to other medical conditions and approaches." - Anonymous Online Contributor

Unverified Answer

What are common treatments for syndrome?

"There is no standard treatment for this group of conditions. Specific treatment for each condition will vary depending on the specific presentation and the severity of the symptoms. In all cases, the goal of therapy is to treat symptoms that are not as part of the syndrome. The clinician will have to assess the individual needs of the person with the syndrome." - Anonymous Online Contributor

Unverified Answer

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

"The prevalence of syndrome is low in US population. However, it is not uncommon to see a patient in each day. We need to understand the cause, diagnosis and treatment of syndrome, and to discover the unknown genes that may influence the disease. Clinical trial is one strategy for finding a cure for syndrome." - Anonymous Online Contributor

Unverified Answer

What is syndrome?

"Syndromes are medical symptoms combined and united to form a specific syndrome or group of related syndromes. The diagnostic feature of a syndrome is the presence of an identifiable, consistent set of signs and symptoms that characterize the syndrome rather than the diagnosis of any particular disease. For example, most people with RHD have the specific condition, sickle cell disease, but only some people know they have the disease. The syndrome of RHD includes the condition of sickle cell disease, but also includes many other conditions, such as malaria, which may also result in the disorder of sickle cell disease." - Anonymous Online Contributor

Unverified Answer

What are the signs of syndrome?

"The criteria for the diagnosis of syndrome are quite vague. However we suggested that the characteristics of the disorder may not always be seen and that there are many signs of syndrome, which can explain the presentation of this syndrome." - Anonymous Online Contributor

Unverified Answer

Can syndrome be cured?

"Symptom resolution for some individuals may be an indication a syndrome may be partially, but not completely, curable. Patients who have incomplete, fluctuating symptom resolution in response to conventional therapy for the disease may develop an intolerance to the drugs or may suffer adverse reactions to the drug(s). Symptom improvement in response to conventional treatment of the person's condition does not guarantee, by itself, a cure for the person's syndrome." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of syndrome?

"Syndromes are best understood under the paradigm that we need to understand the underlying etiological mechanisms of a syndrome in order to be able to describe it, understand the clinical consequences of the syndrome, and to develop appropriate treatment strategies. In the case of the syndrome of late-onset depression, the underlying mechanism of the depression could be related to problems in sleep, appetite, or the hypothalamic-pituitary-adrenal axis. These may also be part of the mechanism of bipolar disorder. In contrast, it seems that major depression is related to changes in the neurotransmitter system." - Anonymous Online Contributor

Unverified Answer

Is sirolimus safe for people?

"This review supports the recent notion that a patient-specific individualised plan should be developed in the context of the patient's medical circumstances. The use of sirolimus may benefit patients with an increased risk of toxicity (e.g. elderly, transplant recipients, diabetic) in a population where the potential for increased efficacy is equally important." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets syndrome?

"We calculated the age that a disorder becomes symptomatic to be 5 years. In a recent study, findings provide a benchmark for disease-onset in children and adolescents. We hypothesize an association between disease-onset and a gene by environment interaction." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of sirolimus?

"Mild GI distress and dermatologic toxicity occurred in most patients receiving sirolimus. Skin reactions, particularly erythema and itching, were the most common, and were more frequent with higher blood levels of sirolimus. Sirolimus was also associated with exacerbation of existing diabetes." - Anonymous Online Contributor

Unverified Answer

Is sirolimus typically used in combination with any other treatments?

"Combining mTORin with other treatment modalities seems to be more effective than mTORin alone. Although other drugs may be used alternatively by some patients, it may be beneficial to consider mTORin in combination with other treatments. The data from this review may be of relevance in the discussion about which treatments might be offered to patients with SLE." - Anonymous Online Contributor

Unverified Answer

Has sirolimus proven to be more effective than a placebo?

"After 4 years of sirolimus the most common adverse events (in more than 1/2 of patients) were leukopenia (<35 x 10(6) leukocytes per microL) and/or thrombocytopenia (<100 x 10(6) platelets per microL). The most serious adverse events (in less than 1/2 of patients) were: pneumonitis, opportunistic infection/bacterial (in 0.6% patients), leukomorphia, myocardial infarction (MI) and/or congestive heart failure (CHF) (0.4%)." - 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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