RVU120(SEL120) for Preleukemia

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Sylvester Comprehensive Cancer Center, University of Miami Hospital and Clinics, Miami, FL
Preleukemia+7 More
RVU120(SEL120) - Drug
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a drug may help treat leukemia.

See full description

Eligible Conditions

  • Preleukemia
  • Acute Myeloid Leukemia (AML)
  • High-risk Myelodysplastic Syndrome (MDS)

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Compared to trials

Study Objectives

This trial is evaluating whether RVU120(SEL120) will improve 2 primary outcomes, 5 secondary outcomes, and 4 other outcomes in patients with Preleukemia. Measurement will happen over the course of Up to 18 months.

Up to 18 months
AML surface markers
Anti-leukemic activity
Genetic profile changes in AML blasts
Incidence of Adverse Events (Safety and Tolerability)
Phosphorylated protein levels in AML blasts
Recommended dose (RD)
The Area Under the Curve (AUC)
The Maximum Observed Concentration (C[max])
The Terminal Half-life (t[1/2])
The Volume of Distribution at Steady State (Vss)
Transcriptomic profile changes in AML blasts

Trial Safety

Safety Estimate

1 of 3

Compared to trials

Trial Design

1 Treatment Group

RVU120(SEL120)
1 of 1
Experimental Treatment

This trial requires 54 total participants across 1 different treatment group

This trial involves a single treatment. RVU120(SEL120) 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.

RVU120(SEL120)
Drug
The first part of the study consists of dose-escalation cohorts where patients will receive ascending doses of RVU120(SEL120) to determine the recommended dose (RD) for further clinical development. The second part of the study is an enrichment cohort where additional 6 to 20 patients will be treated with RVU120(SEL120) to support the evaluation of the RD.

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 18 months
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 18 months for reporting.

Closest Location

Sylvester Comprehensive Cancer Center, University of Miami Hospital and Clinics - Miami, FL

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. You must have received 1 prior treatment for Preleukemia or one of the other 7 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:
Serum albumin ≥ 30g/L (3.0g/dL)
Written informed consent provided prior to any study-related procedure.
Age ≥18 years.
AML diagnosis according to the 2016 World Health Organisation (WHO) classification (Arber et al. 2016) with relapsed or refractory disease who have received no more than 3 prior lines of therapy and with no available therapy who have exhausted the applicable standard of care; or Myelodysplastic Syndrome (MDS) diagnosis according to the 2016 WHO classification (Arber et al. 2016) with high-risk disease per the Revised International Prognostic Scoring System and with relapsed or refractory disease, who have received no more than 3 prior lines of therapy and with no available therapy who have exhausted the applicable standard of care.
Eastern Cooperative Oncology Group (ECOG) performance score of 0-2.
Patients must have been off anti-cancer treatment for 2 weeks or 5 half-lives, whichever is longer. Note: Hydroxyurea is exempted if used to reduce total white blood cell (WBC) count (see below); radiation must have been completed at least 4 weeks prior to first dose of study drug.
Patients must have recovered from the toxic effects of previous treatments to at least Grade 1, for neurotoxicity or alopecia to Grade 2.
Peripheral white blood cell (WBC) count, no upper limit at Screening, but must be <10x10^9/L on Day 1 prior to first dose of study drug. Note: Patients with excessive blasts may be treated with hydroxyurea until 2 days prior to first dose of study drug to reduce WBC;
Platelet count >10,000/µL; Platelet transfusion prior to first dose is permitted
Normal coagulation (elevated INR, prothrombin time or APTT <1.3 x ULN acceptable);

Patient Q&A Section

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

"Around 80% of adults have at least one syndrome each year. Around 30 million adults in the United States will develop at least one syndrome a year. This makes up 13.5% of the US adult population. There is a sex bias in the prevalence of a number of syndromes. A higher prevalence is seen in people over the age of 65. A higher proportion of men have diabetes than women (17.0% versus 10.1%)." - Anonymous Online Contributor

Unverified Answer

What is syndrome?

"Symptom syndromes are terms used to describe a collection of symptoms that do not fall to the criteria of a single disease. In this case there are no obvious and well-defined criteria for the "symptom syndrome" of COPD." - Anonymous Online Contributor

Unverified Answer

What are the signs of syndrome?

"There are often multiple signs and symptoms of the syndrome, including systemic involvement in more than one system, and often a combination of the disorders mentioned in this article. Some signs and symptoms of some of the syndromes are listed above.\n\nSigns of the disorder are usually nonspecific and often the hallmark of the syndrome is the lack of a specific diagnosis.\n\nThe syndrome of systemic lupus erythematosus can be difficult to diagnose as many of the signs may be nonspecific." - Anonymous Online Contributor

Unverified Answer

Can syndrome be cured?

"The findings reported here indicate that when all diagnostic criteria are met a significant proportion of patients with syndromic ASD, a "typical ASD syndrome" or a mixed ASD disorder can be cured with treatment. A subset of patients could achieve and maintain general health and quality of life after being successfully treated." - Anonymous Online Contributor

Unverified Answer

What are common treatments for syndrome?

"Treatment is often centered on disease type, progression and severity. A wide spectrum of interventions is possible for common syndromes such as neurological and autoimmune diseases, psychiatric disorders, cardiovascular and sleep apnea." - Anonymous Online Contributor

Unverified Answer

What causes syndrome?

"Syndromes are a relatively recent phenomenon. At one time they included only disorders with defined pathologies such as multiple myeloma, cerebral palsy or Huntington's disease. Today they typically include a variety of disorders associated with different genetic or metabolic abnormalities. Syndromes are a relatively recent phenomenon. At one time they included only disorders with defined pathologies such as multiple myeloma, cerebral palsy or Huntington's disease. Today they typically include a variety of disorders associated with different genetic or metabolic abnormalities. Syndromes are no more than a way to classify and label a number of disorders, although they often include a 'definite' diagnosis of a single genetic anomaly." - Anonymous Online Contributor

Unverified Answer

Is rvu120(sel120) typically used in combination with any other treatments?

"In this population-based survey of treatment usage, Rvu120(sel120) typically was not used in combination with other drugs. Data from a recent study of this survey may inform future trial design for children with MELAS." - Anonymous Online Contributor

Unverified Answer

How serious can syndrome be?

"Symptom severity varies considerably across patients and can be underestimated by clinicians or reported on by patients. Patients suffering from syndrome will generally experience much larger symptoms than patients receiving standard care in terms of physical and psychological distress, symptom severity and interference with quality of life." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of syndrome?

"It is still not known what is the primary cause of syndrome. Our cases are probably not due to any one specific cause and instead represent cases of nonadherence to treatment. The association with autoimmune disease emphasizes the importance of early treatment in the absence of autoimmune disorders to prevent development of progressive disease." - Anonymous Online Contributor

Unverified Answer

Who should consider clinical trials for syndrome?

"The patient with a specific syndrome is usually the preferred group for clinical trials. Patients who do not fit in any of the groups listed here are either too few to justify clinical trials or they are just too prevalent to justify clinical trials. Clinical trials specifically tailored to patients with a syndrome can help to show statistically significant improvement in symptoms, and it may shorten the time required for the study to answer these questions. Clinical trials that are not tailored to the specific syndrome need to be interpreted in a more circumspect manner. If the clinical trial is performed on all of the patients regardless of the condition, it takes longer, and there are more chances for the results to be meaningless." - Anonymous Online Contributor

Unverified Answer

What are the chances of developing syndrome?

"Although the presence or absence of the syndrome of multiple system atrophy is influenced by many different factors such as age, age of onset, sex, genetic factors and environmental factors, none of this influences the risk of developing syndrome of multiple system atrophy, meaning that the syndrome is genetically influenced." - Anonymous Online Contributor

Unverified Answer

Has rvu120(sel120) proven to be more effective than a placebo?

"A small clinical study shows that rvu120(sel120) is significantly more effective than a placebo in the treatment of symptomatic hypercalcinosis. The authors conclude that rvu120(sel120) is effective in alleviating hypercalcemia, improving the quality of life of patients, and in some cases halting the progressive enlargement of calcium deposits in the body." - 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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