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Anti-metabolites
Sirolimus + Azacitidine for Myelodysplastic Syndrome
Phase 2
Waitlist Available
Led By Neil Palmisiano, MD
Research Sponsored by Sidney Kimmel Cancer Center at Thomas Jefferson University
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
AML (Arm B): Relapsed/refractory/unable to tolerate conventional chemotherapy
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
Study Summary
This trialstudies how well two drugs, sirolimus and azacitidine, work in treating high-risk myelodysplastic syndrome and recurrent acute myeloid leukemia. The drugs work in different ways to stop cancer cell growth.
Who is the study for?
Adults with high-risk myelodysplastic syndrome or recurrent acute myeloid leukemia not suitable for intensive chemo. Must be over 18, have a life expectancy of at least 4 weeks, able to take oral meds, and have acceptable organ function. Excludes those with severe diseases, active infections, HIV/AIDS, pregnant/breastfeeding women, or on certain drugs.Check my eligibility
What is being tested?
The trial is testing the effectiveness of sirolimus (an inhibitor of cell growth) combined with azacitidine (a chemotherapy drug) in patients who either haven't responded well to other treatments or can't tolerate them.See study design
What are the potential side effects?
Potential side effects include immune system suppression leading to increased infection risk; mouth sores; nausea and vomiting; liver issues; blood count changes causing fatigue or bleeding risks; and possible allergic reactions.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
You have acute myeloid leukemia (AML) that has come back after treatment, has not responded to treatment, or you cannot tolerate standard chemotherapy.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Rate of response
Secondary outcome measures
Inhibition of mTOR signaling by sirolimus measured by intracellular flow cytometry for phosphorylation of the downstream signaling target S6 ribosomal protein as a surrogate for mTOR activity
Pharmacokinetic assessment to assess levels of the drug in vivo
Quality of life (QOL) assessed by the European Organization for Research and Treatment of Cancer (EORTC) QOL and the Mental Health Inventory (MHI)
+1 moreSide effects data
From 2008 Phase 4 trial • 293 Patients • NCT0011874229%
Diarrhoea
18%
Abdominal Pain
16%
Nausea
16%
Headache
16%
Fatigue
16%
Hepatitis C
14%
Vomiting
14%
Pyrexia
14%
Leukopenia
12%
Oedema Peripheral
11%
Insomnia
10%
Hyperkalaemia
10%
Tremor
10%
Back Pain
10%
Hypertension
10%
Anaemia
9%
Cough
9%
Pruritis
9%
Arthralgia
8%
Neutropenia
8%
Abdominal Pain Upper
8%
Dizziness
8%
Pain in Extremity
8%
Hepatic Enzyme Increased
7%
Dyspnoea
7%
Constipation
7%
Sinusitis
7%
Weight Decreased
6%
Blood Creatinine Increased
6%
Liver Function Test Abnormal
6%
White Blood Cell Count Decreased
5%
Jaundice
5%
Decreased Appetite
5%
Muscle Spasms
5%
Renal Failure
5%
Weight Increased
5%
Upper Respiratory Tract Infection
5%
Nasopharyngitis
5%
Asthenia
5%
Incision Site Pain
5%
Depression
4%
Anorexia
4%
Night Sweats
4%
Oropharyngeal Pain
4%
Rhinorrhoea
3%
Hyperlipidaemia
3%
Pleural Effusion
3%
Thrombocytopenia
3%
Myalgia
3%
Rash
3%
Acne
3%
Incisional Hernia
2%
Pneumonia
2%
Hypokalaemia
2%
Sepsis
1%
Hypercholesterolaemia
1%
Inappropriate Antidiuretic Hormone Secretion
1%
Hypoglycaemia
1%
Portal Vein Thrombosis
1%
Cardiac Failure Congestive
1%
Clostridium Difficile Colitis
1%
Abdominal Hernia
1%
Blood Alkaline Phosphatase Increased
1%
Gastrointestinal Tract Adenoma
1%
Encephalopathy
1%
Febrile Neutropenia
1%
Gastritis
1%
Renal Failure Acute
1%
Blood Glucose Increased
1%
Spinal Osteoarthritis
1%
Multi-Organ Failure
1%
Ventricular Tachycardia
1%
Crohn's Disease
1%
Hepatic Neoplasm Malignant
1%
Chest Pain
1%
Non-Small Cell Lung Cancer Metastatic
1%
Urinary Retention
1%
Transplant Rejection
1%
Benign Prostatic Hyperplasia
1%
Cerebral Haemorrhage
1%
Confusional State
1%
Convulsion
1%
Peritonitis
1%
Haemorrhage Intracranial
1%
Deep Vein Thrombosis
1%
Inguinal Hernia
1%
Viral Infection
1%
Acarodermatitis
1%
Gastrointestinal Haemorrhage
1%
Atrial Fibrillation
1%
Malaise
1%
Hepatic Cancer Metastatic
1%
Adenocarcinoma
1%
B-Cell Lymphoma
1%
Desmoid Tumour
1%
Pulmonary Embolism
1%
Stomatitis
1%
Influenza
1%
Staphylococcal Infection
1%
Umbilical Hernia
1%
Hepatic Function Abnormal
1%
Atrial Flutter
1%
Hyponatraemia
1%
Bacteraemia
1%
Cellulitis
1%
Clostridial Infection
1%
Diverticulitis
1%
Escherichia Urinary Tract Infection
1%
Lactobacillus Infection
1%
Lobar Pneumonia
1%
Pseudomonal Sepsis
1%
Post Procedural Haemorrhage
1%
Procedural Pain
1%
Biliary Anastomosis Complication
1%
Complications of Transplanted Kidney
1%
Bile Duct Obstruction
1%
Bile Duct Stenosis
1%
Biliary Tract Disorder
1%
Autoimmune Hepatitis
1%
Cholestasis
1%
Hepatic Artery Stenosis
1%
Hepatic Failure
1%
Epstein-Barr Virus Associated Lymphoproliferative Disorder
1%
Lung Disorder
1%
Pulmonary Oedema
1%
Sinus Congestion
1%
Embolism Venous
1%
Orthostatic Hypotension
1%
Vasculitis
1%
Hyperglycaemia
1%
Graft Versus Host Disease
100%
80%
60%
40%
20%
0%
Study treatment Arm
CellCept + CNI (Tacrolimus or Cyclosporine)
CellCept + Sirolimus
Trial Design
3Treatment groups
Experimental Treatment
Group I: MDS or AML with prior Azacitadine therapyExperimental Treatment2 Interventions
Patients receive sirolimus PO on days 1-10 or 1-12 and azacitidine IV on days 4-8, 11, and 12 or days 4-10. Treatment repeats every 28 days in the absence of disease progression or unacceptable toxicity.
Group II: High risk Myleodysplastic Syndrome (MDS)Experimental Treatment2 Interventions
Patients receive sirolimus PO on days 1-10 or 1-12 and azacitidine IV on days 4-8, 11, and 12 or days 4-10. Treatment repeats every 28 days in the absence of disease progression or unacceptable toxicity.
Group III: Acute Myeloid Leukemia (AML)Experimental Treatment2 Interventions
Patients receive sirolimus PO on days 1-10 or 1-12 and azacitidine IV on days 4-8, 11, and 12 or days 4-10. Treatment repeats every 28 days in the absence of disease progression or unacceptable toxicity.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Sirolimus
2013
Completed Phase 4
~2750
Azacitidine
2012
Completed Phase 3
~1440
Find a Location
Who is running the clinical trial?
Sidney Kimmel Cancer Center at Thomas Jefferson UniversityLead Sponsor
162 Previous Clinical Trials
10,854 Total Patients Enrolled
Neil Palmisiano, MDPrincipal InvestigatorSidney Kimmel Cancer Center at Thomas Jefferson University
1 Previous Clinical Trials
39 Total Patients Enrolled
Margaret Kasner, MDPrincipal InvestigatorSidney Kimmel Cancer Center at Thomas Jefferson University
6 Previous Clinical Trials
181 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have acute myeloid leukemia (AML) that has come back after treatment, has not responded to treatment, or you cannot tolerate standard chemotherapy.
Research Study Groups:
This trial has the following groups:- Group 1: High risk Myleodysplastic Syndrome (MDS)
- Group 2: Acute Myeloid Leukemia (AML)
- Group 3: MDS or AML with prior Azacitadine therapy
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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