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Anti-metabolites

Sirolimus + Azacitidine for Myelodysplastic Syndrome

Phase 2
Waitlist Available
Led By Margaret Kasner, 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
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group

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.
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.
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.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Rate of response
Secondary study objectives
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
Toxicity referring to toxic events during the full course of treatment that are attributed as possibly, probably or definitely due to treatment, graded according to the National Institutes of Health (NIH) Common Toxicity Criteria (CTC) v. 4.0

Side effects data

From 2008 Phase 4 trial • 293 Patients • NCT00118742
29%
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%
Anaemia
10%
Hyperkalaemia
10%
Tremor
10%
Back Pain
10%
Hypertension
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%
Muscle Spasms
5%
Renal Failure
5%
Jaundice
5%
Decreased Appetite
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%
Thrombocytopenia
3%
Pleural Effusion
3%
Myalgia
3%
Rash
3%
Acne
3%
Incisional Hernia
2%
Sepsis
2%
Pneumonia
2%
Hypokalaemia
1%
Renal Failure Acute
1%
Hepatic Neoplasm Malignant
1%
Hypoglycaemia
1%
Urinary Retention
1%
Clostridium Difficile Colitis
1%
Cerebral Haemorrhage
1%
Hepatic Artery Stenosis
1%
Hepatic Failure
1%
Portal Vein Thrombosis
1%
Multi-Organ Failure
1%
Epstein-Barr Virus Associated Lymphoproliferative Disorder
1%
Gastrointestinal Tract Adenoma
1%
Febrile Neutropenia
1%
Encephalopathy
1%
Atrial Flutter
1%
Transplant Rejection
1%
Confusional State
1%
Blood Alkaline Phosphatase Increased
1%
Chest Pain
1%
Benign Prostatic Hyperplasia
1%
Non-Small Cell Lung Cancer Metastatic
1%
Ventricular Tachycardia
1%
Gastritis
1%
Crohn's Disease
1%
Abdominal Hernia
1%
Inappropriate Antidiuretic Hormone Secretion
1%
Gastrointestinal Haemorrhage
1%
Cardiac Failure Congestive
1%
Blood Glucose Increased
1%
Spinal Osteoarthritis
1%
Hypercholesterolaemia
1%
Convulsion
1%
Peritonitis
1%
Haemorrhage Intracranial
1%
Deep Vein Thrombosis
1%
Inguinal Hernia
1%
Viral Infection
1%
Acarodermatitis
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%
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%
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

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

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
Azacitidine
2012
Completed Phase 3
~1440
Sirolimus
2013
Completed Phase 4
~2750

Find a Location

Who is running the clinical trial?

Sidney Kimmel Cancer Center at Thomas Jefferson UniversityLead Sponsor
163 Previous Clinical Trials
10,963 Total Patients Enrolled
Margaret Kasner, MDPrincipal InvestigatorSidney Kimmel Cancer Center at Thomas Jefferson University
6 Previous Clinical Trials
181 Total Patients Enrolled
Neil Palmisiano, MDPrincipal InvestigatorSidney Kimmel Cancer Center at Thomas Jefferson University
1 Previous Clinical Trials
39 Total Patients Enrolled

Media Library

Azacitidine (Anti-metabolites) Clinical Trial Eligibility Overview. Trial Name: NCT01869114 — Phase 2
Myelodysplastic Syndrome Research Study Groups: High risk Myleodysplastic Syndrome (MDS), Acute Myeloid Leukemia (AML), MDS or AML with prior Azacitadine therapy
Myelodysplastic Syndrome Clinical Trial 2023: Azacitidine Highlights & Side Effects. Trial Name: NCT01869114 — Phase 2
Azacitidine (Anti-metabolites) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01869114 — Phase 2
~13 spots leftby Apr 2028
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