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mTOR Inhibitor
Arm 1 for Thrombocytopenia
Phase 2
Waitlist Available
Led By Courtney DiNardo, MD
Research Sponsored by M.D. Anderson Cancer Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Aged ≥18 years at the time of signing the informed consent
Confirmed P/LP germline RUNX1 variant per ClinGen Myeloid Malignancy Variant Curation Expert Panel (MM-VCEP) RUNX1-specific variant curation rules
Timeline
Screening 3 weeks
Treatment Varies
Follow Up through study completion; an average of 1 year
Awards & highlights
Study Summary
This trial aims to study the safety and effects of low-dose sirolimus in individuals with RUNX1-FPD.
Who is the study for?
Adults over 18 with RUNX1 Familial Platelet Disorder who have a platelet count of ≥50,000/µL and normal heart, liver, and kidney function can join. They must not have had certain blood disorders or treatments like sirolimus recently and should be free from serious infections or uncontrolled bleeding.Check my eligibility
What is being tested?
The trial is testing the safety and effects of low-dose Sirolimus on patients with thrombocytopenia due to RUNX1-FPD. Participants will provide bone marrow samples before and after treatment to assess changes in hematopoietic function.See study design
What are the potential side effects?
Sirolimus may cause side effects such as increased risk of infection, problems with liver or kidney function, high cholesterol levels, potential for blood clots or bleeding issues. Specific side effect experiences can vary among individuals.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am 18 years old or older.
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I have a confirmed genetic mutation in RUNX1.
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I am willing to give bone marrow samples at the start and end of my treatment.
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My kidney function, based on a specific test, is adequate.
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My liver tests are within normal limits.
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My heart pumps well, with an ejection fraction over 50%.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ through study completion; an average of 1 year
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~through study completion; an average of 1 year
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Safety and adverse events (AEs)
Side 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%
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%
Renal Failure
5%
Jaundice
5%
Muscle Spasms
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%
Myalgia
3%
Pleural Effusion
3%
Hyperlipidaemia
3%
Thrombocytopenia
3%
Rash
3%
Acne
3%
Incisional Hernia
2%
Sepsis
2%
Pneumonia
2%
Hypokalaemia
1%
Gastritis
1%
Urinary Retention
1%
Abdominal Hernia
1%
Multi-Organ Failure
1%
Ventricular Tachycardia
1%
Cerebral Haemorrhage
1%
Renal Failure Acute
1%
Hypoglycaemia
1%
Benign Prostatic Hyperplasia
1%
Non-Small Cell Lung Cancer Metastatic
1%
Crohn's Disease
1%
Clostridium Difficile Colitis
1%
Hepatic Neoplasm Malignant
1%
Hepatic Failure
1%
Chest Pain
1%
Blood Alkaline Phosphatase Increased
1%
Inappropriate Antidiuretic Hormone Secretion
1%
Gastrointestinal Haemorrhage
1%
Cardiac Failure Congestive
1%
Hepatic Artery Stenosis
1%
Portal Vein Thrombosis
1%
Epstein-Barr Virus Associated Lymphoproliferative Disorder
1%
Gastrointestinal Tract Adenoma
1%
Febrile Neutropenia
1%
Encephalopathy
1%
Atrial Flutter
1%
Blood Glucose Increased
1%
Transplant Rejection
1%
Confusional State
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
Trial Design
1Treatment groups
Experimental Treatment
Group I: Arm 1Experimental Treatment1 Intervention
Participants will visit the study clinic 2 times during Week 1, one (1) time during Weeks 2-4, and then 1 time every 2 weeks after that (Weeks 6, 8, 10, and so on) until Week 22 (Month 6). Then participants will have a follow-up visit at Week 24 and again at Week 52 (Month 12). Participants will take sirolimus by mouth every day, at about the same time each day. Swallow the tablet(s) whole with a full glass of water (about 1 cup). Do not crush or chew the tablet(s). Participants may take sirolimus with or without food.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Sirolimus
2013
Completed Phase 4
~2750
Find a Location
Who is running the clinical trial?
M.D. Anderson Cancer CenterLead Sponsor
2,975 Previous Clinical Trials
1,789,503 Total Patients Enrolled
7 Trials studying Thrombocytopenia
174 Patients Enrolled for Thrombocytopenia
Courtney DiNardo, MDPrincipal InvestigatorM.D. Anderson Cancer Center
10 Previous Clinical Trials
588 Total Patients Enrolled
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there any available positions for patients to participate in this clinical trial?
"Clinicaltrials.gov indicates that the specified trial is presently not seeking participants. The initial posting date was August 31, 2024, with the most recent update on February 8, 2024. While this study is currently inactive in terms of recruitment, it's worth noting that there are three other ongoing trials actively enrolling volunteers."
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