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mTOR Inhibitor

Sirolimus for Blood Disorder

Phase 2

Recruiting

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

Must not have

History of lymphoma or other hematologic malignancies

Uncontrolled bleeding

Timeline

Screening 3 weeks

Treatment Varies

Follow Up through study completion; an average of 1 year

Awards & highlights

No Placebo-Only Group

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.

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.

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 below

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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%.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a history of lymphoma or blood cancer.

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I do not have any ongoing issues with uncontrolled bleeding.

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I have been diagnosed with a blood cancer or myelodysplastic syndrome.

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I haven't taken sirolimus, mTOR inhibitors, or B-cell therapies in the last 28 days.

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I have not used cannabidiol in the week before the study starts.

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I haven't had a heart attack in the last 6 months and my heart failure is not severe.

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I have not received any live vaccines in the last 28 days.

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I have been diagnosed with a chronic viral infection or tuberculosis.

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

This trial's timeline: 3 weeks for screening, Varies for treatment, and through study completion; an average of 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Safety and adverse events (AEs)

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

Cough

Pruritis

Arthralgia

Neutropenia

Abdominal Pain Upper

Dizziness

Pain in Extremity

Hepatic Enzyme Increased

Dyspnoea

Constipation

Sinusitis

Weight Decreased

Blood Creatinine Increased

Liver Function Test Abnormal

White Blood Cell Count Decreased

Muscle Spasms

Jaundice

Renal Failure

Decreased Appetite

Weight Increased

Upper Respiratory Tract Infection

Nasopharyngitis

Asthenia

Incision Site Pain

Depression

Anorexia

Night Sweats

Oropharyngeal Pain

Rhinorrhoea

Hyperlipidaemia

Thrombocytopenia

Pleural Effusion

Myalgia

Rash

Acne

Incisional Hernia

Sepsis

Pneumonia

Hypokalaemia

Renal Failure Acute

Hypoglycaemia

Urinary Retention

Clostridium Difficile Colitis

Cerebral Haemorrhage

Hepatic Artery Stenosis

Portal Vein Thrombosis

Gastrointestinal Tract Adenoma

Encephalopathy

Transplant Rejection

Confusional State

Blood Alkaline Phosphatase Increased

Multi-Organ Failure

Chest Pain

Non-Small Cell Lung Cancer Metastatic

Atrial Flutter

Benign Prostatic Hyperplasia

Ventricular Tachycardia

Febrile Neutropenia

Hepatic Failure

Hepatic Neoplasm Malignant

Gastritis

Epstein-Barr Virus Associated Lymphoproliferative Disorder

Crohn's Disease

Abdominal Hernia

Inappropriate Antidiuretic Hormone Secretion

Gastrointestinal Haemorrhage

Cardiac Failure Congestive

Blood Glucose Increased

Spinal Osteoarthritis

Hypercholesterolaemia

Convulsion

Peritonitis

Haemorrhage Intracranial

Deep Vein Thrombosis

Inguinal Hernia

Viral Infection

Acarodermatitis

Atrial Fibrillation

Malaise

Hepatic Cancer Metastatic

Adenocarcinoma

B-Cell Lymphoma

Desmoid Tumour

Pulmonary Embolism

Stomatitis

Influenza

Staphylococcal Infection

Umbilical Hernia

Hepatic Function Abnormal

Hyponatraemia

Bacteraemia

Cellulitis

Clostridial Infection

Diverticulitis

Escherichia Urinary Tract Infection

Lactobacillus Infection

Lobar Pneumonia

Pseudomonal Sepsis

Post Procedural Haemorrhage

Procedural Pain

Biliary Anastomosis Complication

Complications of Transplanted Kidney

Bile Duct Obstruction

Bile Duct Stenosis

Biliary Tract Disorder

Autoimmune Hepatitis

Cholestasis

Lung Disorder

Pulmonary Oedema

Sinus Congestion

Embolism Venous

Orthostatic Hypotension

Vasculitis

Hyperglycaemia

Graft Versus Host Disease

100%

80%

60%

40%

20%

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

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

0 / 14Eligibility criteria met