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Sirolimus for Aplastic Anemia

Phase 2
Recruiting
Led By Neal S Young, M.D.
Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age greater than or equal to 2 years old
Previous diagnosis of SAA by bone marrow biopsy and cytogenetics, treated with lymphodepleting therapy ATG, cyclophosphamide or alemtuzumab that included cyclosporine administered at least 12 months prior
Must not have
Patients on CYP3A4 inhibitors and cannot replace these medications with other equivalent medications for the period of study
Patients with infections not adequately responding to appropriate therapy
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 months
Awards & highlights

Summary

This trial is studying sirolimus to see how well it works in preventing aplastic anemia from returning after treatment with cyclosporine is stopped.

Who is the study for?
This trial is for people aged 2 and older who weigh more than 12 kg, have severe aplastic anemia (SAA) previously treated with immunosuppressive therapy including cyclosporine, and show a positive response to treatment. They must not be pregnant or breastfeeding, should agree to use birth control if of childbearing potential, haven't used sirolimus or similar drugs recently, aren't on certain medications that affect the study drug's action, don't have other blood disorders like leukemia or myelodysplastic syndrome, and are generally healthy enough to participate.
What is being tested?
The trial tests whether taking Sirolimus orally can prevent SAA from returning after stopping cyclosporine treatment. Participants will either receive Sirolimus daily for three months while being closely monitored (Group 1), or they'll stop cyclosporine without receiving Sirolimus but still under close observation (Group 2). The effectiveness will be measured through regular blood tests and bone marrow biopsies over five years.
What are the potential side effects?
Sirolimus may cause side effects such as increased risk of infections due to immune system suppression; it could also potentially affect kidney function and increase cholesterol levels. There might be risks related to liver function changes and lung problems as well.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am at least 2 years old.
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I was treated for severe aplastic anemia over a year ago with specific therapies.
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My blood tests show I've responded well to a treatment that reduces white blood cells.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I am taking medications that affect liver enzyme levels and cannot switch them.
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My infection is not improving despite treatment.
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I am not too sick or weak to handle sirolimus treatment.
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I can understand the study details and can give informed consent myself or have someone who can.
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I have not received a live vaccine in the last 30 days.
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My aplastic anemia relapsed after stopping cyclosporine in the last 6 months.
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I haven't taken sirolimus or similar drugs in the last 3 months.
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I am currently on chemotherapy or medication affecting my blood.
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I have been diagnosed with myelodysplastic syndrome or acute myeloid leukemia.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
To determine if the rate of relapse at 24 months after CSA discontinuation can be improved by conversion to sirolimus in severe aplastic anemia patients who have responded to IST.

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%
Jaundice
5%
Renal Failure
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%
Pleural Effusion
3%
Myalgia
3%
Hyperlipidaemia
3%
Thrombocytopenia
3%
Rash
3%
Acne
3%
Incisional Hernia
2%
Sepsis
2%
Pneumonia
2%
Hypokalaemia
1%
Urinary Retention
1%
Clostridium Difficile Colitis
1%
Benign Prostatic Hyperplasia
1%
Renal Failure Acute
1%
Hypoglycaemia
1%
Blood Alkaline Phosphatase Increased
1%
Gastritis
1%
Cerebral Haemorrhage
1%
Crohn's Disease
1%
Abdominal Hernia
1%
Chest Pain
1%
Hepatic Failure
1%
Multi-Organ Failure
1%
Hepatic Neoplasm Malignant
1%
Non-Small Cell Lung Cancer Metastatic
1%
Ventricular Tachycardia
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

2Treatment groups
Experimental Treatment
Active Control
Group I: SirolimusExperimental Treatment1 Intervention
sirolimus
Group II: Standard of CareActive Control1 Intervention
no intervention
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?

National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
3,889 Previous Clinical Trials
47,834,089 Total Patients Enrolled
11 Trials studying Aplastic Anemia
922 Patients Enrolled for Aplastic Anemia
Neal S Young, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
18 Previous Clinical Trials
2,886 Total Patients Enrolled
1 Trials studying Aplastic Anemia
27 Patients Enrolled for Aplastic Anemia
Bhavisha A Patel, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
1 Previous Clinical Trials
39 Total Patients Enrolled
~23 spots leftby Aug 2026
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