Study Summary
This trial will study the effect of sirolimus on idiopathic multicentric Castleman disease, a rare disorder that can cause enlarged lymph nodes and other problems.
Eligible Conditions
- Castleman Disease
- Castleman's Disease, Multicentric
Treatment Effectiveness
Study Objectives
0 Primary · 6 Secondary · Reporting Duration: 3, 6, 9, and 12 months ± 2 weeks
Month 12
Disease activity, as measured by the CHAP scale
Disease activity, as measured by the MCD-related Overall Symptom Score
Proportion of patients achieving a lymph node response, following the modified Cheson response criteria
Month 9
Proportion of patients achieving a positive clinical benefit response (CBR)
Up to 73 weeks
Proportion of patients that indicate that they are currently receiving sirolimus at the end of the Follow Up Phase
Pharmaceutical Preparations
Trial Safety
Safety Progress
This is further along than 68% of similar trials
Side Effects for
CellCept + CNI (Tacrolimus or Cyclosporine)
29%Diarrhoea
18%Abdominal Pain
16%Hepatitis C
16%Nausea
16%Fatigue
16%Headache
14%Vomiting
14%Pyrexia
14%Leukopenia
12%Oedema Peripheral
11%Insomnia
10%Back Pain
10%Hyperkalaemia
10%Tremor
10%Anaemia
10%Hypertension
9%Arthralgia
9%Pruritis
9%Cough
8%Pain in Extremity
8%Abdominal Pain Upper
8%Hepatic Enzyme Increased
8%Dizziness
8%Neutropenia
7%Sinusitis
7%Constipation
7%Weight Decreased
7%Dyspnoea
6%White Blood Cell Count Decreased
6%Blood Creatinine Increased
6%Liver Function Test Abnormal
5%Nasopharyngitis
5%Depression
5%Jaundice
5%Asthenia
5%Upper Respiratory Tract Infection
5%Decreased Appetite
5%Weight Increased
5%Muscle Spasms
5%Incision Site Pain
5%Renal Failure
4%Night Sweats
4%Oropharyngeal Pain
4%Anorexia
4%Rhinorrhoea
3%Rash
3%Hyperlipidaemia
3%Thrombocytopenia
3%Incisional Hernia
3%Pleural Effusion
3%Acne
3%Myalgia
2%Sepsis
2%Pneumonia
2%Hypokalaemia
1%Gastrointestinal Haemorrhage
1%Bile Duct Stenosis
1%Autoimmune Hepatitis
1%Convulsion
1%Lung Disorder
1%Blood Glucose Increased
1%Benign Prostatic Hyperplasia
1%Biliary Tract Disorder
1%Acarodermatitis
1%Lobar Pneumonia
1%Inguinal Hernia
1%Bile Duct Obstruction
1%Hepatic Failure
1%Ventricular Tachycardia
1%Procedural Pain
1%Hyperglycaemia
1%Clostridium Difficile Colitis
1%Viral Infection
1%Bacteraemia
1%Escherichia Urinary Tract Infection
1%Cellulitis
1%Clostridial Infection
1%Diverticulitis
1%Influenza
1%Lactobacillus Infection
1%Pseudomonal Sepsis
1%Staphylococcal Infection
1%Umbilical Hernia
1%Crohn's Disease
1%Abdominal Hernia
1%Peritonitis
1%Gastritis
1%Biliary Anastomosis Complication
1%Complications of Transplanted Kidney
1%Post Procedural Haemorrhage
1%Cholestasis
1%Hepatic Artery Stenosis
1%Portal Vein Thrombosis
1%Hepatic Function Abnormal
1%Chest Pain
1%Multi-Organ Failure
1%Epstein-Barr Virus Associated Lymphoproliferative Disorder
1%Desmoid Tumour
1%Gastrointestinal Tract Adenoma
1%Adenocarcinoma
1%Malaise
1%Hepatic Cancer Metastatic
1%B-Cell Lymphoma
1%Hepatic Neoplasm Malignant
1%Pulmonary Embolism
1%Febrile Neutropenia
1%Non-Small Cell Lung Cancer Metastatic
1%Sinus Congestion
1%Embolism Venous
1%Pulmonary Oedema
1%Atrial Fibrillation
1%Cardiac Failure Congestive
1%Encephalopathy
1%Orthostatic Hypotension
1%Cerebral Haemorrhage
1%Deep Vein Thrombosis
1%Vasculitis
1%Hypoglycaemia
1%Haemorrhage Intracranial
1%Blood Alkaline Phosphatase Increased
1%Atrial Flutter
1%Hyponatraemia
1%Transplant Rejection
1%Confusional State
1%Urinary Retention
1%Graft Versus Host Disease
1%Renal Failure Acute
1%Spinal Osteoarthritis
1%Stomatitis
1%Inappropriate Antidiuretic Hormone Secretion
1%Hypercholesterolaemia
Trial Design
1 Treatment Group
Sirolimus
1 of 1
Experimental Treatment
24 Total Participants · 1 Treatment Group
Primary Treatment: Sirolimus · No Placebo Group · Phase 2
Sirolimus
Drug
Experimental Group · 1 Intervention: Sirolimus · Intervention Types: DrugTreatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Sirolimus
FDA approved
Trial Logistics
Trial Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 3, 6, 9, and 12 months ± 2 weeks
Who is running the clinical trial?
University of PennsylvaniaLead Sponsor
1,822 Previous Clinical Trials
41,116,199 Total Patients Enrolled
2 Trials studying Castleman Disease
1,130 Patients Enrolled for Castleman Disease
David C Fajgenbaum, MD, MBA, MSPrincipal InvestigatorUniversity of Pennsylvania
Eligibility Criteria
Age Any Age · All Participants · 6 Total Inclusion Criteria
Mark “Yes” if the following statements are true for you:You have evidence of active disease, defined as at least two abnormalities in the criteria comprising the CBR criteria, with at least one abnormality being enlarged/evaluable lymph node(s) as described in Cheson criteria.
About The Reviewer
Michael Gill - B. Sc.
First Published: October 27th, 2021
Last Reviewed: November 8th, 2022
Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.
References
- Arenas, Daniel J., Katherine Floess, Dale Kobrin, Ruth-Anne Langan Pai, Maya B. Srkalovic, Mark-Avery Tamakloe, Rozena Rasheed, et al.. 2020. “Increased Mtor Activation in Idiopathic Multicentric Castleman Disease”. Blood. American Society of Hematology. doi:10.1182/blood.2019002792.
- van Rhee, Frits, Eric Oksenhendler, Gordan Srkalovic, Peter Voorhees, Megan Lim, Angela Dispenzieri, Makoto Ide, et al.. 2020. “International Evidence-based Consensus Diagnostic and Treatment Guidelines for Unicentric Castleman Disease”. Blood Advances. American Society of Hematology. doi:10.1182/bloodadvances.2020003334.
- Fajgenbaum, David C., Ruth-Anne Langan, Alberto Sada Japp, Helen L. Partridge, Sheila K. Pierson, Amrit Singh, Daniel J. Arenas, et al.. 2019. “Identifying and Targeting Pathogenic Pi3k/akt/mtor Signaling in IL-6 Blockade–refractory Idiopathic Multicentric Castleman Disease”. Journal of Clinical Investigation. American Society for Clinical Investigation. doi:10.1172/jci126091.
- van Rhee F, Oksenhendler E, Srkalovic G, Voorhees P, Lim M, Dispenzieri A, Ide M, Parente S, Schey S, Streetly M, Wong R, Wu D, Maillard I, Brandstadter J, Munshi N, Bowne W, Elenitoba-Johnson KS, Fossa A, Lechowicz MJ, Chandrakasan S, Pierson SK, Greenway A, Nasta S, Yoshizaki K, Kurzrock R, Uldrick TS, Casper C, Chadburn A, Fajgenbaum DC. International evidence-based consensus diagnostic and treatment guidelines for unicentric Castleman disease. Blood Adv. 2020 Dec 8;4(23):6039-6050. doi: 10.1182/bloodadvances.2020003334.
- 2019. "Sirolimus in Previously Treated Idiopathic Multicentric Castleman Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03933904.
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