Symptoms and General Pathology

All Conditions

Blood and Lymph Conditions

Nutritional and Metabolic Diseases

Digestive System Diseases

Neoplasms

Immune System Diseases

Mouth and Tooth Diseases

Heart and Blood Diseases

Rare Diseases

Recurrence

Hemorrhage

Constipation

Diarrhea

Lymphadenopathy

Amyloidosis

Nausea

Gastrointestinal Hemorrhage

Immunoglobulin Light-chain Amyloidosis

Purpura

Hepatomegaly

Macroglossia

Signs and Symptoms, Digestive

Proteostasis Deficiencies

Metabolic Diseases

Hemostatic Disorders

Blood Coagulation Disorders

Hematologic Diseases

Skin Manifestations

Lymphatic Diseases

Gastrointestinal Diseases

Neoplasms, Plasma Cell

Neoplasms by Histologic Type

Lymphoproliferative Disorders

Immunoproliferative Disorders

Paraproteinemias

Liver Diseases

Hypertrophy

Pathological Conditions, Anatomical

Tongue Diseases

Mouth Diseases

Stomatognathic Diseases

AL Amyloidosis

Hypoglycemic Agents

All Drugs and Chemicals

Gliclazide

Patients receive isatuximab IV on days 1, 8, 15, and 22 of course 1 and on days 1 and 15 of subsequent courses. Treatment repeats every 28 days for 24 courses in the absence of disease progression or unacceptable toxicity.

Isatuximab

Laboratory Biomarker Analysis

Emma Scott

This phase II trial studies how well isatuximab works in treating patients with primary amyloidosis that has come back or does not respond to treatment. Monoclonal antibodies, such as isatuximab, may interfere with the ability of cancer cells to grow and spread.

PRIMARY OBJECTIVES:

I. To assess the efficacy as measured by the confirmed overall hematologic response rate (partial response or better) of isatuximab in relapsed/refractory systemic light chain (AL) amyloidosis.

SECONDARY OBJECTIVES:

I. To evaluate toxicities in the treatment of relapsed/refractory AL amyloidosis with isatuximab.

II. To evaluate time to hematologic response. III. To evaluate duration of response. IV. To evaluate progression-free survival (PFS). V. To evaluate overall survival (OS).

TERTIARY OBJECTIVES:

I. To evaluate efficacy of isatuximab in relapsed/refractory immunoglobulin amyloid light chain (AL) amyloidosis as measured by organ specific response rates (cardiac, renal, gastrointestinal \[GI\], liver, soft tissue, nerve), in the subset of patients that can be evaluated for organ response.

II. To evaluate time to organ response in the subset of patients that can be evaluated for organ response.

OUTLINE:

Patients receive isatuximab intravenously (IV) on days 1, 8, 15, and 22 of course 1 and on days 1 and 15 of subsequent courses. Treatment repeats every 28 days for 24 courses in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up within 30 days and then every at least every 6 months for up to 4 years.

S1702 Isatuximab in Treating Patients With Relapsed or Refractory Primary Amyloidosis

A Phase II Study of Isatuximab (SAR650984) (NSC-795145) for Patients With Previously Treated AL Amyloidosis

Will include partial response, very good partial response and complete response. Hematologic response will be reported with a binomial confidence interval.

Will be evaluated using the Common Terminology Criteria for Adverse Events version 4.0. The maximum grade for each toxicity will be recorded for each patient, and frequency tables will be reviewed to determine toxicity patterns.

Organ response will be evaluated in the subset of patients with evaluable organ involvement using current criteria. The proportion of patients with organ response will be estimated as the number of patients with documented organ response out of the number of patients with documented evaluable organ involvement at baseline.

Will be estimated using the Kaplan-Meier method.

National Cancer Institute (NCI)

SWOG Cancer Research Network

Polycythemia Vera

Polycythemia

Primary Myelofibrosis

Thrombocytosis

Thrombocythemia, Essential

Splenomegaly

Myeloproliferative Disorders

Bone Marrow Diseases

Bone Marrow Neoplasms

Hematologic Neoplasms

Blood Platelet Disorders

Hemorrhagic Disorders

Essential Thrombocythemia

Chronic Myeloproliferative Disorders

Antineoplastic Agents

Nivolumab

Antineoplastic Agents, Immunological

Immune Checkpoint Inhibitors

Patients receive nivolumab IV over 60 minutes once every 2 weeks for 8 doses and then once every 12 weeks thereafter. Treatment may continue for up to 4 years in the absence of disease progression or unacceptable toxicity.

Quality-of-Life Assessment

Srdan Verstovsek

This phase II trial studies how well nivolumab works in treating patients with primary myelofibrosis, post-essential thrombocythemia myelofibrosis, or post-polycythemia vera myelofibrosis. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.

PRIMARY OBJECTIVES:

I. To determine the efficacy/clinical activity of nivolumab in patients with myelofibrosis (MF).

SECONDARY OBJECTIVES:

I. To determine the safety of nivolumab in patients with MF.

TERTIARY OBJECTIVES:

I. To explore time to response and duration of response. II. To assess changes in symptom burden. III. To explore changes in bone marrow fibrosis. IV. To explore changes in Janus kinase 2 valine at amino acid position 617 (JAK2V617F) (or other molecular marker) allele burden or changes in cytogenetic abnormalities.

OUTLINE:

Patients receive nivolumab intravenously (IV) over 60 minutes once every 2 weeks for 8 doses and then once every 12 weeks thereafter. Treatment may continue for up to 4 years in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up at 30, 60, and 100 days.

Nivolumab in Treating Patients With Primary Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis, or Post-Polycythemia Vera Myelofibrosis

Phase 2 Study of Nivolumab in Patients With Primary Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis, or Post-Polycythemia Vera Myelofibrosis

Responses will be categorized according to the revised International Working Group-Myeloproliferative Neoplasms Research and Treatment and European LeukmiaNet consensus criteria for myelofibrosis.

M.D. Anderson Cancer Center

Patients receive nivolumab IV over 60 minutes once every 2 weeks for 8 doses and then once every 12 weeks thereafter. Treatment may continue for up to 4 years in the absence of disease progression or unacceptable toxicity.

Laboratory Biomarker Analysis: Correlative studies

Nivolumab: Given IV

Quality-of-Life Assessment: Ancillary studies

Treatment (Nivolumab)

Age, Categorical

Age, Continuous

Sex: Female, Male

Race (NIH/OMB)

United States

Region of Enrollment

Dr. Srdan Verstovsek, MD. Professor

Objective Response Rate, Defined as Complete Remission + Partial Remission + Clinical Improvement

Overall Study

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Diseases and Abnormalities at or Before Birth

Nervous System Diseases

Polycystic Kidney Diseases

Polycystic Kidney, Autosomal Dominant

Pain

Abdominal Pain

Cysts

Kidney Diseases

Neurologic Manifestations

Kidney Diseases, Cystic

Urologic Diseases

Urogenital Diseases

Female Urogenital Diseases

Pregnancy Complications

Female Urogenital Diseases and Pregnancy Complications

Male Urogenital Diseases

Congenital Abnormalities

Abnormalities, Multiple

Ciliopathies

Genetic Diseases, Inborn

Autosomal Dominant Multiple Pterygium Syndrome

Polycystic Liver Disease

Hematinics

Gastrointestinal Agents

Liver Extracts

Octreotide

Antineoplastic Agents, Hormonal

Participants received Octreotide LAR® Depot injections (up to 40 mg) intramuscularly every 28 days (+/- 5 days) for one year

Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year

Participants received Octreotide LAR® Depot injections (up to 40 mg)intramuscularly every 28 days (+/- 5 days) for one year

Placebo

Marie C. Hogan, M.D., Ph.D.

This study will evaluate the effect of Octreotide LAR® on the liver volumes of patients with severe polycystic liver disease who are not candidates or decline surgical treatments such as liver cyst fenestration, liver resection or liver transplantation. A total of 42 patients will be recruited -14 who will receive placebo and 28 the study drug. Preliminary evidence indicates that this drug is safe and non-toxic in other disease states. Treatment with this drug holds promise not only for individuals with liver involvement, but also for many more patients with polycystic kidney disease.

The primary aim of this study is to compare the effect of Octreotide LAR® Depot on the liver volume of patients with severe polycystic liver disease who are not candidates or decline surgical treatments such as liver cyst fenestration, liver resection or liver transplantation compared with placebo. The secondary aims of the study are: (1)Assess the effect of Octreotide LAR® Depot on the total kidney volume and iothalamate clearance in patients with polycystic kidney disease associated with severe polycystic liver disease who are not candidates or decline surgical treatments such as liver cyst fenestration, liver resection or liver transplantation. (2)Evaluate quality of life changes associated with the administration of Octreotide LAR® Depot in these patients. (3)Assess toxicity of Octreotide LAR® Depot in patients with polycystic liver disease (PLD).

Note: Subjects who completed this 1 year randomized trial were offered enrollment into an open-label (all subjects received Octreotide) extension trial for an additional two years of treatment.

Octreotide in Severe Polycystic Liver Disease

Pilot Study Of Long-Acting Octreotide (Octreotide LAR® Depot) In The Treatment Of Patients With Severe Polycystic Liver Disease

Percent change from baseline in liver volume, measured in milliliters by Magnetic Resonance Imaging (MRI)or Computed Tomography (CT) scans

Percent change from baseline in renal volume, measured in milliliters by MRI or CT scans

Percent change from baseline in renal function/GFR, measured by clearance of iothalamate with monitoring of bladder emptying using ultrasound

Scores on the Medical Outcomes Study 36-Item Short-Form General Health Survey (SF-36), version 2. Subjects completed the SF-36 which consists of 8 sub-scales which are additionally summarized into 2 summary components (physical and mental). The subscales and the summary scales both range from 0 to 100, with (0 = worst imaginable, 100 = best imaginable).

Novartis

National Center for Research Resources (NCRR)

Mayo Clinic

MD, PhD, Assistant Professor of Medicine, College of Medicine

Participants received Octreotide LAR® Depot injections intramuscularly every 28 days (+/- 5 days) for one year

Total

Age Continuous

Glomerular Filtration Rate measures how fast the kidneys are filtering. 13 subjects were excluded from the analysis of the kidney data: 4 subjects had renal transplant before enrollment (3 in octreotide and 1 in placebo groups), 8 subjects had a diagnosis of ADPLD (4 in each group), 1 placebo subject has missing kidney data due to incomplete image coverage.

Glomerular Filtration Rate

Body Mass Index (BMI) is a health index for comparing weight to height. BMI is a person's weight in kilograms (kg) divided by his or her height in meters squared. The body mass index is an indication if a person is at a suitable weight for his height on an approximation of body fat. A body mass index of under 20 is considered to be underweight, while a body mass index between 20 to 25 is considered healthy. A body mass index in the range of 25 to 30 is regarded as overweight. A body mass index over 30 is regarded as obese.

Body Mass Index

Body Weight

Creatinine in the serum is a blood test, and is an indicator of kidney function.

Serum creatinine

Fasting plasma glucose

A protein urine test measures the amount of proteins, such as albumin, found in a urine sample. Urine albumin is an indicator of kidney function.

Urine Albumin

Systolic blood pressure is the upper number on a blood pressure reading and measures contraction of the ventricles, or lower chambers of the heart

Systolic Blood Pressure

Diastolic blood pressure is the lower number on a blood pressure reading and measures dilation of the ventricles, or lower chambers of the heart.

Diastolic Blood Pressure

Liver volume was measured by magnetic resonance imaging (MRI) or computed tomography \[CT\].

Liver volume

Kidney volume was measured by magnetic resonance imaging (MRI) or computed tomography \[CT\].

13 subjects were excluded from the analysis of the kidney data: 4 subjects had renal transplant before enrollment (3 in octreotide and 1 in placebo groups), 8 subjects had a diagnosis of ADPLD (4 in each group), 1 placebo subject has missing kidney data due to incomplete image coverage. (Octreotide group n = 21 and placebo group n = 8).

Kidney volume

ADPKD genotype with PKD1 gene mutation

ADPKD genotype with PKD2 gene mutation

ADPKD genotype with no genetic mutation detected

ADPLD genotype with PRKCSH gene mutation

ADPLD genotype with SEC63 gene mutation

ADPLD genotype with no genetic mutation detected

Autosomal dominant polycystic kidney disease (ADPKD) is caused by mutations in one of two genes: PKD1 or PKD2, and is characterized by the development of kidney cysts and other manifestations, of which polycystic live disease (PLD) is most common. Autosomal dominant PLD (ADPLD) also exists as a genetically distinct disease with few or absent kidney cysts. Like ADPKD, ADPLD is genetically heterogeneous, with the 1st two genes identified (PRKCSH \& SEC63) accounting for approximately 1/3 to 1/2 of isolated ADPLD cases.

Genotypes and genetic mutations

Marie Hogan, MD, PhD, Assistant Prof of Medicine, College of Medicine

P values \<0.05 were considered statistically significant.

Percent Change in Liver Volume

13 subjects were excluded from the analysis of the kidney data: 4 subjects had renal transplant before enrollment (3 in octreotide and 1 in placebo groups), 8 subjects had a diagnosis of ADPLD (4 in each group), 1 placebo subject has missing kidney data due to incomplete image coverage.

Percent Change in Renal Volume

Percent Change in Glomerular Filtration Rate (GFR)

Physical functioning at baseline

Physical functioning at 12 months

Physical role at baseline

Physical role at 12 months

Bodily Pain at baseline

Bodily pain at 12 months

General health at baseline

General health at 12 months

Vitality at baseline

Vitality at 12 months

Social functioning at baseline

Social functioning at 12 months

Emotional role at baseline

Emotional role at 12 month

Mental health at baseline

Mental health at 12 months

Standardized physical component at baseline

Standardized physical component at 12 months

Standardized mental component at baseline

Standardized mental component at 12 month

Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
S1702 Isatuximab in Treating Patients With Relapsed or Refractory Primary Amyloidosis	NCT03499808	Amorphous, Eosi... Constipation Diarrhea Early Satiety Gastrointestina... Hepatomegaly Lymphadenopathy Macroglossia Nausea Primary Systemi... Purpura Recurrent Prima... Refractory Prim...	Isatuximab Laboratory Biom...	18 Years -	SWOG Cancer Research Network
Nivolumab in Treating Patients With Primary Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis, or Post-Polycythemia Vera Myelofibrosis	NCT02421354	Hepatomegaly Myelofibrosis T... Polycythemia Ve... Primary Myelofi... Splenomegaly	Laboratory Biom... Nivolumab Quality-of-Life...	18 Years -	M.D. Anderson Cancer Center
Nivolumab in Treating Patients With Primary Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis, or Post-Polycythemia Vera Myelofibrosis	NCT02421354	Hepatomegaly Myelofibrosis T... Polycythemia Ve... Primary Myelofi... Splenomegaly	Laboratory Biom... Nivolumab Quality-of-Life...	18 Years -	M.D. Anderson Cancer Center
Octreotide in Severe Polycystic Liver Disease	NCT00426153	Polycystic Kidn... Polycystic Live... Hepatomegaly Liver Diseases Kidney, Polycys... Abdominal Pain	Octreotide Placebo	18 Years - 80 Years	Mayo Clinic

Spots Global Cancer Trial Database for hepatomegaly

Every month we try and update this database with for hepatomegaly cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.