When I was at NIH in 1987, I participated in a Clinical Trial. Here's more about what a clinical trial entails.

A clinical trial is an organized study conducted in order to answer specific questions about a new treatment or a new way of using a known treatment. Each study tries to increase medical knowledge and to find new and better ways to help patients. Besides studying new drugs, clinical trials study new combinations of drugs already used, new ways of giving treatment, and how changes in lifestyle can help patients. Other clinical trials compare the best known standard therapy with a newer therapy to see if one produces more cures and causes fewer side effects than the other.

Most clinical trials are carried out in steps called phases. Each phase is designed to find different information. Patients may be eligible for studies in different phases, depending on their general condition, the type and stage of their disorder, and what therapy, if any, they have already had. Patients are seen regularly to determine the effect of the treatment, and treatment is always stopped if side effects become too severe.

With information about ongoing and upcoming clinical trials around the world and all the latest news on medical advances, this section promises to be yet another important source of information for pituitary patients.

 

 


From Rare/Orphan Diseases

Many Patients Unaware of Clinical Trials

Many clinical studies to test new methods of treating illnesses can't get enough volunteers to participate, say recent studies and polls. The American Society for Clinical Oncology surveyed 6,000 cancer patients in a poll and found 84% of them either didn't know about clinical studies or didn't think studies were an option for them. The poll found that only 4% of the patients surveyed had participated in a study.

The New England Journal of Medicine published a study comparing elderly cancer patients enrolled in clinical studies to those not in studies. The article reported that although 63% of people with cancer in the U.S. are age 65 or older, elderly cancer patients make up only 25% of patients in cancer studies. In breast cancer studies the disparity was even greater: the elderly make up 49% of patients but only 9% of study subjects.

There may be several reasons why patients aren't participating in clinical studies. One reason may be that since the majority of people who do participate in studies learn about them from their physicians, perhaps doctors themselves are unaware of clinical studies, or the doctors may be deciding ahead of time that some patients won't be eligible for certain studies, and thus don't talk to those patients about study participation.

Another reason for low participation may be patients' concerns about insurance coverage for the doctor visits, tests, and medicines required for clinical studies. In June, President Clinton announced that Medicare will now reimburse the routine patient care costs of clinical trials to test new therapies. This should help increase the numbers of elderly patients involved in clinical trials. Other insurance plans already often cover study costs. In the survey of cancer patients mentioned earlier, of those who had participated in clinical studies, 86% had secured insurance coverage.

Other misconceptions that patients have about clinical studies are that they won't get the best treatment if they participate, or they will get a placebo (sugar pill) and be treated like a guinea pig. Besides the fact that clinical studies are subject to very strict rules and guidelines, and are evaluated very carefully before they are even started, participants in studies are fully informed about everything in the study before they make any decision to participate. In that same survey of cancer patients participating in studies, 97% said that they received excellent or good quality care, were treated with dignity, and would recommend participation to others.

The National Institutes of Health provides complete information about what clinical trials are, what you should know about participation, and what questions to ask if you're considering enrolling in a study.

 

 

This study is currently recruiting participants.

Summary

Number

13-CH-0170

Sponsoring Institute

National Institute of Child Health and Human Development (NICHD)

Recruitment Detail

Type: Participants currently recruited/enrolled 
Gender: Male & Female 
Min Age: 
Max Age: 17

Referral Letter Required

No

Population Exclusion(s)

None

Special Instructions

Currently Not Provided

Keywords

Child;
Cushing Syndrome;
Metabolism;
Mifepristone;
Pharmacokinetic-Pharmacodynamic

Recruitment Keyword(s)

None

Condition(s)

Cushing's Syndrome;
Cushing Syndrome

Investigational Drug(s)

Mifepristone

Investigational Device(s)

None

Intervention(s)

Drug: mifepristone

Supporting Site

National Institute of Child Health and Human Development

Background:

 

- There are currently no approved therapies for children with Cushing's disease who are not cured by surgery alone. A drug called mifepristone has been approved to treat adults with Cushing's syndrome and elevated blood glucose caused by Cushing's. The drug is marketed under the name Korlym(Registered Trademark). The study drug may have a different effect on a child's body than an adult's, so researchers want to know how much of the drug to give children and what effect it will have. They want to learn if mifepristone improves Cushing's disease in children as it does in adults. They also want to know about the drug's side effects in children.

Objectives:

- To study the effect of a medication called mifepristone in children with Cushing's disease that has not been helped by pituitary surgery.

Eligibility:

- Children ages 6 to 17 with active Cushing's disease following pituitary surgery and who have a body weight higher than expected for their height and age.

Design:

- Participants will be screened for up to 8 weeks with a physical exam, medical history, and medical tests including blood tests and X-rays.

- Participants will take tablets of the study drug each day for 12 weeks.

- Participants will stay at the clinic for 4 nights at the beginning of the study. They will have three 1-day visits during the study. They will stay at the clinic the last 3 days of the study.

- At these visits, participants will be given several tests. In one test, a small wire is inserted under the skin of the belly and a small monitor is attached taped to the belly. In another, the participant drinks a liquid and blood samples are taken.

- Follow-up visits will occur 4 weeks and 12 weeks after the study ends.

 

--Back to Top--

Eligibility

 

INCLUSION CRITERIA

Patients who are eligible for enrollment must meet the following eligibility criteria:

- Males and females 6-17 years at informed consent

- Active Cushing's disease as demonstrated by the following:

--24 hour Urinary Free Cortisol greater than the upper limit of normal for age on two urine collections during screening and

-- midnight serum cortisol > 4.4 mcg/dL (mean of two determinations on a single day at 2330 and 2400 during screening)

- Previous trans-sphenoidal surgery (TSS) for ACTH secreting pituitary tumor at least 3 months prior to screening

- Increased body weight defined by BMI Z-score of 1.5 or above

- Able to provide consent/assent

- Able to swallow study drug tablets (not crushed or split)

- Willing to use non-hormonal method of contraception in patients of reproductive potential

- Primary health care provider in home location

 

EXCLUSION CRITERIA:

- Hypercortisolism not due to Cushing's disease.

- Type 1 diabetes mellitus

- HbA1c geater than or equal to 9.5% at Screening

- Body weight < 25 kg

- Use of certain medications that are CYP3A substrates with narrow therapeutic ranges, such as simvastatin, lovastatin, cyclosporine, dihydroergotamine, ergotamine, fentanyl, pimozide, quinidine, sirolimus, and tacrolimus during the 4 weeks prior to starting study drug. Use of these medications is also prohibited until 2 weeks after end of dosing.

- Use of certain medications that are strong CYP3A inhibitors such as itraconazole, nefazodone, ritonavir, nelfinavir, indinavir, atazanavir, amprenavir, fosamprenavir, boceprevir, clarithromycin, conivaptan, lopinavir, mibefradil, posaconazole, saquinavir, telaprevir, telithromycin, and voriconazole during the 2 weeks prior to starting study drug.

Use of these medications is also prohibited until 2 weeks after end of dosing. Grapefruit and grapefruit juice are prohibited during this time frame.

- Use of certain medications that are strong inducers on CYP3A such as rifampin, rifabutin, rifapentin, phenobarbital, phenytoin, carbamazepine, St. John's wort during the 2 weeks prior to starting study drug. Use of these medications is also prohibited until 2 weeks after end of dosing.

- Use of medications used to treat hypercortisolism from the duration indicated below prior to Day 1. Use of the medications is also prohibited until after the end of study 4 week follow up visit.

--steroidogenesis inhibitors such as ketoconazole, metyrapone: 4 weeks

--cabergoline, bromocriptine, somatostatin analogs such as octreotide, lanreotide, pasireotide long acting formulations: 8 weeks (immediate release formulations: 2 weeks)

--mitotane: 8 weeks

- Use of systemic glucocorticoid medications beginning 1 month prior to screening or anticipated use of these medications except for the treatment of adrenal insufficiency. Use of glucocorticoid medications is prohibited during the study until after the end of study 4 week study visit.

- Inflammatory, rheumatological, proliferative or other disorder(s) that would be anticipated to worsen with glucocorticoid blockade (e.g. inflammatory bowel disease, rheumatoid arthritis, psoriasis, etc.).

- Uncontrolled hypo- or hyperthyroidism.

- Uncorrected hypokalemia (< 3.5 mEq/L). The screening period may be used to correct hypokalemia prior to starting study drug. Use of potassium and/or mineralocorticoid antagonists is permitted during the study.

- QTc geater than or equal to 450 msec on Screening electrocardiogram

- Unexplained vaginal bleeding in females and/or any history of endometrial pathology.

- Positive pregnancy test in females.

From http://clinicalstudies.info.nih.gov/cgi/detail.cgi?A_2013-CH-0170.html

Introduction

This study is being done to examine the effects of a medication called mifepristone in children with Cushing's disease. This medication has been approved by the Food and Drug Administration (FDA) for use in adult patients with Cushing's syndrome. It is not FDA approved for use in children. The study will investigate how children's bodies absorb and process mifepristone, how it works in children and what effect it has on the use of sugar in the body, on the child's weight and on growth hormone. An important part of the study is to determine the proper dosing and to evaluate the side effects of mifepristone in children.

Children 6 to 17 years old will be enrolled in the study if they have had surgery for Cushing's disease and currently have elevated cortisol levels.

To get started, please click here.

Cortendo Clinical Trial

 

About the Study

OBJECTIVE:

The purpose of this study is to test the effects of different doses of COR-003 on people with endogenous Cushing’s syndrome, primarily by measuring the cortisol levels in urine and secondarily by measuring other health parameters such as blood pressure, weight, liver function, etc. This study is also being conducted to find out if COR-003 is safe to use. This study is open-label, which means both the health providers and the participants in the study are aware of the drug or treatment being given.

STUDY DESIGN:

  • The study will begin with a screening period to make sure subjects are eligible to participate in the study.
  • After the screening period, subjects who are eligible for participation will each be given several different doses of COR-003, to be taken by mouth in tablet form.
  • After an individualized dose has been selected, participants will take COR-003 for 6 months.
  • Finally, participants will continue in the study for an additional 6 months at doses to be determined by the study doctor.
  • Throughout the study, participants will meet regularly with a study doctor and will take part in a variety of medical tests to make sure they are doing well and to see if COR-003 is working.
  • Participants in the study should be sure they have the time to participate. Participants will generally be followed for over a year.

See if you may be eligible for this clinical study. By providing your contact information, you will receive more information about the study and your eligibility.

About Cortendo

Cortendo is the sponsor of this study. This means Cortendo planned and organized this study. Cortendo will also collect and analyze the data from the study.

Cortendo is a global pharmaceutical company primarily focused on researching and providing treatments for rare diseases in endocrinology, such as Cushing’s syndrome. The company was founded in Sweden and its worldwide headquarters is located just outside of Philadelphia.

Fill out this form for more information: https://www.cushingssyndromestudy.com/registration.aspx

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Conditions: Cushing's Syndrome;   Adrenal Incidentalomas;   Alcoholism;   Obesity
Intervention:  
2 Recruiting Pattern of Gene Expression in Adipose Tissue From Patients With Cushing Syndrome
Condition: Cushing Syndrome Related to Cortisolic Adenoma
Intervention: Other: analyze the role of glucocorticoid in AT distribution.
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Condition: Cushing's Disease
Intervention: Drug: Pasireotide sub-cutaneous formulation
4 Recruiting Efficacy and Safety of Pasireotide Administered Monthly in Patients With Cushing's Disease
Condition: Cushing's Disease
Interventions: Drug: SOM230 LAR 30 mg;   Drug: SOM230 LAR 10 mg
5 Recruiting Mifepristone in Children With Refractory Cushing's Disease
Condition: Cushing's Disease
Intervention: Drug: mifepristone
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Condition: Cushing Syndrome
Intervention: Procedure: surgery
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Condition: Cushing's Disease
Interventions: Drug: Pasireotide;   Drug: Cabergoline
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Condition: Cushing's Disease
Intervention: Drug: Bexarotene
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Condition: Endogenous Cushing's Syndrome
Intervention: Drug: COR-003
10 Recruiting New Imaging Techniques in the Evaluation of Patients With Ectopic Cushing Syndrome
Condition: Cushing Syndrome
Intervention:  
11 Recruiting Adrenalectomy Versus Follow-up in Patients With Subclinical Cushings Syndrome
Condition: Adrenal Tumour With Mild Hypercortisolism
Intervention: Procedure: Adrenalectomy
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Condition: Cushing's Disease
Intervention: Drug: LCI699
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Conditions: Cushing's Syndrome;   Insulin Resistance
Intervention: Procedure: Surgery
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Condition: Pituitary Neoplasm
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15 Unknown  Study of Depression, Peptides, and Steroids in Cushing's Syndrome
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Intervention:  
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Conditions: Pituitary Adenomas;   Prolactinomas;   Cushing's Disease
Intervention: Drug: Lapatinib
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Conditions: Abnormalities;   Craniopharyngioma;   Cushing's Syndrome;   Endocrine Disease;   Pituitary Neoplasm
Intervention:  
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Conditions: Cushing's Syndrome;   Hereditary Neoplastic Syndrome;   Lentigo;   Neoplasm;   Testicular Neoplasm
Intervention:  
19 Recruiting Study to Allow Access to Pasireotide for Patients Benefiting From Pasireotide Treatment in a Novartis-sponsored Study.
Conditions: Cushing's Disease,;   Acromegaly,;   Neuroendocrine Tumors,;   Pituitary Tumors;   Ectopic ACTH Secreting (EAS) Tumors,;   Dumping Syndrome,;   Prostate Cancer,;   Melanoma Negative for bRAF,;   Melanoma Negative for nRAS
Intervention: Drug: Pasireotide
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Conditions: Adrenal Tumors;   Adrenocortical Carcinoma;   Cushing Syndrome;   Conn Syndrome;   Pheochromocytoma
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Intervention: Drug: Pasireotide
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Condition: Pituitary Tumor
Intervention:  
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Condition: Adrenal Gland Neoplasm
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Condition: Effects of Chemotherapy
Intervention:  
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Condition: Adrenal Tumors
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Condition: Kidney Stones
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Interventions: Biological: Red blood cell units stored <= 10 days;   Biological: Red blood cell units stored >= 21 days
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