Long-Term Follow-Up of Survivors of Pediatric Cushing Disease

Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Brief Summary:

Background:

The pituitary gland produces hormones. A tumor in this gland can cause it to produce too much of the hormone cortisol. Too much cortisol in the body causes Cushing disease. This disease causes many problems. Some of these problems might persist after the disease is cured.

Objective:

To find out the long-term effects of exposure to high levels of cortisol during childhood and adolescence.

Eligibility:

People ages 10-42years who were diagnosed with Cushing disease before age 21 and are now cured and have normal or low cortisol levels

People related to someone with Cushing disease

Design:

Participants will be screened with a medical history.

Participants will complete an online survey. This will include questions about their or their child s physical and mental health.

All participants will be seen at 5 -year intervals after cure of Cushing disease (5yr, 10yr, 15yr, 20yr (last visit))

Participants who have a relative with Cushing disease will have a medical history and blood tests or cheek swabs.

Participants who have the disease will have:

Physical exam

Blood tests

Cheek swab

DXA scan: A machine will x-ray the participant s body to measure bone mineral content.

For participants who are still growing, a hand x-ray

Participants with the disease may also have:

Hormone stimulation test: Participants will get a hormone or another substance that will be measured.

Serial hormone sampling: Participants blood will be measured several times through a thin plastic tube in an arm vein.

Urine tests: Participants urine may be collected over 24 hours.

MRI: Participants may have a dye injected into a vein. They will lie on a table that slides into a machine. The machine will take pictures of the body.

Read more https://clinicaltrials.gov/ct2/show/NCT03831958#eligibility

❓Clinical Trial: Congenital Adrenal Hyperplasia (CAH)


The CAHmelia clinical trials are exploring a new investigational treatment for classic CAH.

CAHmelia 203 and CAHmelia 204 are clinical trials to test tildacerfont in adults with classic CAH, which may offer you and your loved ones hope of a brighter future – one where you may not have to choose between symptom management and long-term health.
Tildacerfont is a new type of oral, once-daily investigational treatment – one that is not a steroid – that is currently being tested in adults with classic CAH. By reducing the amount of androgens your body makes, tildacerfont may improve your classic CAH symptoms.
This investigational treatment will not replace your steroid treatment but may allow you to manage your disease with lower amounts of steroids at normal or near-normal doses.

Who can take part in this trial? You may be able to take part if you:Are at least 18 years of ageHave a confirmed diagnosis of classic CAH due to 21-OH deficiencyHave been on the same daily dose of steroids (GCs and/or mineralocorticoids) for at least 1 month before starting the trialBoth trials are now open for enrollment.
Tildacerfont is an investigational treatment not authorized for use in people outside the clinical trial.
For more information, go to: clarahealth.com/studies/cahmelia

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👍 Research Opportunity! All of Us

I just signed up for this because it may be helpful for researchers at the NIH and elsewhere to learn more about Cushing’s, cancer, whatever else they can learn from my history.
Over 35 years ago, I agreed to be a part of a study at NIH so they could learn more about Cushing’s.  I consider this to be a larger, easier part of what I did back then.

From my bio: https://cushingsbios.com/2018/10/28/maryo-pituitary-bio/

As luck would have it, NIH (National Institutes of Health, Bethesda, Maryland) was doing a clinical trial of Cushing’s. I live in the same area as NIH so it was not too inconvenient but very scary at first to think of being tested there. At that time I only had a choice of NIH, Mayo Clinic and a place in Quebec to do this then-rare pituitary surgery called a Transsphenoidal Resection. I chose NIH – closest and free. After I was interviewed by the Doctors there, I got a letter that I had been accepted into the clinical trial. The first time I was there was for 6 weeks as an inpatient. More of the same tests.

There were about 12 of us there and it was nice not to be alone with this mystery disease. Many of these Cushies (mostly women) were getting bald, couldn’t walk, having strokes, had diabetes. One was blind, one had a heart attack while I was there. Towards the end of my testing period, I was looking forward to the surgery just to get this whole mess over with. While I was at NIH, I was gaining about a pound a day!

The MRI still showed nothing, so they did a Petrosal Sinus Sampling Test. That scared me more than the prospect of surgery. (This test carries the risk of stroke and uncontrollable bleeding from the incision points.) Catheters were fed from my groin area to my pituitary gland and dye was injected. I could watch the whole procedure on monitors. I could not move during this test or for several hours afterwards to prevent uncontrollable bleeding from a major artery. The test did show where the tumor probably was located. Also done were more sophisticated dexamethasone suppression tests where drugs were administered by IV and blood was drawn every hour (they put a heplock in my arm so they don’t have to keep sticking me). I got to go home for a weekend and then went back for the surgery – the Transsphenoidal Resection. I fully expected to die during surgery (and didn’t care if I did) so I signed my will and wrote last letters to those I wanted to say goodbye to. During the time I was home just before surgery, a college classmate of mine (I didn’t know her) did die at NIH of a Cushing’s-related problem. I’m so glad I didn’t find out until a couple months later!

November 3, 1987, the surgeon, Dr. Ed Oldfield, cut the gum above my front teeth under my upper lip so there is no scar. He used tiny tools and microscopes. My tumor was removed successfully. In some cases (not mine) the surgeon uses a plug of fat from the abdomen to help seal the cut. Afterwards, I was in intensive care overnight and went to a neurology ward for a few days until I could walk without being dizzy. I had some major headaches for a day or two but they gave me drugs (morphine) for those. Also, I had cotton plugs in my nostrils. It was a big day when they came out. I had diabetes insipidus (DI) for a little while, but that went away by itself – thank goodness!

I had to use a foam product called “Toothies” to brush my teeth without hitting the incision. Before they let me go home, I had to learn to give myself an injection in my thigh. They sent me home with a supply of injectable cortisone in case my level ever fell too low (it didn’t). I was weaned gradually off cortisone pills (scary). I now take no medications. I had to get a Medic Alert bracelet. I will always need to tell medical staff when I have any kind of procedure – the effects of my excess cortisone will remain forever.

I went back to the NIH for several follow-up visits of a week each where they did all the blood and urine testing again. After a few years NIH set me free. Now I go to my “outside” endocrinologist every year for the dexamethasone suppression test, 24-hour urine and regular blood testing.

Health discoveries come from research. Research starts with you.

Join the largest and most inclusive health research initiative of its kind. You could help researchers find answers to the most pressing health questions.

The All of Us WEAR Study has begun!
As a part of this optional study, you could receive a new Fitbit® to wear. All of Us will be able to get the data the Fitbit collects to help researchers understand how behavior impacts health.

Want to help others, too?  Sign up at https://go.joinallofus.org/

📞 Cushing’s Disease Study

We have an opportunity for you to take part in a Cushing’s Disease study (IPS_4636) for Patients. Our project number for this study is IPS_4636.

Project Details:

  • Web- Camera Interview
  • There is a homework component
  • Interview is 75-minutes long
  • 125 Reward + 100 homework

Things to Note:

  • Patient study only, Caregivers please pass the link along
  • Unique links, please do not pass along for 2nd use
  • One Participant per household
  • Want to share this opportunity? Let us know and we can provide a new link
  • Preliminary questions are Mobile Friendly!
  • Save this email to reference if you have any questions about the study!
  • If you have any problems, email pm3@rarepatientvoice.com and reference the project number.  If you hit reply, you will get an auto do-not-reply email.

If you are interested in this study, please click the link below to answer a few questions to see if you qualify.

Study Link: Link

Thanks as always for your participation! Please be aware that by entering this information you are not guaranteed that you will be selected to participate. As always, we do not share any of your contact information without your permission.

💉 CLINICAL TRIALS | Awareness Project

Health Condition: All Conditions

Demographics: Ages 18+, United States Resident

Special Request(s): Everyone who has taken part in a clinical trial is asked to share. We are interested in learning why you decided to take part in a clinical trial and how your experience went.

Honoraria: Some respondents may be asked to participate in a clinical trial awareness network where there are paid opportunities to tell their stories.

 Apply to learn more.

😷 Clinical Trial: Endogenous Cushing’s Syndrome Patients Sought for Phase 3 Trial Testing Relacorilant

Corcept Therapeutics is recruiting participants for its Phase 3 clinical trial evaluating relacorilant as a potential treatment for Cushing’s syndrome-related side effects such as high blood pressure and impaired glucose tolerance.

Also, findings from the study “A Randomized-Withdrawal, Placebo-Controlled, Phase 3 Study to Assess the Efficacy and Safety of Selective Glucocorticoid Receptor Antagonist, Relacorilant, in Patients with Cushing Syndrome (GRACE Study),” were presented at the 2019 Annual Meeting of the Endocrine Society (ENDO), in New Orleans, Louisiana.

In endogenous Cushing’s syndrome there is an “internal” culprit — usually a benign tumor — that makes the body produce too much of the hormone cortisol. The excessive amount of circulating cortisol can lead to serious problems, such as type 2 diabetes and high blood pressure.

Relacorilant is designed to prevent the effects of excess cortisol by blocking one of its receptors, the glucocorticoid receptor. Results from a Phase 2 trial (NCT02804750) suggest that relacorilant may manage the effects of prolonged cortisol excess in Cushing’s patients faster and without the known side effects of approved medications like Korlym (mifepristone).

Also, the treatment improved glucose tolerance and improved blood pressure in patients, suggesting it could be used to treat those with endogenous Cushing’s syndrome and concurrent type 2 diabetes mellitus, impaired glucose tolerance, and/or uncontrolled high blood pressure (hypertension).

Corcept has now designed the GRACE Phase 3 trial (NCT03697109), a multicenter, double-blind, placebo-controlled, randomized-withdrawal study, to evaluate relacorilant’s safety and effectiveness in these patients.

GRACE will be conducted in two stages. First, all patients will be given oral relacorilant each day for 22 weeks, at doses rising from 100 mg to a maximum of 400 mg.

Those who complete that stage and show improvements in pre-specified parameters of glucose tolerance or hypertension will move into the second, randomized phase of the trial.

Here, they will be randomly assigned to placebo or relacorilant at the same dose they received at the end of the first stage. This new round of treatment will last 12 weeks. Treatment-related adverse events (side effects) also will be assessed for up to 48 weeks (about 11 months) as a main outcome.

Additional primary goals include changes in glucose tolerance and blood pressure between the end of the first and second stages of the study.

Secondary objectives include identifying the proportion of patients achieving a response in glucose tolerance and high blood pressure criteria and the proportion of those who worsened at the end of the first stage, and the changes in quality of life throughout the study.

Researchers plan to enroll 130 people in these U.S. cities: Indianapolis, Indiana; Metairie, Louisiana; Jackson, Mississippi; Albany, New York; Jamaica, New York; Wilmington, North Carolina; Miami, Florida; Summerville, South Carolina; El Paso, Texas; Oklahoma City, Oklahoma, and; Aurora, Colorado. More detailed information is available here.

“We look forward to presenting new findings concerning cortisol modulation in patients with hypercortisolism,” Joseph K. Belanoff, MD, Corcept’s CEO,  said in a press release.

📺 NIH: Discovery Channel’s Documentary Series ‘First In Human’

Unprecedented Access Inside the National Institutes of Health’s Building 10
Premieres August 10

Directed by Emmy® Winner John Hoffman; Produced by McGee Media

Near the nation’s capital, on the campus of the National Institutes of Health, sits Building 10: the largest hospital in the world devoted solely to research. Inside, our country’s most brilliant scientific minds carry out some of medicine’s riskiest and most critical research, testing new treatments in people for the very first time.

With unprecedented access to the halls of Building 10, First in Human reveals for the first time how the medical breakthroughs of tomorrow make their way out of the hi-tech research laboratories and into the hands of our world’s medical professionals. The series explores the lives of the doctors, researchers, and patients who together make progress possible in this cutting-edge testing ground. Narrated by Emmy,® Golden Globe®, and Critics Choice® winning actor Jim Parsons (“The Big Bang Theory,” “Hidden Figures,”) directed by Emmy winner John Hoffman, and executive produced by Hoffman and Emmy winner Dyllan McGee, the three-part documentary series begins airing in August 10 at 9pm ET/PT exclusively on Discovery.

Because the treatments they’re testing are so new and their outcomes are entirely unknown, the doctors leading first in human trials at Building 10 can only partner with patients who have exhausted the options the medical establishment has to offer. This doctor-patient partnership is utterly unique to medicine: live or die, each of these brave “first in human” volunteers immediately becomes part of medical history. Previous trials in the building led to the development of modern chemotherapy treatments, the first treatments for HIV/AIDS, and the first successful gene therapy.

First In Human represents the first time cameras have embedded in Building 10 and followed first in human patients throughout their entire trial. This unique access is the product of Hoffman’s nearly twenty years of filmmaking in partnership with the NIH on projects such as The Alzheimer’s Project and The Weight of the Nation.

“NIH’s Building 10 has given generations hope when they need it most,” comments Rich Ross, Group President, Discovery Channel, Animal Planet, Science Channel, and Velocity.  “We are proud that the NIH has invited Discovery inside their doors for an unvarnished look at how these brave men and women work hand in hand with their doctors and technologists to seek cures to the most confounding diseases that exist.”

“Because of the very nature of first in human trials, most of the patients who enter Building 10 to enroll in them are remarkably unique: all standard care out in the world has failed them,” says Hoffman. “What was most powerful about embedding in Building 10 and following these trials was observing how the doctors and patients came to rely on each other in a true partnership to advance medicine.”

States Parsons, “I know that everyone who watches First in Human will feel the same sense of pride I did when I discovered this incredible institution that our country created. I hope viewers will share the sense of gratitude and awe that I felt when learning about the human beings who bravely put their lives in the hands of some of our most innovative scientists and doctors as they search together for the medicines and cures that give all of us fuller, longer lives. This is truly the story of how we, as human beings, function: both at an elemental level and at our most profound.”

“The NIH Clinical Center’s more than 60-year history has resulted in remarkable medical advances, from the first use of chemotherapy to treat cancer, to the development of the technique to keep the blood supply clean and safe from viruses,” said NIH Director Francis S. Collins, M.D., Ph.D. “For millions of patients around the world, it is known as the National Institutes of Hope.”

From leukemia to sickle cell disease to the rarest diseases on the planet, First in Human captures the stories of the doctors, researchers, staff, and, most importantly, the patients and families in this remarkable facility that together are pushing far into the unknown.

FIRST IN HUMAN is produced by McGee Media for Discovery Channel.  The series is directed by John Hoffman; produced by, John Hoffman, Beth Wichterich, and Michael Epstein; narrated by, Jim Parsons; executive producers Dyllan McGee, Jim Parsons, Todd Spiewak, and Eric Norsoph; producer, Jon Bardin; supervising producer, Stacia Thompson; senior editor, Adriana Pacheco; director of photography, Simon Schneider.  For Discovery Channel, supervising producer, Jon Bardin; executive producer, John Hoffman.

 

From https://corporate.discovery.com/discovery-newsroom/jim-parsons-set-to-narrate-discovery-channels-documentary-series-first-in-human-the-trials-of-building-10/

Help Advance Research for Better Cushing’s Syndrome Treatment

clinical-trials

 

I am so passionate about Clinical Trials, especially for Cushing’s because I was only diagnosed in 1987 because I was a part of a clinical trial at the NIH.  In addition to helping myself, I knew I’d be helping other Cushies coming along after me – something positive I could do while I was at my worst.

I hope that others will consider doing Clinical Trials, if they qualify for them.  You never know who else you might help!

This trial is testing the safety and effectiveness of an investigational drug for the treatment of Cushing’s Syndrome. Under the supervision of qualified physicians, cortisol levels and symptoms of Cushing’s Syndrome will be closely followed along with any signs of side effects.

More about the study:

The study drug (COR-003) is administered by tablets.

  • There will be 90 participants in this trial
  • There is no placebo used in the trial

If you are interested, please find the full study details and eligibility criteria listed here.

Eligibility Criteria:

Participants must:

  • be at least 18 years old
  • have been diagnosed with endogenous Cushing’s Syndrome by a medical professional (not caused by the use of steroid medications)

Participants must not:

  • have been treated with radiation for Cushing’s Syndrome in the past 4 years
  • be currently using weight loss medication
  • have been diagnosed with uncontrolled hypertension, some forms of cancer, adrenal carcinoma, Hepatitis B / C, or HIV

Please complete the online questionnaire to check if you’re eligible for the trial.

If you’re not familiar with clinical trials, here are some FAQs:

What are clinical trials?

Clinical trials are research studies to determine whether investigational drugs or treatments are safe and effective for humans. All new investigational medications and devices must undergo several clinical trials, often involving thousands of people.

Why participate in a clinical trial?

You will have access to investigational treatments that would be available to the general public only upon approval. You will also receive study-related medical care and attention from clinical trial staff at research facilities. Clinical trials offer hope for many people and an opportunity to help researchers find better treatments for others in the future.

Learn why I’m talking about Clinical Trials