Congenital HyperinsulinismNCT02835131
Compassionate Use of SOM230 for Hyperinsulinemic/Hypoglycemia
A study testing Pasireotide in people with Congenital Hyperinsulinism, run by Montefiore Medical Center at 1 site in United States.
Trial summary
Congenital hyperinsulinism is a rare condition that can cause life-threatening hypoglycemia. Current treatment for congenital hyperinsulinism is often suboptimal, and such individuals may respond to a new somatostatin analog, pasireotide. Read more about this study
- Who is it for?
- Ages 18 to 90 years
- Conditions the study is for
- Congenital Hyperinsulinism
- What is being tested?
- Pasireotide
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 90 years
- All sexes
- Adult, Older adult
Who may be able to join
- Male or female patients aged 18 years or older
- Patients with a confirmed diagnosis of hyperinsulinemic hypoglycemia, if possible by genetic testing
- Patients not controlled by medical therapies (e.g. diazoxide or octreotide) and/or pancreatic surgery or patients not eligible for surgery
- World Health Organization/ Eastern Cooperative Oncology Group Performance Status of 0-2.
- Life expectancy ≥12 weeks
- Adequate end organ function as defined by:
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- No evidence of significant liver disease:
- Serum total bilirubin ≤1.5 x upper limit of normal (ULN)
- International Normalized Ratio (INR) < 1.3
- Alanine aminotransferase (ALT) and Aspartate aminotransferase (AST) ≤ 2 x ULN,
- Alkaline phosphatase ≤ 2.5 x ULN
- Written informed consent obtained prior to treatment to be consistent with local regulatory requirements
- Is suffering from a serious or life-threatening disease or condition
- Does not have access to a comparable or satisfactory alternative treatment (i.e., comparable or satisfactory treatment is not available or does not exist)
- Is not eligible for participation in any of the investigators ongoing clinical trials or has recently completed a clinical trial that has been terminated and, after considering other options (for example., trial extensions, amendments, etc.), the clinical team has determined that treatment is necessary and there are no other feasible alternatives for the patient
- There are meaningful human clinical data to support an assessment that the potential benefits to patient outweigh risks.
- Meets any other relevant medical criteria for compassionate use of the investigational product
- Is not being transferred from an ongoing clinical trial for which they are still eligible
Who cannot join
- Patients with a known hypersensitivity to somatostatin analogs or any component of the pasireotide long acting release (LAR) or subcutaneous. formulations.
- Patients with abnormal coagulation (prothrombin time or activated partial thromboplastin time elevated by 30% above normal limits).
- Patients on continuous anticoagulation therapy. Patients who were on anticoagulant therapy must complete a washout period of at least 10 days and have confirmed normal coagulation parameters before study inclusion.
- Patients currently using warfarin / warfarin derivatives
- Patients with symptomatic cholelithiasis.
- Patients who are not biochemically euthyroid. Patients with known history of hypothyroidism are eligible if they are on adequate and stable replacement thyroid hormone therapy for at least 3 months.
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- QT-related exclusion criteria: :
- corrected QT interval (QTcF) at screening > 450 msec in males and QTcF > 460 msec
- History of syncope or family history of idiopathic sudden death
- Sustained or clinically significant cardiac arrhythmias
- Risk factors for Torsades de Pointes such as hypokalemia, hypomagnesemia, cardiac failure, clinically significant/symptomatic bradycardia, or high-grade atrioventricular block
- Concomitant disease(s) that could prolong QT such as autonomic neuropathy (caused by diabetes, or Parkinson's disease), human immunodeficiency virus (HIV) infection, cirrhosis, uncontrolled hypothyroidism or cardiac failure
- Family history of long QT syndrome
- Concomitant medications known to prolong the QT interval.
- Potassium < or = 3.5 mmol/L
- Patients who have any severe and/or uncontrolled medical conditions :
- Uncontrolled diabetes as defined by hemoglobinA1c > 8%,
- Patients with the presence of active or suspected acute or chronic uncontrolled infection or with a history of immunodeficiency, including a positive HIV test result (ELISA and Western blot). An HIV test will not be required; however, previous medical history will be reviewed.
- Non-malignant medical illnesses that are uncontrolled or whose control may be jeopardized by the treatment with this study treatment.
- Life-threatening autoimmune and ischemic disorders.
- Patients who have a history of another primary malignancy, with the exception of locally excised non-melanoma skin cancer and carcinoma in situ of uterine cervix. Patients who have had no evidence of disease from another primary cancer for 1 or more years are allowed to participate in the study.
- Patients with history of liver disease, such as cirrhosis or chronic active hepatitis B or
- Presence of Hepatitis B surface antigen (HbsAg)
- Presence of Hepatitis C antibody (anti-HCV)
- History of, or current alcohol misuse/abuse within the past 12 months.
- Known gallbladder or bile duct disease, acute or chronic pancreatitis
- Patients with hypomagnesaemia (< 0.7 mmol/L)
- Patients with a history of non-compliance to medical regimens or who are considered potentially
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing. Highly effective contraception methods include:
- Total abstinence (when this is in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception
- Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy) or tubal ligation at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment
- Combination of any two of the following (a+b or a+c, or b+c):
- Use of oral, injected or implanted hormonal methods of contraception or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example hormone vaginal ring or transdermal hormone contraception.
- Placement of an intrauterine device (IUD) or intrauterine system (IUS)
- Barrier methods of contraception: Condom or Occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/vaginal suppository
- In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment.
- If the patient is a sexually active male he is excluded unless he agrees to use a condom during intercourse while taking pasireotide and for 3 months after stopping pasireotide medication . They should not father a child in this period. A condom is required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid
Trial locations and contacts
1 site in 1 country.
United States1 site
Montefiore Medical Center
The Bronx, New York, 10461
About this study
Official title: Compassionate Use of SOM230 for Individual Patient (NS, 14-Jan-1986) With Hyperinsulinemic/Hypoglycemia
Congenital hyperinsulinism is a rare condition that can cause life-threatening hypoglycemia. Current treatment for congenital hyperinsulinism is often suboptimal, and such individuals may respond to a new somatostatin analog, pasireotide. This is a compassionate use study of the effects of pasireotide on individuals with suboptimally treated congenital hyperinsulinism.
Read the detailed description
Congenital hyperinsulinism is a rare condition that can cause life-threatening hypoglycemia. Current treatment for congenital hyperinsulinism is often suboptimal, with diazoxide as the mainstay of treatment. Individuals who are not adequately treated with diazoxide may undergo pancreatectomy. Octreotide has been used with some success in congenital hyperinsulinism but there is limited data on pasireotide, the newest somatostatin receptor agonist, which, compared to octreotide, has 30-40 times greater affinity for somatostatin receptors 1 (SSTR1) and 5 (SSTR5), 5 times greater for somatostatin receptor 3 (SSTR3) and a comparable affinity for somatostatin receptor 2 (SSTR2).
In human islets, SSTR2 \& SSTR5 are present in beta cells. Therefore, there is reason to believe that pasireotide may have greater effectiveness than octreotide in preventing hypoglycemia due to hyperinsulinism. Indeed hyperglycemia is a known effect of pasireotide when used for treatment of Cushing's disease and Acromegaly. The current study is of compassionate use for prevention of hypoglycemia in individuals with congenital hyperinsulinemic hypoglycemia who are not adequately treated with diazoxide.
- Conditions
- Congenital Hyperinsulinism
- Being tested
- Pasireotide
- Keywords
- hyperinsulinemia, hypoglycemia, somatostatin, pasireotide, congenital
- Medical subject headings
- Congenital Hyperinsulinism, Hyperinsulinism, Hypoglycemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PasireotideDrug
Also called Signifor, SOM230
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jul 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital HyperinsulinismStudies of the same condition, open and under way.
- More trials run by Montefiore Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Montefiore Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Vafa Tabatabaie, Montefiore Medical Center
- Investigators
- Vafa Tabatabaie, Principal investigator, Montefiore Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02835131
- Sponsor's protocol ID
- 2015-5564
- Status last verified
- Jul 2022
- First submitted
- 13 Jul 2016
- Last updated
- 29 Jul 2022
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Compassionate Use of SOM230 for Individual Patient (NS, 14-Jan-1986) With Hyperinsulinemic/Hypoglycemia
- ClinicalTrials.gov ID
- NCT02835131
- Acronym / sponsor ID
- 2015-5564
- Conditions
- Congenital Hyperinsulinism
- Keywords
- hyperinsulinemia, hypoglycemia, somatostatin, pasireotide, congenital
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Montefiore Medical CenterUniversity, hospital or other
- First posted
- 15 Jul 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Congenital hyperinsulinism is a rare condition that can cause life-threatening hypoglycemia. Current treatment for congenital hyperinsulinism is often suboptimal, and such individuals may respond to a new somatostatin analog, pasireotide. This is a compassionate use study of the effects of pasireotide on individuals with suboptimally treated congenital hyperinsulinism.
Detailed description
Congenital hyperinsulinism is a rare condition that can cause life-threatening hypoglycemia. Current treatment for congenital hyperinsulinism is often suboptimal, with diazoxide as the mainstay of treatment. Individuals who are not adequately treated with diazoxide may undergo pancreatectomy. Octreotide has been used with some success in congenital hyperinsulinism but there is limited data on pasireotide, the newest somatostatin receptor agonist, which, compared to octreotide, has 30-40 times greater affinity for somatostatin receptors 1 (SSTR1) and 5 (SSTR5), 5 times greater for somatostatin receptor 3 (SSTR3) and a comparable affinity for somatostatin receptor 2 (SSTR2).
In human islets, SSTR2 \& SSTR5 are present in beta cells. Therefore, there is reason to believe that pasireotide may have greater effectiveness than octreotide in preventing hypoglycemia due to hyperinsulinism. Indeed hyperglycemia is a known effect of pasireotide when used for treatment of Cushing's disease and Acromegaly. The current study is of compassionate use for prevention of hypoglycemia in individuals with congenital hyperinsulinemic hypoglycemia who are not adequately treated with diazoxide.
Study design
Who may qualify
- Ages
- 18 to 90 years
- Sex
- All
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- Male or female patients aged 18 years or older
- Patients with a confirmed diagnosis of hyperinsulinemic hypoglycemia, if possible by genetic testing
- Patients not controlled by medical therapies (e.g. diazoxide or octreotide) and/or pancreatic surgery or patients not eligible for surgery
- World Health Organization/ Eastern Cooperative Oncology Group Performance Status of 0-2.
- Life expectancy ≥12 weeks
Key exclusion criteria (37 listed)
- Patients with a known hypersensitivity to somatostatin analogs or any component of the pasireotide long acting release (LAR) or subcutaneous. formulations.
- Patients with abnormal coagulation (prothrombin time or activated partial thromboplastin time elevated by 30% above normal limits).
- Patients on continuous anticoagulation therapy. Patients who were on anticoagulant therapy must complete a washout period of at least 10 days and have confirmed normal coagulation parameters before study inclusion.
- Patients currently using warfarin / warfarin derivatives
- Patients with symptomatic cholelithiasis.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PasireotideAlso: Signifor, SOM230 | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jul 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Vafa Tabatabaie principal investigator
Montefiore Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Pasireotide in people with Congenital Hyperinsulinism, run by Montefiore Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 37 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 29 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jul 2022.