AzTrials
AzTrials

Congenital HyperinsulinismPhase 2NCT03053284

Pasireotide in Hyperinsulinemic Hypoglycemia

A Phase 2 study testing Pasireotide 0.6Mg Solution for Injection and Saline Solution in people with Congenital Hyperinsulinism and Insulinoma, run by Montefiore Medical Center.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
Search all trials

Trial summary

This is a small controlled pilot study to assess the effect of subcutaneous pasireotide on preventing hypoglycemia due to hyperinsulinism, including congenital hyperinsulinism and insulinoma. Read more about this study

Who is it for?
Ages 18 to 70 years
What is being tested?
Pasireotide 0.6Mg Solution for Injection and Saline Solution
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
0 participants
Where
No sites listed yet
Study dates
Apr 2017to Apr 2018
Phase
Phase 2
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 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Male or female patients aged 18 to 70 years old
  • Patients with hyperinsulinemic hypoglycemia due to either congenital hyperinsulinemic hypoglycemia or insulinoma, as determined by an endocrinologist
  • If no prior diagnosis of either insulinoma or congenital hyperinsulinemic hypoglycemia by an endocrinologist, the participant must meet the following criteria:
  • A history of symptoms of hypoglycemia, (with or without a blood glucose <50mg/dL at time of symptoms)
  • Improvement of symptoms with ingestion of carbohydrates
  • At least one documented blood glucose <50mg/dL with concomitant insulin >3 mmol/L and c-peptide >0.2nmol/L, with a negative sulfonylurea screen
Show all 16Show fewer
  • At least 1 episode of glucose <50mg/dL in the last year
  • Written informed consent obtained prior to treatment to be consistent with local regulatory requirements
  • No evidence of significant liver disease:
  • Serum total bilirubin < 2 x ULN
  • INR < 1.3 unless on anticoagulation
  • ALT and AST < 2 x ULN
  • Alkaline phosphatase < 2.5 x ULN
  • Patients receiving anti-hypoglycemic treatment are eligible
  • Patients who are treatment naïve, or those who were previously, but not currently, treated with anti-hypoglycemic therapy are also eligible
  • Patients with insulinoma who are operative candidates are eligible if surgery is not emergently needed, and study participation would not delay the timing of a surgical intervention

Who cannot join

  • Age <18, age >70 (for both insulinoma and congenital hyperinsulinism)
  • Known hypersensitivity to somatostatin or analogues
  • Diabetic patients with poor glycemic control as evidenced by HbA1c >8%
  • Patients who are hypothyroid and not on adequate replacement therapy
  • Patients with symptomatic cholelithiasis and acute or chronic pancreatitis
  • QTcF at screening > 450 msec in males and QTcF > 460 msec in females
Show all 18Show fewer
  • Hypokalaemia, hypomagnesaemia, family history of long QT syndrome or concomitant medications with known risk of Torsades de pointes (TdP)
  • Patients who have congestive heart failure (NYHA Class III or IV), unstable angina, sustained ventricular tachycardia, clinically significant bradycardia, advanced heart block, history of acute MI less than one year prior to study entry or clinically significant impairment in cardiovascular function
  • Severe non-malignant medical illness that may be jeopardized by treatment with a single dose of pasireotide
  • History of another primary malignancy, with the exception of locally excised non-melanoma skin cancer and carcinoma in situ of uterine cervix unless there is no evidence of disease in the last year
  • Patients with serum creatinine >2.0 X ULN
  • Patients with WBC <3 X 109/L; Hb 90% < LLN; PLT <100 X 109/L
  • Patients with the presence of active or suspected acute or chronic uncontrolled infection
  • Patients who have undergone major surgery/surgical therapy for any cause within 4 weeks prior screening
  • History of unexplained syncope or family history of idiopathic sudden death
  • Sexually active males unless they use a condom during intercourse while taking drug and for 3 months following last dose of pasireotide and 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.
  • Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test
  • 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 and 30 days following last dose of pasireotide.

About this study

Official title: Pasireotide for Prevention of Hypoglycemia in Patients With Hyperinsulinemic Hypoglycemia

This is a small controlled pilot study to assess the effect of subcutaneous pasireotide on preventing hypoglycemia due to hyperinsulinism, including congenital hyperinsulinism and insulinoma.

Read the detailed description

Pasireotide is a somatostatin analog with affinity for several somatostatin receptors including those on pancreatic beta cells; when activated these receptors affect the secretion of glucagon and insulin. Pasireotide is also known to decrease glucagon-like peptide 1 (GLP-1) and gastric inhibitory polypeptide (GIP) secretion. Hyperglycemia is a well-documented adverse effect of pasireotide in its approved indications for treatment of Cushing's disease and acromegaly.

In light of this, the investigators hypothesize that pasireotide may be an effective therapy for hypoglycemia due to hyperinsulinism. Therefore a small controlled pilot study to assess the effect of subcutaneous (s.c.) pasireotide on preventing hypoglycemia due to hyperinsulinism over 7 hours of observation in both fasting and fed states is proposed.

Being tested
Pasireotide 0.6Mg Solution for Injection, Saline Solution
Medical subject headings
Congenital Hyperinsulinism, Insulinoma, Hyperinsulinism

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Pasireotide 0.6Mg Solution for InjectionDrug

    Also called SOM230

    Pasireotide 0.6Mg Solution for Injection will be given once per study visit

  • Saline SolutionDrug

    Also called Placebo

    Saline Solution injection will be given once per study visit

Study groups in NCT03053284
Study groupRoleWhat they receive
PlaceboPlacebo comparatorNormal saline s.c. injection once
Receives: Drug: Saline Solution
PasireotideExperimentalPasireotide 0.6mg s.c. once
Receives: Drug: Pasireotide 0.6Mg Solution for Injection

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Hypoglycemia

    Occurence, frequency and severity of hypoglycemia (serum glucose < 55 mg/dL)

    Measured: 7 hours

Secondary outcomes (1)

  1. Serum glucose regulators

    Insulin, GLP-1, glucagon and cortisol levels

    Measured: 7 hours

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Feb 2017
  2. Study startApr 2017 (estimated)
  3. Primary completionJan 2018 (estimated)
  4. Study completionApr 2018 (estimated)

Why it stopped: Funding was withdrawn by Sponsor prior to start of study

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Crossover
Masking
Single blind
Who is masked
Participant
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03053284
Sponsor's protocol ID
2016-7044
Status last verified
May 2021
First submitted
9 Feb 2017
Last updated
14 May 2021
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Schmid HA, Brueggen J. Effects of somatostatin analogs on glucose homeostasis in rats. J Endocrinol. 2012 Jan;212(1):49-60. doi: 10.1530/JOE-11-0224. Epub 2011 Oct 10.

    PubMed 21987782

  • Braun M. The somatostatin receptor in human pancreatic beta-cells. Vitam Horm. 2014;95:165-93. doi: 10.1016/B978-0-12-800174-5.00007-7.

    PubMed 24559918

  • Boscaro M, Ludlam WH, Atkinson B, Glusman JE, Petersenn S, Reincke M, Snyder P, Tabarin A, Biller BM, Findling J, Melmed S, Darby CH, Hu K, Wang Y, Freda PU, Grossman AB, Frohman LA, Bertherat J. Treatment of pituitary-dependent Cushing's disease with the multireceptor ligand somatostatin analog pasireotide (SOM230): a multicenter, phase II trial. J Clin Endocrinol Metab. 2009 Jan;94(1):115-22. doi: 10.1210/jc.2008-1008. Epub 2008 Oct 28.

    PubMed 18957506

  • Yorifuji T. Congenital hyperinsulinism: current status and future perspectives. Ann Pediatr Endocrinol Metab. 2014 Jun;19(2):57-68. doi: 10.6065/apem.2014.19.2.57. Epub 2014 Jun 30.

    PubMed 25077087

  • de Heide LJ, Laskewitz AJ, Apers JA. Treatment of severe postRYGB hyperinsulinemic hypoglycemia with pasireotide: a comparison with octreotide on insulin, glucagon, and GLP-1. Surg Obes Relat Dis. 2014 May-Jun;10(3):e31-3. doi: 10.1016/j.soard.2013.11.006. Epub 2013 Dec 4. No abstract available.

    PubMed 24448101

  • Quinn TJ, Yuan Z, Adem A, Geha R, Vrikshajanani C, Koba W, Fine E, Hughes DT, Schmid HA, Libutti SK. Pasireotide (SOM230) is effective for the treatment of pancreatic neuroendocrine tumors (PNETs) in a multiple endocrine neoplasia type 1 (MEN1) conditional knockout mouse model. Surgery. 2012 Dec;152(6):1068-77. doi: 10.1016/j.surg.2012.08.021. Epub 2012 Oct 24.

    PubMed 23102680

  • Tirosh A, Stemmer SM, Solomonov E, Elnekave E, Saeger W, Ravkin Y, Nir K, Talmor Y, Shimon I. Pasireotide for malignant insulinoma. Hormones (Athens). 2016 Apr;15(2):271-276. doi: 10.14310/horm.2002.1639.

    PubMed 26732164

  • Eigler T, Ben-Shlomo A. Somatostatin system: molecular mechanisms regulating anterior pituitary hormones. J Mol Endocrinol. 2014 Aug;53(1):R1-19. doi: 10.1530/JME-14-0034. Epub 2014 Apr 29.

    PubMed 24780840

  • Hansen L, Hartmann B, Mineo H, Holst JJ. Glucagon-like peptide-1 secretion is influenced by perfusate glucose concentration and by a feedback mechanism involving somatostatin in isolated perfused porcine ileum. Regul Pept. 2004 Apr 15;118(1-2):11-8. doi: 10.1016/j.regpep.2003.10.021.

    PubMed 14759551

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Pasireotide 0.6Mg Solution for Injection and Saline Solution in people with Congenital Hyperinsulinism and Insulinoma, run by Montefiore Medical Center.

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 16 inclusion and 18 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: withdrawn, as of the sponsor's last update on 14 May 2021. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

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.

What does the primary outcome measure?

Hypoglycemia (time frame: 7 hours). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03053284, which also lists 9 linked publications.

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 14 May 2021.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Keep searching with a free account

You’ve used your 3 free searches for this session. Log in or create a free AzTrials account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in