Tirzepatide for Pseudotumor Cerebri

(TIIHT Trial)

Overseen By Beth Perry, RN

Age: 18 - 65

Sex: Any

Trial Phase: Phase 4

Sponsor: Duke University

Must not be taking: GLP-1 agonists, Anti-obesity drugs

Disqualifiers: Bariatric surgery, Pancreatitis, Diabetes, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test tirzepatide, a medication, to determine if it can lower intracranial pressure (pressure inside the skull) in people with pseudotumor cerebri, a condition that mimics a brain tumor. Participants will receive either tirzepatide or a placebo (inactive treatment) to compare their effects over a year. The trial suits individuals experiencing symptoms like headaches and vision problems due to increased pressure, have a BMI of 30 or higher, and have not undergone specific surgical procedures for this condition. As a Phase 4 trial, the study involves an FDA-approved medication, focusing on understanding its benefits for more patients.

Do I need to stop my current medications for the trial?

You may need to stop certain medications before joining the trial. If you are taking another medication to lower intracranial pressure, you must stop it at least 30 days before enrolling. Also, you should not be taking another GLP-1 agonist or anti-obesity medication.

What is the safety track record for this treatment?

Previous studies have generally shown that tirzepatide is well-tolerated. Research indicates it can lower the risk of vision problems and stabilize or improve brain function. While some side effects have been reported, they are usually manageable. The FDA has already approved tirzepatide for other uses, suggesting its safety. Participants in the trial will be monitored for side effects, but existing evidence is promising for safety. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Unlike traditional treatments for pseudotumor cerebri, which often involve medications like acetazolamide or surgical interventions to reduce cerebrospinal fluid pressure, tirzepatide offers a unique approach. Tirzepatide is a dual GIP and GLP-1 receptor agonist, originally developed for diabetes management, that may help with weight loss and metabolic improvements, potentially addressing some underlying factors of pseudotumor cerebri. Researchers are excited about tirzepatide because it not only offers a novel mechanism of action but also provides the convenience of once-weekly dosing, which could improve patient adherence compared to the daily regimens of current options.

What evidence suggests that tirzepatide might be an effective treatment for pseudotumor cerebri?

Research has shown that tirzepatide, which participants in this trial may receive, may help with pseudotumor cerebri by lowering intracranial pressure (ICP). This treatment activates two specific receptors, GIP and GLP-1, potentially offering advantages over older treatments. In one study, patients with a difficult-to-treat form of this condition experienced a 23.2% reduction in risk after using tirzepatide for three months. GLP-1 receptor agonists, like tirzepatide, have also shown promise in reducing the need for other medications by aiding in weight loss. This is important because losing weight is often crucial in managing pseudotumor cerebri. ^{1 2 6 7 8}

Who Is on the Research Team?

Alexa N Bramall, MD

Principal Investigator

Duke University

Are You a Good Fit for This Trial?

This trial is for individuals with benign intracranial hypertension, also known as pseudotumor cerebri, which can cause headaches. Participants should not have other conditions that might interfere with the study.

Inclusion Criteria

BMI ≥30 kg/m2

Able to provide informed consent

Women of child-bearing age must use birth control (non-oral contraceptive method or add a barrier method of contraception).

Exclusion Criteria

I have type II diabetes.

I have had surgery or procedures to lower brain pressure.

My kidney function is severely reduced.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Tirzepatide or placebo weekly with escalating doses over 12 months

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Tirzepatide

Trial Overview The trial tests Tirzepatide against a placebo to see if it can reduce intracranial pressure over 12 months. Participants will be randomly assigned to either the drug or placebo group in equal numbers and won't know which they're receiving.

How Is the Trial Designed?

Group I: Tirzepatide Active Control 1 Intervention

Participants receive Tirzepatide weekly on the following dose schedule for a year: Dosage Treatment time 2.5mg/0.5mL: Month 1 (4 Weeks) 5mg/0.5mL: Month 2 (4 Weeks) 7.5mg/0.5mL: Month 3 (4 Weeks) 10mg/0.5mL: Month 4 (4 Weeks) 12.5mg/0.5mL: Month 5-6 15mg/0.5 mL: Month 7-12

Group II: Placebo Placebo Group 1 Intervention

Participants receive Tirzepatide Placebo weekly on the following dose schedule for a year: Dosage Treatment time 2.5mg/0.5mL: Month 1 (4 Weeks) 5mg/0.5mL: Month 2 (4 Weeks) 7.5mg/0.5mL: Month 3 (4 Weeks) 10mg/0.5mL: Month 4 (4 Weeks) 12.5mg/0.5mL: Month 5-6 15mg/0.5 mL: Month 7-12

Who Is Running the Clinical Trial?

Duke University

Lead Sponsor

Trials

2,495

Recruited

5,912,000+

Eli Lilly and Company

Industry Sponsor

Trials

2,708

Recruited

3,720,000+

Dr. Daniel Skovronsky

Eli Lilly and Company

Chief Medical Officer since 2018

MD from Harvard Medical School

David A. Ricks

Eli Lilly and Company

Chief Executive Officer since 2017

BSc from Purdue University, MBA from Indiana University

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39677436/

Efficacy of Tirzepatide Dual GIP/GLP-1 Receptor Agonist In ... Our results demonstrate that tirzepatide, when used as an adjunctive therapy, provides significant therapeutic benefits in IIH management.

2. medrxiv.org medrxiv.org/content/10.1101/2024.11.12.24317193v1.full.pdf

Efficacy of Tirzepatide Dual GIP/GLP-1 Receptor Agonist In ... Our findings suggest that dual GIP/GLP-1 receptor activation may offer advantages over traditional single-receptor therapies, potentially ...

3. aes.amegroups.org aes.amegroups.org/article/view/8489/html

Beyond weight loss: a case study and narrative review of the ... Key Content and Findings: GLP-1RAs have demonstrated the potential for significantly greater weight loss in patients with IIH, with a reduced requirement for ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT07191873

Tirzepatide in Idiopathic Intracranial Hypertension Trial This will be a randomized, double-blind, parallel, placebo-controlled trial of 60 participants. The primary analysis will be a statistical ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11643283/

Efficacy of Tirzepatide Dual GIP/GLP-1 Receptor Agonist In ... In cases of refractory IIH, traditionally challenging to manage, the tirzepatide group showed a 23.2% reduction in risk at three months (RR ...

6. thejournalofheadacheandpain.biomedcentral.com thejournalofheadacheandpain.biomedcentral.com/articles/10.1186/s10194-023-01631-z

Treatment with GLP-1 receptor agonists is associated with ... Treatment with GLP-1 receptor agonists is associated with significant weight loss and favorable headache outcomes in idiopathic intracranial ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12426903/

Efficacy and Safety of GLP‐1 and Dual GIP/GLP‐1 Receptor ... In this systematic review and meta‐analysis, treatment with GLP‐1 or dual GIP/GLP‐1 RAs was associated with a significantly reduced risk of ...

8. researchgate.net researchgate.net/figure/Outcomes-Risk-For-Tirzepatide-Group-Compared-to-Control-Group_tbl2_385784793

Outcomes Risk For Tirzepatide Group Compared to Control ... Several studies reported papilledema resolution and a lower risk of visual deterioration. Cognitive function either improved or remained stable. Adverse effects ...