The Safety and Efficacy of Rapamycin on Communicating Hydrocephalus Secondary to Intraventricular Hemorrhage

Trial Title: The Safety and Efficacy of Rapamycin on Communicating Hydrocephalus Secondary to Intraventricular Hemorrhage

NCT ID: NCT06563817

Condition: Communicating Hydrocephalus
Cerebral Intraventricular Hemorrhage
Secondary Normal Pressure Hydrocephalus
Post Hemorrhagic Hydrocephalus

Conditions: Official terms:
Neoplasm Metastasis
Hydrocephalus
Cerebral Hemorrhage
Hydrocephalus, Normal Pressure
Cerebral Intraventricular Hemorrhage
Hemorrhage
Sirolimus

Conditions: Keywords:
Communicating Hydrocephalus
Cerebral Intraventricular Hemorrhage
Rapamycin
Secondary Normal Pressure Hydrocephalus
Post Hemorrhagic Hydrocephalus

Study type: Interventional

Study phase: Phase 2

Overall status: Recruiting

Study design:

Allocation: N/A

Intervention model: Single Group Assignment

Intervention model description: All enrolled patients receive treatment with sirolimus (rapamycin), administered in capsule form at a dosage of 0.5 mg per capsule. The capsules, provided by North China Pharmaceutical under the trade name Yixinke, were stored at room temperature. The prescribed regimen involved a daily oral dosage of 1.5 mg for a duration of four weeks.

Primary purpose: Treatment

Masking: None (Open Label)

Intervention:

Intervention type: Drug
Intervention name: Rapamycin
Description: All enrolled patients receive treatment with sirolimus (rapamycin)#The prescribed regimen involved a daily oral dosage of 1.5 mg for a duration of four weeks.
Arm group label: Rapamycin treatment group

Other name: Sirolimus

Summary: This prospective, multicenter, open-label clinical trial is designed to evaluate the safety and efficacy of rapamycin in the treatment of communicating hydrocephalus secondary to intraventricular hemorrhage. Additionally, the underlying pathogenic mechanisms associated with this particular type of hydrocephalus will be investigated in greater depth, and populations that may benefit from rapamycin therapy will be identified.

Detailed description: Communicating hydrocephalus secondary to intraventricular hemorrhage is a serious neurological disorder with the main clinical manifestations of ventricular dilatation, gait disturbance, cognitive dysfunction, and urinary incontinence. At present, the sole treatment option for these patients is cerebrospinal fluid shunting. However, complications resulting from this therapy have necessitated multiple surgeries for some patients, which has a significant impact on their quality of life and financial resources. However, recent studies have identified the PI3K-AKT-mTOR pathway as a key contributor to the sequelae of hemorrhagic hydrocephalus. Furthermore, these studies demonstrated that rapamycin, an inhibitor of the PI3K-AKT-mTOR pathway, inhibited cerebrospinal fluid secretion and ventricular dilation in an animal model of hemorrhagic hydrocephalus sequelae. In light of these findings, we propose a prospective, multicenter, open-label clinical trial to evaluate the efficacy and safety of rapamycin in the treatment of communicating hydrocephalus secondary to intraventricular hemorrhage. The study design was that of a prospective, multicenter, open-label clinical trial. All patients were administered sirolimus (rapamycin) in a dosage of 0.5 mg per capsule. The capsules were provided by the North China Pharmaceutical Company and were stored at room temperature. The treatment course was four weeks, with a dosage of 1.5 mg orally per day. Efficacy and adverse effects were assessed at two weeks, four weeks, the end of treatment, and 12 weeks after the end of treatment, respectively.

Criteria for eligibility:
Criteria:
Inclusion Criteria: 1. Patients with ventricular dilatation due to intraventricular hemorrhage who clinically present with any one or more of new gait disturbances, cognitive deficits, and urinary incontinence after remission of intraventricular hemorrhage symptoms, and whose brain imaging shows an Evans index (EI) of ≥0.3 2. Age ≥ 18 years and ≤ 70 years 3. Signed informed consent form Exclusion Criteria: 1. Participation in another medical trial 2. Have other disease that may affect the patient's symptoms (including gait disturbance, cognitive impairment, urinary incontinence) 3. Allergy to the investigational drug 4. Reduced liver function (increased INR or alanine transaminase concentrations in plasma elevated more than 1.5 times reference values) 5. Reduced kidney function with GFR < 50 6. Concomitant treatment with strong CYP3A4/5 inducers or inhibitors, such as diltiazem, ketoconazole, or rifampicin. 7. Active or uncontrolled chronic infection 8. Women who are pregnant or breastfeeding 9. Patients who are bedridden or require urinary catheters for extended periods of time.

Gender: All

Minimum age: 18 Years

Maximum age: 70 Years

Healthy volunteers: No

Locations:

Facility:
Name: Beijing Tiantan Hospital, Capital Medical University

Address:
City: Beijing
Zip: 100070
Country: China

Status: Recruiting

Contact:
Last name: Runfa Tian, MD

Phone: 15910996812

Phone ext: +86
Email: trftc@126.com

Contact backup:
Last name: Guoyi Gao, MD

Phone: 13801874393

Phone ext: +86
Email: gao3@sina.com

Start date: August 2024

Completion date: July 2025

Lead sponsor:
Agency: Beijing Tiantan Hospital
Agency class: Other

Source: Beijing Tiantan Hospital

Record processing date: ClinicalTrials.gov processed this data on November 12, 2024

Source: ClinicalTrials.gov page: https://clinicaltrials.gov/ct2/show/NCT06563817