A Phase 2 Study of Sonodynamic Therapy Using SONALA-001 and Exablate 4000 Type 2.0 in Patients wi… (NCT05123534) | Clinical Trial Compass
SuspendedPhase 2
A Phase 2 Study of Sonodynamic Therapy Using SONALA-001 and Exablate 4000 Type 2.0 in Patients with DIPG
Stopped: The study is suspended due to lack of funding but not due to safety concerns.
United States15 participantsStarted 2022-08-12
Plain-language summary
The primary objectives of this trial are to evaluate the safety and tolerability of sonodynamic therapy (SDT) using SONALA-001 and Exablate Type 2.0 device and to determine the maximum tolerated dose (MTD) or recommended phase 2 dose (RP2D) of MR-Guided Focused Ultrasound (MRgFUS) energy in combination with SONALA-001 in subjects with diffuse intrinsic pontine glioma
Funding Source - FDA OOPD
Who can participate
Age range5 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Newly diagnosed, radiographically typical DIPG, defined as a tumor with a pontine epicenter and diffuse involvement of more than ⅔ of the pons and without evidence of dissemination, are eligible with or without histologic confirmation.
✓. Prior treatment consisting of a minimum of 54 Gy standard focal radiotherapy administered over 42-49 days.
✓. Must be ≥ 4 weeks and ≤ 24 weeks post radiotherapy and must have recovered from acute effects to CTCAE (version 5) Grade 1 or baseline prior to Day 1.
✓. Must have stable to improved imaging by RAPNO (subjects ≤ 21 years of age) and mRANO (subjects \> 21 years of age) criteria comparing the scan obtained post radiotherapy to the scan obtained during the screening period. The comparison of diagnosis imaging scan and screening imaging scan must be reviewed by the study site's neuroradiologist and the principal investigator. If subjects present within 3 months after completion of radiotherapy and the comparison of the diagnosis scan to the screening scan is consistent with RAPNO/mRANO progression but pseudoprogression is a consideration, then the subject can be re-screened if the subject falls out of the screening window.
✓. Subjects who are on steroids must be on a stable to decreasing dose of steroids (maximum dexamethasone of 1 mg/m2/day) prior to Day 1.
✓. Minimum of 5 years of age. Subjects younger than 5 years old may be eligible after discussion with the Sponsor Medical Monitor/designee.
What they're measuring
1
To evaluate the safety and tolerability of SDT in DIPG subjects to generate data that will aid in the design of a larger Phase 2b trial.
Timeframe: Day 1 (SDT Study Treatment Day) to 12 months post-treatment
2
To determine the Maximum Tolerated Dose (MTD) or Recommended Phase 2 Dose (RP2D) of MR-Guided Focused Ultrasound (MRgFUS) energy in combination with SONALA-001 (SDT) in subjects with DIPG.
✓. A minimum head circumference of 52 cm as measured by physical exam. Subjects with a minimum head circumference smaller than 52 cm may be eligible after discussion with the Sponsor Medical Monitor/designee.
✓. Females of childbearing potential (FOCP) must have a negative serum or urine pregnancy test at screening. Subjects of childbearing or child fathering potential must be willing to use highly effective birth control during the entire study. Acceptable forms of birth control include hormonal contraceptives (oral, injectable, transdermal, or intravaginal) or intrauterine device (IUD) for at least one week prior to ALA SDT, condom and spermicidal or diaphragm and spermicidal. Other acceptable forms of birth control include a) abstinence for subjects who are not sexually active; or b) if the subject is in a monogamous relationship with a partner who is sterile (e.g., vasectomy performed at least 6 months prior to subject's ALA SDT treatment). Subjects who become sexually active or begin to have relations with a partner who is not sterile during the trial must agree to use an effective form of birth control for the duration of the study. FOCP taking hormonal therapy must be on treatment for at least 12 weeks prior to study entry and must not change their dosing regimen during the study.
Exclusion criteria
✕. Evidence of progressive disease by radiologic criteria (RAPNO for subjects ≤ 21 years of age or mRANO for subjects \> 21 years of age).
✕. Increasing steroid dose prior to Day 1.
✕. Diagnosis of porphyria.
✕. Hypersensitivity against porphyrins.
✕. Current malignancies or relevant history of other malignancy, physical, or psychiatric illness, any medical disorder that may require treatment or make the subject unlikely to fully complete the study, or any condition that presents undue risk from the investigational product or procedures.
✕. Known history of human immunodeficiency virus (HIV), hepatitis B or C infection, or any active systemic infection.
✕. Use of potentially phototoxic substances (e.g., St. John's wort, griseofulvin, thiazide diuretics, sulfonylureas, phenothiazines, tetracyclines, sulfonamides, quinolones, hypericin extracts, topical preparations containing ALA) within 24 hours before and after SONALA-001 infusion.
✕. Prior or concurrent therapy with any other anticancer (including radiotherapy, with the exception of radiotherapy administered per inclusion criteria #2 and #3) or investigational drug or other investigational intervention. NOTE: The use of bevacizumab or similar agent to control radiation therapy-induced edema is allowed for up to a maximum of 5 doses. The last dose must have been administered no later than 14 days prior to Day 1. See Section 6 (Prior and Concomitant Treatment) for a list of prohibited and restricted medications.