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History of Changes for Study: NCT05123534
A Phase 1/2 Study of SONALA-001 and Exablate 4000 Type 2 in Patients With DIPG
Latest version (submitted November 3, 2022) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 November 5, 2021 None (earliest Version on record)
2 November 22, 2021 Sponsor/Collaborators, Study Status and Study Identification
3 March 28, 2022 Study Status
4 April 29, 2022 Recruitment Status, Study Status, Contacts/Locations and Oversight
5 June 7, 2022 Study Status and Contacts/Locations
6 August 4, 2022 Study Status and Contacts/Locations
7 August 12, 2022 Contacts/Locations and Study Status
8 October 13, 2022 Study Status
9 October 18, 2022 Study Description, Study Status and Study Identification
10 November 3, 2022 Eligibility and Study Status
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Study NCT05123534
Submitted Date:  November 5, 2021 (v1)

Open or close this module Study Identification
Unique Protocol ID: SDT-201
Brief Title: A Phase 1/2 Study of SONALA-001 and Exablate 4000 Type 2 in Patients With DIPG
Official Title: A Dose-escalation Study Examining the Safety, Pharmacokinetics, and Preliminary Efficacy of Ascending Drug and Energy Dose Combinations for Sonodynamic Therapy Using SONALA-001 in Combination With Exablate 4000 Type-2 MR-Guided Focused Ultrasound in Subjects With Diffuse Intrinsic Pontine Glioma
Secondary IDs:
Open or close this module Study Status
Record Verification: November 2021
Overall Status: Not yet recruiting
Study Start: February 2022
Primary Completion: May 2024 [Anticipated]
Study Completion: May 2025 [Anticipated]
First Submitted: October 11, 2021
First Submitted that
Met QC Criteria:
November 5, 2021
First Posted: November 17, 2021 [Actual]
Last Update Submitted that
Met QC Criteria:
November 5, 2021
Last Update Posted: November 17, 2021 [Actual]
Open or close this module Sponsor/Collaborators
Sponsor: SonALAsense, Inc.
Responsible Party: Sponsor
Collaborators: InSightec
Open or close this module Oversight
U.S. FDA-regulated Drug: Yes
U.S. FDA-regulated Device: Yes
Unapproved/Uncleared Device: Yes
Pediatric Postmarket Surveillance:
Data Monitoring:
Open or close this module Study Description
Brief 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 device and to 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
Detailed Description:
Open or close this module Conditions
Conditions: Diffuse Intrinsic Pontine Glioma
Keywords:
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 2
Interventional Study Model: Sequential Assignment
A Bayesian optimal interval (BOIN) dose escalation method will be used to determine the MTD-C of the drug SONALA-001 and device Exablate Type-2 combination.
Number of Arms: 9
Masking: None (Open Label)
Allocation: Non-Randomized
Enrollment: 18 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
Experimental: Cohort 1
5 mg/kg iv SONALA-001 (ALA) and 200J MR-guided Focused Ultrasound (MRgFUS)
Combination Product: SONALA-001 (ALA) and MR-Guided Focused Ultrasound device (MRgFUS)
SONALA-001(ALA) given 6-9 hours prior to receiving the MRgFUS.
Other Names:
  • Exablate Type-2
Experimental: Cohort 2
5 mg/kg iv SONALA-001 (ALA) and 400J MR-guided Focused Ultrasound (MRgFUS)
Combination Product: SONALA-001 (ALA) and MR-Guided Focused Ultrasound device (MRgFUS)
SONALA-001(ALA) given 6-9 hours prior to receiving the MRgFUS.
Other Names:
  • Exablate Type-2
Experimental: Cohort 3
5 mg/kg iv SONALA-001 (ALA) and 800J MR-guided Focused Ultrasound (MRgFUS)
Combination Product: SONALA-001 (ALA) and MR-Guided Focused Ultrasound device (MRgFUS)
SONALA-001(ALA) given 6-9 hours prior to receiving the MRgFUS.
Other Names:
  • Exablate Type-2
Experimental: Cohort 4
10 mg/kg iv SONALA-001 (ALA) and 200J MR-guided Focused Ultrasound (MRgFUS)
Combination Product: SONALA-001 (ALA) and MR-Guided Focused Ultrasound device (MRgFUS)
SONALA-001(ALA) given 6-9 hours prior to receiving the MRgFUS.
Other Names:
  • Exablate Type-2
Experimental: Cohort 5
10 mg/kg iv SONALA-001 (ALA) and 400J MR-guided Focused Ultrasound (MRgFUS)
Combination Product: SONALA-001 (ALA) and MR-Guided Focused Ultrasound device (MRgFUS)
SONALA-001(ALA) given 6-9 hours prior to receiving the MRgFUS.
Other Names:
  • Exablate Type-2
Experimental: Cohort 6
10 mg/kg iv SONALA-001 (ALA) and 800J MR-guided Focused Ultrasound (MRgFUS)
Combination Product: SONALA-001 (ALA) and MR-Guided Focused Ultrasound device (MRgFUS)
SONALA-001(ALA) given 6-9 hours prior to receiving the MRgFUS.
Other Names:
  • Exablate Type-2
Experimental: Cohort 7
15 mg/kg iv SONALA-001 (ALA) and 200J MR-guided Focused Ultrasound (MRgFUS)
Combination Product: SONALA-001 (ALA) and MR-Guided Focused Ultrasound device (MRgFUS)
SONALA-001(ALA) given 6-9 hours prior to receiving the MRgFUS.
Other Names:
  • Exablate Type-2
Experimental: Cohort 8
15 mg/kg iv SONALA-001 (ALA) and 400J MR-guided Focused Ultrasound (MRgFUS)
Combination Product: SONALA-001 (ALA) and MR-Guided Focused Ultrasound device (MRgFUS)
SONALA-001(ALA) given 6-9 hours prior to receiving the MRgFUS.
Other Names:
  • Exablate Type-2
Experimental: Cohort 9
15 mg/kg iv SONALA-001 (ALA) and 800J MR-guided Focused Ultrasound (MRgFUS)
Combination Product: SONALA-001 (ALA) and MR-Guided Focused Ultrasound device (MRgFUS)
SONALA-001(ALA) given 6-9 hours prior to receiving the MRgFUS.
Other Names:
  • Exablate Type-2
Open or close this module Outcome Measures
Primary Outcome Measures:
1. Safety of ALA SDT
[ Time Frame: Day 1 to 12 months ]

Tabulation of treatment-related adverse events
2. MTD
[ Time Frame: Day 1 to Day 29 ]

Frequency of DLTs in each Cohort
Secondary Outcome Measures:
1. Objective Response (RAPNO)
[ Time Frame: Day 0 to 12 months ]

Percentage of subjects achieving CR or PR.
2. Area under the plasma concentration vs time from Time 0 to the last measurable timepoint (AUC0-t) of ALA in blood plasma
[ Time Frame: Day 1 to 24 hours post SONALA-001 dosing ]

Samples will be collected at 10 time points during 24 hours pre/post SONALA-001 administration
3. Area under the plasma concentration vs time from Time 0 to infinity (AUC0-∞) of ALA in blood plasma
[ Time Frame: Day 1 to 24 hours post SONALA-001 dosing ]

Samples will be collected at 10 time points during 24 hours pre/post SONALA-001 administration
4. Terminal elimination half-life (t1/2) of ALA in blood plasma
[ Time Frame: Day 1 to 24 hours post SONALA-001 dosing ]

Samples will be collected at 10 time points during 24 hours pre/post SONALA-001 administration
5. elimination rate constant of ALA in blood plasma
[ Time Frame: Day 1 to 24 hours post SONALA-001 dosing ]

Samples will be collected at 10 time points during 24 hours pre/post SONALA-001 administration
6. Clearance of ALA in blood plasma
[ Time Frame: Day 1 to 24 hours post SONALA-001 dosing ]

Samples will be collected at 10 time points during 24 hours pre/post SONALA-001 administration
7. Time to maximum drug concentration (Tmax) of ALA in blood plasma
[ Time Frame: Day 1 to 24 hours post SONALA-001 dosing ]

Samples will be collected at 10 time points during 24 hours pre/post SONALA-001 administration
8. Area under the plasma concentration vs time from Time 0 to the last measurable timepoint (AUC0-t) of PpIX in blood plasma
[ Time Frame: Day 1 to 24 hours post SONALA-001 dosing ]

Samples will be collected at 10 time points during 24 hours pre/post SONALA-001 administration
9. Area under the plasma concentration vs time from Time 0 to infinity (AUC0-∞) of PpIX in blood plasma
[ Time Frame: Day 1 to 24 hours post SONALA-001 dosing ]

Samples will be collected at 10 time points during 24 hours pre/post SONALA-001 administration
10. Terminal elimination half-life (t1/2) of PpIX in blood plasma
[ Time Frame: Day 1 to 24 hours post SONALA-001 dosing ]

Samples will be collected at 10 time points during 24 hours pre/post SONALA-001 administration
11. elimination rate constant of PpIX in blood plasma
[ Time Frame: Day 1 to 24 hours post SONALA-001 dosing ]

Samples will be collected at 10 time points during 24 hours pre/post SONALA-001 administration
12. Clearance of PpIX in blood plasma
[ Time Frame: Day 1 to 24 hours post SONALA-001 dosing ]

Samples will be collected at 10 time points during 24 hours pre/post SONALA-001 administration
13. Time to maximum drug concentration (Tmax) of PpIX in blood plasma
[ Time Frame: Day 1 to 24 hours post SONALA-001 dosing ]

Samples will be collected at 10 time points during 24 hours pre/post SONALA-001 administration
14. Performance of MRgFUS
[ Time Frame: Day 1, Day 29 ]

Percentage of treatments in which the MRgFUS system works as planned.
15. Performance of MRgFUS
[ Time Frame: Day 1, Day 29 ]

Percentage of treatments in which procedure deviations were noted.
16. Progression-free survival
[ Time Frame: Day 1 to 12 months ]

Time from first dose of SONALA-001 to progression or death due to any cause.
17. Duration of response
[ Time Frame: Day 1 to 12 months ]

Time from date of first documented CR or PR until progression or death due to any cause.
18. Overall survival
[ Time Frame: Day 1 to 12 months ]

Time from first dose of SONALA-001 to death due to any cause.
Open or close this module Eligibility
Minimum Age: 5 Years
Maximum Age:
Sex: All
Gender Based:
Accepts Healthy Volunteers: No
Criteria:

Inclusion Criteria:

  1. Newly diagnosed, radiographically typical DIPG, defined as a tumor with a pontine epicenter and diffuse involvement of more than 2/3 of the pons and without evidence of dissemination, are eligible with or without histologic confirmation.
    • Subjects with pontine lesions that do not meet radiographic criteria will be eligible if there is histologic confirmation of DIPG.
    • Subjects may be asked to agree to provide access to previously obtained biopsy results.
    • If tumor status is unknown or archival tumor tissue is not available, subjects may be asked to agree to submit a post-mortem biopsy specimen to enable molecular profiling of tumor.
  2. Prior treatment consisting of a minimum of 54 Gy standard focal radiotherapy administered over 42-49 days.
  3. Must be ≥ 4 weeks and ≤ 16 weeks post radiotherapy and must have recovered from acute effects to CTCAE Grade 1 or baseline prior to Day 1.
  4. Must have stable to improved imaging by RAPNO criteria and be on a stable to decreasing dose of steroids (maximum dexamethasone of 1 mg/m^2/day) prior to Day 1, as obtained during the Screening period.
  5. A minimum head circumference of 52 cm. Subjects with a minimum head circumference of 52 cm, younger than 5 years old, may be eligible after discussion with the medical monitor.
  6. An Overall Skull Density Ratio greater than 0.45 (±0.05) as calculated from the screening CT.
  7. 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.
  8. Ability to provide written, signed, and dated (personally or via a legally authorized representative) informed consent/and assent at screening as applicable to participate in the study.
  9. Karnofsky Performance Scale (KPS for > 16 years of age) or Lansky Performance Score (LPS for ≤ 16 years of age) assessed within 14 days of Visit 1 must be ≥ 50%. Subjects who are unable to walk because of neurologic deficits, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
  10. Subjects must have adequate organ and marrow function as defined below:
    • Absolute neutrophil count ≥ 1,000/mm^3, without the use of GCSF within 7 days prior to Day 1.
    • Platelets ≥ 100,000/ mm^3 (unsupported, defined as no platelet transfusion within 7 days prior to Day 1)
    • Hemoglobin ≥ 8 g/dl (may have received last transfusion within 7 days prior to Day 1.)
    • Total bilirubin ≤ 1.5 times institutional upper limit of normal (ULN)
    • ALT(SGPT) < 3 x institutional ULN
    • AST(SGOT) < 3 x institutional ULN
    • Albumin ≥ 3 g/dl
    • Potassium ≥ lower limit of normal LLN
    • Serum total calcium (correct for serum albumin) or ionized calcium ≥ LLN
    • Renal Function as defined below:
    • Age 5-17 years: GFR ≥ 70 mL/min/1.73m2as estimated by Schwartz formula (Schwartz, 2009). If eGFR is abnormal for age based on Schwartz formula, accurate measurement should be obtained by 24 hour creatinine clearance.
    • Age ≥ 18 years: Creatinine clearance (CLcr) ≥ 60 mL/min/1.73m2as estimated by the Cockcroft-Gault (C-G) equation. If estimated CLCr is abnormal, accurate measurement should be obtained by 24 hour creatinine clearance.
  11. Cardiac Function as defined below:
    • Left ventricular ejection fraction ≥ 50 by gated radionuclide study OR shortening fraction of ≥ 27% by echocardiogram
    • Presence of no ventricular arrhythmias except for benign premature ventricular contractions.
    • QTc (Frideriica) interval < 450 ms.
  12. All colony-forming growth factor(s) (i.e., filgrastim, sargramostim or erythropoietin) must have been discontinued for at least 7 days prior to Day 1 or 14 days if PEG formulations were received.
  13. An understanding, ability, and willingness to fully comply with study procedures and restrictions.

Exclusion Criteria:

  1. Evidence of progressive disease by radiologic criteria (RAPNO)
  2. Increasing steroid dose prior to Day 1
  3. Diagnosis of porphyria
  4. Hypersensitivity against porphyrins
  5. Current 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
  6. Known history of human immunodeficiency virus (HIV), hepatitis B or C infection, or any active systemic infection
  7. 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
  8. Prior or concurrent therapy with any other anticancer (including radiotherapy) or investigational drug or other investigational intervention.
  9. Use of herbal and fish oil supplements within 7 days prior to Visit 1
  10. Use of blood thinning agents within 7 days prior to Visit 1
  11. Significant acute deterioration in neurologic status within 7 days prior to Day 1, in the opinion of the investigator
  12. Inability to undergo MRI (e.g., presence of a pacemaker)
  13. Pregnancy or breastfeeding
  14. An Overall Skull Density Ratio of 0.45 (±0.05) or less as calculated from the screening CT
  15. Inability to participate in the opinion of the investigator, by being unwilling or unable to return for required follow-up visits or to obtain follow-up studies to assess toxicity to therapy or to adhere to study plan, procedures and restrictions.
Open or close this module Contacts/Locations
Central Contact Person: Jacquie Kuritzky
Telephone: 925-459-5385
Email: jkuritzky@sonalasense.com
Central Contact Backup: Anna Houlihan
Telephone: 925-466-4586
Email: ahoulihan@sonalasense.com
Study Officials: Stuart Marcus, MD
Principal Investigator
SonALAsense, Inc.
Locations: United States, Arizona
Ivy Brain Tumor Center
Phoenix, Arizona, United States, 85013
Contact:Principal Investigator: Nader Sanai, MD
United States, California
University of California, San Francisco
San Francisco, California, United States, 94143
Contact:Principal Investigator: Sabine Mueller, MD
United States, District of Columbia
Children's National / Children's Research Institute
Washington, District of Columbia, United States, 20010-2916
Contact:Principal Investigator: Roger Packer, MD
Open or close this module IPDSharing
Plan to Share IPD: No
Open or close this module References
Citations:
Links:
Available IPD/Information:

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