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TerminatedNCT03490396Updated Nov 22, 2019

Gelclair at Conditioning or After Oral Mucositis Diagnosed vs. Magic Mouth Wash in Stem Cell Transplant Recipients

A Phase 4 interventional study of Gelclair and First® Mouthwash BLM in Oral Mucositis, sponsored by Midatech Pharma US Inc.. Terminated at 2 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2019-11-22.

Sponsored by Midatech Pharma US Inc. · Phase 4, Interventional, and Other

Why this study was terminated
low recruitment
Phase
Phase 4
Study type
Interventional
Enrollment
28
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study summary

Patients receiving high-dose chemotherapy/conditioning prior to stem cell transplantation (SCT) are at high risk for developing painful lesions in the oral cavity, known as oral mucositis (OM).

In this high risk adult population, the study objectives are to investigate the efficacy and tolerability of Gelclair® (GEL; an FDA cleared medical device indicated for the management of painful oral lesions) and ideal timing of initiation of therapy (at the time of conditioning or after mild OM is diagnosed) for the management of oral mucositis (OM), relative to a commercially available compounded mouth wash (First® Mouthwash BLM "Magic Mouth Wash"; MMW) initiated after mild OM is diagnosed. The study may be adapted based on an interim analysis and recommendations of the interim data review committee.

Read the detailed description

Adult patients at high risk for developing OM receiving one of the following myeloablative (MA) pre-transplant conditioning regimens prior to allogeneic transplant along with methotrexate (MTX) as part of graft vs. host disease (GVHD) prophylaxis meeting all other eligibility criteria will be enrolled:

  • FluBu based regimens: either fludarabine: 30 mg/m\^2 x 4 days and busulfan 0.8 mg/kg IV q6h x 4 days; both given daily starting at day -4 OR fludarabine: 40 mg/m\^2 and busulfan: 3.2 mg/kg both given daily on days -6 through -3.
  • Bu/Cy: busulfan, 0.8 mg/kg IV q6h x 4 days (-7 through -4); cyclophosphamide: 60 mg/kg IV once on days -3 and -2
  • Cy/TBI: Cyclophosphamide, 60 mg/kg IV given twice between days -3 and -1 and TBI fractionated (generally over 3 days) for a total of 12Gy

GVHD Prophylaxis:

  • Regimens including methotrexate (MTX; 15 mg/m\^2 planned to be given on days 1, 3, 6 and 11); addition of other agents given along with MTX (e.g., tacrolimus, sirolimus) is acceptable.

Duration of treatment:

  • Arm 1: GEL treatment a minimum of 4x/day initiated from 1st day of conditioning through OM resolution (G0), up to a maximum of 20d.
  • Arms 2 (GEL) and 3 (MMW): Treatment a minimum of 4x/day initiated when G1 or G2 OM diagnosed during observation period (through Day +14 relative to stem cell infusion) through OM resolution (G0), up to a maximum of 20d.
02

Conditions studied

  • Oral Mucositis

Keywords

  • Oral Mucositis
  • Stomatitis
  • Myeloablative
03

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Be age ≥ 18 years old.
  • Have Karnofsky performance status score ≥ 70.
  • Be scheduled to receive one of 3 myeloablative conditioning regimens (defined in population) followed by allogeneic SCT for hematological malignancy.
  • Have anticipated in-patient status for 14 to 20 days from the time of transplant.
  • Be willing and capable of swishing/gargling oral gel/solution as required per protocol.
  • Be willing and capable of completing the assessments and adhering to protocol requirements.
  • Be willing and able to provide written informed consent.

To be randomized to begin treatment, subjects randomized to Arms 2 or 3 must also meet the following criterion:

-Be diagnosed with G1 or G2 OM via WHO OM scale during observation period from conditioning to Day +14.

Exclusion criteria

Exclusion Criteria:

  • Subjects receiving pre-transplant conditioning/GVHD prophylaxis regimens other than those defined, herein.
  • Use of topical or systemic agents/treatments for OM within 2 weeks of treatment day 1.
  • Evidence of uncontrolled infection (oral/oropharyngeal or systemic), including oral herpes or unexplained febrile illness (≥ 99.5F /37.5C) requiring systemic anti-infectives, within 7d of treatment Day 1.
  • Subjects with active oral lesions or other mouth/throat soreness within 7d of study randomization.
  • Any other criteria, in the opinion of the investigator that would make the subject unsuitable for study participation.

For subjects randomized to Treatment Arms 2 or 3 during observation period:

-OM ≥ G3 diagnosed prior to initiating randomized treatment during observation period (conditioning through Day +14; i.e., missed treatment window).

04

Study design

Phase
Phase 4
Primary purpose
Other
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Single (Outcomes assessor)
Enrollment
28 participants (actual)

Study arms

  • Experimental
    Arm 1 (Gelclair at time of conditioning)

    All subjects in study Arm 1 will receive GEL starting on the first day of conditioning.

    Device: Gelclair

  • Active comparator
    Arm 2 (Gelclair when OM diagnosed)

    Subjects in Arm 2 will be observed from initiation of conditioning to Day +14. If subjects develop G1 or G2 OM via WHO OM scale during this period, they will at that time be randomized to immediately receive GEL.

    Device: Gelclair

  • Active comparator
    Arm 3 (MMW when OM diagnosed)

    Subjects in Arm 2 will be observed from initiation of conditioning to Day +14. If subjects develop G1 or G2 OM via WHO OM scale during this period, they will at that time be randomized to immediately receive MMW.

    Combination Product: First® Mouthwash BLM

Interventions

  • DeviceGelclair

    Polyvinylpyrrolidone (PVP) and Sodium Hyaluronate-Containing Oral Gel

    Also known as: Gelclair Bioadherent Oral Gel

  • Combination productFirst® Mouthwash BLM

    Viscous Lidocaine, Diphenhydramine, and Aluminum-magnesium Hydroxide/Simethicone Antacid Suspension Mouthwash

    Also known as: Magic Mouth Wash

05

What researchers measure

Primary outcomes

  1. Incidence/occurrence of any grade Oral Mucositis

    Incidence/develop of any grade of OM as assessed via WHO OM grading scale (Grades possible: 1-4)

    Time frame: Initial study period (initiation of conditioning through day +14 post-transplant)

  2. Area under the curve in mouth and throat soreness (MTS)

    Time frame: While OM ongoing during study period (initiation of conditioning through day +28 post-transplant)

Secondary outcomes

  1. Time to onset of any grade OM

    WHO Grades 1-4

    Time frame: Initial study period (initiation of conditioning through day +14 post-transplant)

  2. Duration of any grade OM

    WHO Grades 1-4

    Time frame: Study period (initiation of conditioning through day +28 post-transplant)

  3. Severity of OM

    WHO Grades 1-4

    Time frame: Study period (initiation of conditioning through day +28 post-transplant)

  4. Incidence of severe OM

    WHO Grades 3-4

    Time frame: Study period (initiation of conditioning through day +28 post-transplant)

  5. Time to onset of severe OM

    WHO Grades 3-4

    Time frame: Study period (initiation of conditioning through day +28 post-transplant)

  6. Duration of severe OM

    WHO Grades 3-4

    Time frame: Study period (initiation of conditioning through day +28 post-transplant)

  7. Magnitude of OM-related pain control

    Based on subject grading of mouth and throat soreness (VAS 0 (no pain) to 10 (max pain possible)) prior to each randomized/rescue OM treatment.

    Time frame: While OM ongoing during study period (initiation of conditioning through day +28 post-transplant)

  8. Duration of pain control

    Based on time at a given mouth and throat soreness level and/or need for rescue treatment to control mouth and throat soreness.

    Time frame: While OM ongoing during study period (initiation of conditioning through day +28 post-transplant)

  9. Opiate and other background pain medication use

    Time frame: While OM ongoing during study period (initiation of conditioning through day +28 post-transplant)

Other outcomes

  1. Weight change over study treatment period

    Time frame: Study period (initiation of conditioning through day +28 post-transplant)

  2. Incidence of treatment-emergent infection

    e.g., bacteremia/febrile neutropenia, including oral infections (e.g., thrush).

    Time frame: While OM ongoing during study period (initiation of conditioning through day +28 post-transplant)

  3. Duration of treatment-emergent infections

    e.g., bacteremia/febrile neutropenia, including oral infections (e.g., thrush).

    Time frame: While OM ongoing during study period (initiation of conditioning through day +28 post-transplant)

  4. Use of anti-infectives for treatment-emergent infections

    Exploratory Endpoint

    Time frame: While OM ongoing during study period (initiation of conditioning through day +28 post-transplant)

  5. Duration of anti-infective use for treatment-emergent infections

    Time frame: While OM ongoing during study period (initiation of conditioning through day +28 post-transplant)

  6. Dose level of anti-infectives for treatment-emergent infections

    Time frame: While OM ongoing during study period (initiation of conditioning through day +28 post-transplant)

  7. Days of hospitalization post-SCT

    Time frame: Study period (initiation of conditioning through day +28 post-transplant)

  8. Incidence of need for a modified diet

    For example, soft, liquid, TPN

    Time frame: While OM ongoing during study period (initiation of conditioning through day +28 post-transplant)

  9. Duration of need for a modified diet

    For example, soft, liquid, TPN

    Time frame: While OM ongoing during study period (initiation of conditioning through day +28 post-transplant)

  10. Treatment Compliance with randomized OM treatment

    Assessed by determining the number of randomized treatments actually taken relative to the number of treatments required (i.e., treatment compliance)

    Time frame: Study period (initiation of conditioning through day +28 post-transplant)

  11. Use of rescue treatments other than randomized agent for managing OM

    Time frame: While OM ongoing during study period (initiation of conditioning through day +28 post-transplant)

  12. Incidence of treatment-emergent xerostomia ≥ G2

    Time frame: Study period (initiation of conditioning through day +28 post-transplant)

  13. Duration of treatment-emergent xerostomia ≥ G2

    Time frame: Study period (initiation of conditioning through day +28 post-transplant)

  14. Use of treatments/medications to manage xerostomia

    Time frame: Study period (initiation of conditioning through day +28 post-transplant)

  15. Duration of use for treatments/medications to manage xerostomia

    Time frame: Study period (initiation of conditioning through day +28 post-transplant)

  16. Impact of OM on activities of daily living

    Via validated oral mucositis daily questionnaire (OMDQ)

    Time frame: Study period (initiation of conditioning through day +28 post-transplant)

  17. Diarrhea associated with OM

    Via validated oral mucositis daily questionnaire (OMDQ)

    Time frame: Study period (initiation of conditioning through day +28 post-transplant)

  18. Exploratory Safety/Tolerability of GEL and MMW

    Assessed by treatment-emergent and related adverse events/serious adverse events/unanticipated adverse device effects and subject reported tolerability.

    Time frame: Study period (initiation of conditioning through day +28 post-transplant)

06

Study locations

2 sites
  • Massachusetts General Hospital
    Boston, Massachusetts 02114, United States
  • Brigham & Women's Hospital/Dana-Farber Cancer Institute
    Boston, Massachusetts 02120, United States
07

References and documents

Individual participant data

Plan to share: No

08

Registry details

Key details

Study ID
NCT03490396
Lead sponsor
Midatech Pharma US Inc.
Collaborators
PharPoint Research, Inc.
Responsible party
Sponsor
First posted
Apr 6, 2018
Start date
May 15, 2018
Primary completion
Nov 15, 2019
Completion
Nov 15, 2019
Last update
Nov 22, 2019

Study contacts

Mary Kay Delmedico, PhD
study director · Midatech Pharma US Inc.

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
Yes
View the source record on ClinicalTrials.gov ↗

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