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Efficacy, Safety and Pharmacokinetics of Topical Timolol in Infants With Infantile Hemangioma (IH) (TIM01)

This study is currently recruiting participants.
Verified October 2017 by Chiara Melloni, Duke University Medical Center
Sponsor:
ClinicalTrials.gov Identifier:
NCT02913612
First Posted: September 26, 2016
Last Update Posted: October 3, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
The EMMES Corporation
Information provided by (Responsible Party):
Chiara Melloni, Duke University Medical Center
August 12, 2016
September 26, 2016
October 3, 2017
May 5, 2017
September 28, 2018   (Final data collection date for primary outcome measure)
Comparison of partial response of hemangioma color within the treat arm compared to the untreated controls [ Time Frame: 180 days ]
Partial response of hemangioma color from baseline to 180 days within each treatment arm and compared with untreated controls
Same as current
Complete list of historical versions of study NCT02913612 on ClinicalTrials.gov Archive Site
  • Efficacy Comparison of proportion of infants with partial response or greater change in volume [ Time Frame: 180 days ]
    Percentage of participants with partial response of change in volume from baseline to 180 days within each treatment arm compared with the untreated controls.
  • Efficacy comparison of proportion of infants with partial response between the two treatment arms [ Time Frame: 180 days ]
    Percentage of participants with partial response of change in color from baseline to 180 days between the two treatment arms
  • Efficacy comparison of partial response of infants with partial response of hemangioma volume between the treatment arms [ Time Frame: 180 days ]
    Percentage of participants with partial response of change in volume from baseline to 180 days between the two arms.
  • Change in Efficacy Improvement of hemangioma complication within the treatment arms [ Time Frame: Baseline and Day 180 ]
    Change in the dynamic complication scale results within each treatment arm
  • Efficacy assessment of time to partial response in both treatment arms [ Time Frame: 180 days ]
    Assess time to partial response by comparing baseline to day 30, day 60, day 120 and 120 in both treatment arms
  • Change in Efficacy assessment of Quality of Life assessment for infants in both treatment arms [ Time Frame: Baseline and Day 180 ]
    Absolute change in the Quality of Life score within each treatment arm
  • Rate of Serious Adverse Events and Adverse Events of special interest in treated infants in both arms [ Time Frame: 180 days ]
    Rate of serious adverse events and adverse events of special interest for infants in both treatment arms
  • Pharmacokinetics (PK) Analysis measuring clearance of Timolol in Plasma specimen [ Time Frame: Up to 12 hours ]
    The PK blood samples will be 1.0 ml each and collected between the following timeframes after application of Timolol: within 2 hours, 2-4 hours, 5-7 hours, 8-10 hours or 11-12 hours.
  • Pharmacokinetics (PK) Analysis measuring Volume of Distribution of Timolol in plasma specimen [ Time Frame: Up to 12 hours ]
    The PK blood samples will be 1.0 ml each and collected between the following timeframes after application of Timolol: within 2 hours, 2-4 hours, 5-7 hours, 8-10 hours or 11-12 hours.
  • Pharmacokinetics (PK) Analysis measuring area under the curve of Timolol in plasma specimen [ Time Frame: Up to 12 hours ]
    The PK blood samples will be 1.0 ml each and collected between the following timeframes after application of Timolol: within 2 hours, 2-4 hours, 5-7 hours, 8-10 hours or 11-12 hours.
  • Pharmacokinetics (PK) Analysis measuring maximum concentration of Timolol in plasma specimen [ Time Frame: Up to 12 hours ]
    The PK blood samples will be 1.0 ml each and collected between the following timeframes after application of Timolol: within 2 hours, 2-4 hours, 5-7 hours, 8-10 hours or 11-12 hours.
Same as current
Not Provided
Not Provided
 
Efficacy, Safety and Pharmacokinetics of Topical Timolol in Infants With Infantile Hemangioma (IH)
Efficacy, Safety, and Pharmacokinetics of Timolol in Infants With Infantile Hemangioma (IH)
Multi-center, double-masked randomized, efficacy, safety and pharmacokinetic (PK) study.
Primary: Describe the efficacy of 0.25% and 0.5% topical timolol maleate GFS as assessed through IH changes in color. Secondary: Describe the safety and PK of topical timolol maleate GFS for treatment of IH.
Interventional
Phase 2
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Infantile Hemangioma
  • Drug: 0.25% Timolol Maleate Gel Forming Solution
    50:50 Randomized 0.25% Timolol Maleate Gel Forming Solution
  • Drug: 0.5% Timolol Maleate Gel Forming Solution
    50:50 Randomized 0.5% Timolol Maleate Gel Forming Solution
  • Experimental: Intervention Group
    Subjects assigned to this arm will be randomized to either 0.25% Timolol or 0.5% Timolol for 180 days. If during the 180 days the subject is considered a treatment failure, the subject will be unblinded. If the subject is on 0.25% timolol they will be changed to 0.5% timolol, if on 0.5% timolol the treating physician will decide to either continue 0.5% timolol or withdraw the subject and begin alternative treatment.
    Interventions:
    • Drug: 0.25% Timolol Maleate Gel Forming Solution
    • Drug: 0.5% Timolol Maleate Gel Forming Solution
  • No Intervention: Non-Intervention Group
    Subjects assigned to this group will not receive treatment. They subject will only be photographed on the same schedule as the intervention group.
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
110
September 28, 2018
September 28, 2018   (Final data collection date for primary outcome measure)

Inclusion Criteria

  1. Documented informed consent from legal guardian
  2. 0-60 days postnatal age at time of first study dose or when enrolled into the non-intervention cohort.
  3. Clinical diagnosis of superficial cutaneous or mucosal infantile hemangioma (must include all of the following): a. Superficial lesion in the dermis b. Thin <2 mm in thickness c. Small <5 cm2 d. Involves skin or keratinized mucosa

Exclusion Criteria

  1. History of previous treatment with any pharmacologic or laser therapy for IH
  2. Ongoing therapy with an oral beta blocker or oral corticosteroid for an unrelated condition (e.g., cardiac arrhythmia, adrenal insufficiency, upper airway obstruction, tetralogy of fallot (TOF), hypertension, reactive airways disease)
  3. IH that requires systemic therapy (defined by dynamic complication scale >3)
  4. IH of the non-keratinized mucosa
  5. Infants with more than one hemangioma that requires therapy
  6. Hemodynamically significant cardiovascular disease, as determined by the investigator
  7. Known allergy to beta blockers or vehicle
  8. Heart rate <100 beats per minute at screening visit
  9. Known prenatal or postnatal diagnosis of 2nd/3rd degree atrioventricular block
  10. History of Reactive Airways Disease (RAD)
  11. Any condition which would make the participant, in the opinion of the investigator unsuitable for the study.
Sexes Eligible for Study: All
up to 60 Days   (Child)
No
Contact: Chiara Melloni, MD, MHS 919-668-8646 chiara.melloni@duke.edu
Contact: Beth Drolet, MD 414-955-2818 bdrolet@mcw.edu
United States
 
 
NCT02913612
Pro00068212
Yes
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Not Provided
Chiara Melloni, Duke University Medical Center
Chiara Melloni
The EMMES Corporation
Principal Investigator: Chiara Melloni, MD, MHS Duke Clinical Research Institute
Study Chair: Beth Drolet, MD Medical College of Wisconsin
Study Chair: Kristen Holland, MD Medical College of Wisconsin
Duke University
October 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP