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A Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus in Patients With Epidermolysis Bullosa

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ClinicalTrials.gov Identifier: NCT03836001
Recruitment Status : Recruiting
First Posted : February 11, 2019
Last Update Posted : May 16, 2019
Sponsor:
Collaborators:
Epidermolysis Bullosa Research Partnership
Menlo Therapeutics
Information provided by (Responsible Party):
Albert Chiou, Stanford University

Tracking Information
First Submitted Date  ICMJE February 7, 2019
First Posted Date  ICMJE February 11, 2019
Last Update Posted Date May 16, 2019
Actual Study Start Date  ICMJE April 18, 2019
Estimated Primary Completion Date September 30, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 7, 2019)
The proportion of patients who achieve at least a 3-point reduction in NRS itch severity from baseline and after two months of treatment. [ Time Frame: 2 months ]
Subjects will be asked to complete itch surveys (NRS) and daily before bedtime regarding their daily average itch score and their wound score while bathing/bandage changing.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03836001 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus in Patients With Epidermolysis Bullosa
Official Title  ICMJE A Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus in Patients With Epidermolysis Bullosa
Brief Summary To determine if Serlopitant (when taken by mouth) is safe and works on itch in patients aged 13 and above with EB.
Detailed Description

The investigator will determine whether more patients taking serlopitant 5 mg daily as compared to placebo can achieve a 3 point or greater reduction in itch severity as measured by numeric rating scale (NRS) score following two months of treatment.

Secondary objectives include;

  1. continuous change in NRS itch score over time,
  2. the proportion of patients who achieve at a least 30% or 50% reduction in NRS severity from baseline at the end of two months of treatment
  3. proportion of patients achieving 2 point and 4 point reductions in itch severity following two months of treatment, and
  4. change in median NRS score over time as compared to baseline.
  5. assessment on the safety of Serlopitant in adolescents (≥13 y.o.) and adults with pruritus associated with epidermolysis bullosa (EB).
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
This is an investigator-initiated, single-center, randomized, double-blind, placebo controlled, parallel arm trial evaluating the effects of serlopitant at 5 mg by mouth daily on EB-related itch.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description:
This is a double-blind study.
Primary Purpose: Treatment
Condition  ICMJE Epidermolysis Bullosa
Intervention  ICMJE
  • Drug: Serlopitant Tablet
    Serlopitant is a small molecule, highly selective NK1-R (neurokinin-1 receptor) antagonist. Two critical mediators of the urge to scratch are Substance P, or SP, and its receptor, NK1-R. SP is a naturally occurring peptide in the tachykinin neuropeptide family. Tachykinins have a broad range of functions in the nervous and immune systems. SP binding of NK1-R has been shown to be a key mediator of sensory nerve signaling, including the itch-scratch reflex and the vomiting reflex.
    Other Name: VPD-737
  • Drug: Placebo Oral Tablet
    The placebo is a tablet that looks like a drug but has no drug or other active ingredient in it.
    Other Name: Sugar pill
Study Arms  ICMJE
  • Placebo Comparator: Placebo Oral Tablet
    We aim to recruit at least 20 patients who will undergo two months of dosing with placebo (inactive drug or sugar pill), followed by one month of wash-out. All patients will be offered the option of participating in a 12-month open label extension with serlopitant at 5 mg (taken by mouth) daily for continued safety monitoring.
    Intervention: Drug: Placebo Oral Tablet
  • Active Comparator: Serlopitant Tablet
    We aim to recruit at least 20 patients who will undergo two months of Serlopitant dosing, followed by one month of wash-out. All patients will be offered the option of participating in a 12-month open label extension with serlopitant 5 mg (taken by mouth) daily for continued safety monitoring.
    Intervention: Drug: Serlopitant Tablet
Publications * Chiou AS, Choi S, Barriga M, Dutt-Singkh Y, Solis DC, Nazaroff J, Bailey-Healy I, Li S, Shu K, Joing M, Kwon P, Tang JY. Phase 2 Trial of a Neurokinin-1 Receptor Antagonist for the Treatment of Chronic Itch in Epidermolysis Bullosa Patients: A Randomized Clinical Trial. J Am Acad Dermatol. 2019 Sep 18. pii: S0190-9622(19)32751-3. doi: 10.1016/j.jaad.2019.09.014. [Epub ahead of print]

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: February 7, 2019)
40
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 28, 2022
Estimated Primary Completion Date September 30, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Males or females who are at least 13 years of age.
  2. Willing and able to understand and sign informed assent/consent. Adolescents will need a parent or guardian willing and able to give consent.
  3. Clinical diagnosis of epidermolysis bullosa (dystrophic, junctional or simplex).
  4. History of chronic pruritus of at least 6 weeks in duration
  5. On the Screening Visit or Screening phone call, patients must have an NRS pruritus score of at least 5 on average itch score in the past 24 hours
  6. Female subjects must be of non-childbearing potential (ie, post-menopausal for at least 1 year, had a hysterectomy, or had a tubal ligation) or, if of childbearing potential, must have a confirmed negative urine pregnancy test prior to study treatment and be willing to use effective contraception for the duration of the trial. Effective contraception is defined as follows: oral/implant/injectable/ transdermal contraceptives, intrauterine device, condom with spermicide, or diaphragm with spermicide. Abstinence or partner's vasectomy is acceptable if the female agrees to use effective contraception if she decides to discontinue abstinence or to have sexual intercourse with a non-vasectomized partner.
  7. Judged to be in good health based upon the results of a physical examination, medical history, and safety laboratory tests.

Exclusion Criteria:

  1. Have any medical condition or disability that would interfere with the assessment of safety or efficacy in this trial or would compromise the ability of the subject to travel to Stanford or to undergo study procedures or to give informed consent.
  2. Have a history of sensitivity to any components of the study material.
  3. Are females of childbearing potential who are unwilling to use adequate contraception or who are breast feeding.
  4. Have any chronic or acute medical condition that, in the opinion of the investigator, might interfere with the study results or place the subject at undue risk.
  5. Have chronic renal disease, i.e., serum creatinine greater than 2 times the upper limit of normal.
  6. Have chronic liver disease. Subjects with hepatitis B and C who have normal liver function may be enrolled.
  7. Have a current malignancy (such as Hodgkin's lymphoma, B or T cell lymphoma, or myeloma) or blood cell dyscrasia (e.g., polycythemia or myelofibrosis) that would lead to systemic chronic pruritus.
  8. Subjects with untreated hyperthyroidism.
  9. Have pruritus of psychogenic etiology (delusions of parasitosis, obsessive compulsive disorder and major depression) or neuropathic etiology (due to shingles, spinal cord injury or with neurologic deficit).
  10. Have pruritus due to urticaria, drug allergy, or infection (such as pityriasis rosacea or tinea or active human immunodeficiency virus [HIV]). Note: Subjects with HIV who have undetectable viral load, and stable retro-viral therapy may enroll.
  11. Have taken investigational medications within 30 days prior to Screening.
  12. Are unwilling to discontinue specific medications that, in the view of the investigator may have significant interactions with the trial drug, for at least two weeks prior to initiation of study and throughout the study period (this includes miconazole, delavirdine, conivaptan, Clarithromycin, telithromycin, nefazodone, itraconazole, ketoconazole, indinavir, lopinavir, nelfinavir, ritonavir, saquinavir).
  13. Are unable or unwilling to maintain their current anti-itch and opioid-based pain medications at a stable dosage through the course of the two months of active treatment (including but not limited to opioid pain medications, antihistamines, and gabapentin)
  14. Started or changed medications, creams, or emollients including over-the-counter (OTC) preparations or bath oil treatment specifically for relief of pruritus within 30 days prior to Screening.
  15. Within in the past 12 months, have expressed suicidal ideation with some intent to act.
  16. Have any social or medical condition (e.g. alcoholism, drug dependency, psychotic state) that, in the investigator's opinion, might interfere with the subject's ability to comply with the requirements of the protocol.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 13 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Clinical Trial Coodinator 650-721-7166 ceteng7@stanford.edu
Contact: Clinical Trial Coordinator 6507217149 baileyhi@stanford.edu
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03836001
Other Study ID Numbers  ICMJE 49084
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Albert Chiou, Stanford University
Study Sponsor  ICMJE Stanford University
Collaborators  ICMJE
  • Epidermolysis Bullosa Research Partnership
  • Menlo Therapeutics
Investigators  ICMJE
Principal Investigator: Albert S Chiou, MD/MBA Stanford University
PRS Account Stanford University
Verification Date May 2019

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