Epidermolysis BullosaPhase 2NCT03836001
A Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus in Patients With Epidermolysis Bullosa
A Phase 2 study testing Serlopitant Tablet in people with Epidermolysis Bullosa, run by Stanford University at 1 site in United States.
Trial summary
To determine if Serlopitant (when taken by mouth) is safe and works on itch in patients aged 13 and above with EB. Read more about this study
- Who is it for?
- Ages 13 years and older
- Conditions the study is for
- Epidermolysis Bullosa
- What is being tested?
- Serlopitant Tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 28 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Apr 2019to 24 Jun 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 13 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Males or females who are at least 13 years of age.
- Willing and able to understand and sign informed assent/consent. Adolescents will need a parent or guardian willing and able to give consent.
- Clinical diagnosis of epidermolysis bullosa (dystrophic, junctional or simplex).
- History of chronic pruritus of at least 6 weeks in duration
- 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
- 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.
- Judged to be in good health based upon the results of a physical examination, medical history, and safety laboratory tests.
Who cannot join
- 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.
- Have a history of sensitivity to any components of the study material.
- Are females of childbearing potential who are unwilling to use adequate contraception or who are breast feeding.
- 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.
- Have chronic renal disease, i.e., serum creatinine greater than 2 times the upper limit of normal.
- Have chronic liver disease. Subjects with hepatitis B and C who have normal liver function may be enrolled.
Show all 19Show fewer
- 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.
- Have a history of thyroid cancer, thyroid nodules, inadequately treated thyroid disease, or abnormal TSH or free T4 at screening.
- Have a history of abnormalities in adrenal or pituitary function (pituitary adenoma, adrenal insufficiency, or adrenal nodule).
- Screening cortisol level < 3 mcg/dL
- Unevaluated abnormalities in cortisol, ACTH, or prolactin.
- 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).
- 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.
- Have taken investigational medications within 30 days prior to Screening.
- 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).
- 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)
- 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.
- Within in the past 12 months, have expressed suicidal ideation with some intent to act.
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
To determine if Serlopitant (when taken by mouth) is safe and works on itch in patients aged 13 and above with EB.
Read the detailed description
The investigator will determine whether more patients taking serlopitant 5 mg daily as compared to placebo can achieve at least a 3-point reduction in the 24-hour Average Itch Numeric Rating Scale (NRS) following two months of treatment.
Secondary objectives include;
- comparative weekly change in daily worst itch NRS,
- comparative weekly change in daily average itch NRS,
- the proportion of patients who achieve at least 30% or 50% reduction in Average Itch NRS at month 2,
- proportion of patients achieving 2-point and 4-point reductions in Average Itch NRS at month 2,
- Patient Global Impression of Change (PGIC) at month 2, the change in participant static assessment of itch at month 2, and
- assessment of the safety of serlopitant in adolescents (≥13 y.o.) and adults with epidermolysis bullosa-related itch.
- Conditions
- Epidermolysis Bullosa
- Being tested
- Serlopitant Tablet, Placebo Oral Tablet
- Medical subject headings
- Epidermolysis Bullosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Serlopitant TabletDrug
Also called VPD-737
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.
Placebo Oral TabletDrug
Also called Sugar pill
The placebo is a tablet that looks like a drug but has no drug or other active ingredient in it.
| Study group | Role | What they receive |
|---|---|---|
| Placebo Oral Tablet | Placebo comparator | Participants will undergo two months of dosing with a placebo (inactive drug or sugar pill), followed by one month of washout. After the washout period, all participants were invited to participate in an open-label extension study with serlopitant 5 mg daily. The duration of the open-label extension study was either 12 months (for those who enrolled before May 2020) or 3 months (for those who registered after May 2020) due to drug availability. Receives: Drug: Placebo Oral Tablet |
| Serlopitant Tablet | Active comparator | Participants will undergo two months of Serlopitant 5mg daily per oral, followed by one month of washout. After the washout period, all participants were invited to participate in an open-label extension study with serlopitant 5 mg daily. The duration of the open-label extension study was either 12 months (for those who enrolled before May 2020) or 3 months (for those who registered after May 2020) due to drug availability. Receives: Drug: Serlopitant Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients Who Achieve at Least a 3-point Reduction in AI-NRS.
Participants will be asked to complete a daily itch diary with their average itch numeric rating scale (AI-NRS) over the past 24 hours. Score range: 0 to 10, higher scores mean more itching.
Measured: baseline and after two months of treatment
Secondary outcomes (8)
Number of Patients Who Achieve at Least a 2-point Reduction in AI-NRS.
Participants will be asked to complete a daily itch diary with their average itch numeric rating scale (AI-NRS) over the past 24 hours. Score range: 0 to 10, higher scores mean more itching.
Measured: baseline and after two months of treatment
Number of Patients Who Achieve at Least a 4-point Reduction in AI-NRS.
Participants will be asked to complete a daily itch diary with their average itch numeric rating scale (AI-NRS) over the past 24 hours. Score range: 0 to 10, higher scores mean more itching.
Measured: baseline and after two months of treatment
Number of Patients Who Achieve at Least a 30% Reduction in AI-NRS.
Participants will be asked to complete a daily itch diary with their average itch numeric rating scale (AI-NRS) over the past 24 hours. Score range: 0 to 10, higher scores mean more itching.
Measured: baseline and after two months of treatment
Number of Patients Who Achieve at Least a 50% Reduction in AI-NRS.
Participants will be asked to complete a daily itch diary with their average itch numeric rating scale (AI-NRS) over the past 24 hours. Score range: 0 to 10, higher scores mean more itching.
Measured: baseline and after two months of treatment
Weekly Worst Itch NRS
Participants will be asked to complete a daily itch diary with their worst itch numeric rating scale (WI-NRS) over the past 24 hours. Score range: 0 to 10, higher scores mean more itching.
Measured: baseline and week 1, 2, 3, 4, 5, 6, 7, and 8
Weekly AI-NRS
Participants will be asked to complete a daily itch diary with their average itch numeric rating scale (AI-NRS) over the past 24 hours. Score range: 0 to 10, higher scores mean more itching.
Measured: baseline and week 1, 2, 3, 4, 5, 6, 7, and 8
Patient Global Impression of Change (PGIC)
PGIC categorized as "Very much better", "Moderately better", "A little better", "No change", "A little worse", "Moderately worse", and "Very much worse".
Measured: month 2
Change in Static Participant Assessment of Itch
Severity of itch over past 7 days assessed as Very Severe, Severe, Moderate, Mild, or None.
Change is reported as the number of participants with 3-category improvement, 2-category improvement, 1-category improvement, no change, or worse.
Measured: month 2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2019
- Study start18 Apr 2019
- Primary completion6 Dec 2021
- Study completion24 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Epidermolysis BullosaStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- 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 blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Collaborators
- Epidermolysis Bullosa Research Partnership, Vyne Therapeutics Inc.
- Responsible party
- Principal investigator, Albert Chiou, Stanford University
- Investigators
- Albert S Chiou, MD/MBA, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03836001
- Sponsor's protocol ID
- 49084
- Status last verified
- Jan 2023
- First submitted
- 7 Feb 2019
- Last updated
- 14 Feb 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
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 patients with epidermolysis bullosa: A randomized clinical trial. J Am Acad Dermatol. 2020 Jun;82(6):1415-1421. doi: 10.1016/j.jaad.2019.09.014. Epub 2019 Sep 18.
PubMed 31541747
Study at a glance
From the registry record, last updated by the sponsor on 14 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus in Patients With Epidermolysis Bullosa
- ClinicalTrials.gov ID
- NCT03836001
- Acronym / sponsor ID
- 49084
- Conditions
- Epidermolysis Bullosa
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Collaborators
- Epidermolysis Bullosa Research Partnership, Vyne Therapeutics Inc.
- Study start
- 18 Apr 2019 (actual)
- Primary completion
- 6 Dec 2021 (actual)
- Study completion
- 24 Jun 2022 (actual)
- First posted
- 11 Feb 2019 (actual)
- Results
- Posted 23 Dec 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 at least a 3-point reduction in the 24-hour Average Itch Numeric Rating Scale (NRS) following two months of treatment.
Secondary objectives include;
- comparative weekly change in daily worst itch NRS,
- comparative weekly change in daily average itch NRS,
- the proportion of patients who achieve at least 30% or 50% reduction in Average Itch NRS at month 2,
- proportion of patients achieving 2-point and 4-point reductions in Average Itch NRS at month 2,
- Patient Global Impression of Change (PGIC) at month 2, the change in participant static assessment of itch at month 2, and
- assessment of the safety of serlopitant in adolescents (≥13 y.o.) and adults with epidermolysis bullosa-related itch.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- 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.
| Arm | Type | Receives |
|---|---|---|
| Placebo Oral Tablet | Placebo comparator | Drug: Placebo Oral TabletParticipants will undergo two months of dosing with a placebo (inactive drug or sugar pill), followed by one month of washout. After the washout period, all participants were invited to participate in an open-label extension study with serlopitant 5 mg daily. The duration of the open-label extension study was either 12 months (for those who enrolled before May 2020) or 3 months (for those who registered after May 2020) due to drug availability. |
| Serlopitant Tablet | Active comparator | Drug: Serlopitant TabletParticipants will undergo two months of Serlopitant 5mg daily per oral, followed by one month of washout. After the washout period, all participants were invited to participate in an open-label extension study with serlopitant 5 mg daily. The duration of the open-label extension study was either 12 months (for those who enrolled before May 2020) or 3 months (for those who registered after May 2020) due to drug availability. |
Who may qualify
- Ages
- 13 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Males or females who are at least 13 years of age.
- Willing and able to understand and sign informed assent/consent. Adolescents will need a parent or guardian willing and able to give consent.
- Clinical diagnosis of epidermolysis bullosa (dystrophic, junctional or simplex).
- History of chronic pruritus of at least 6 weeks in duration
- 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
Key exclusion criteria (19 listed)
- 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.
- Have a history of sensitivity to any components of the study material.
- Are females of childbearing potential who are unwilling to use adequate contraception or who are breast feeding.
- 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.
- Have chronic renal disease, i.e., serum creatinine greater than 2 times the upper limit of normal.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Serlopitant TabletAlso: VPD-737Serlopitant 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. | Drug | Serlopitant Tablet |
| Placebo Oral TabletAlso: Sugar pillThe placebo is a tablet that looks like a drug but has no drug or other active ingredient in it. | Drug | Placebo Oral Tablet |
Primary outcome
Number of Patients Who Achieve at Least a 3-point Reduction in AI-NRS.
Time frame: baseline and after two months of treatment
Participants will be asked to complete a daily itch diary with their average itch numeric rating scale (AI-NRS) over the past 24 hours. Score range: 0 to 10, higher scores mean more itching.
8 secondary outcomes
Number of Patients Who Achieve at Least a 2-point Reduction in AI-NRS.
Time frame: baseline and after two months of treatment
Number of Patients Who Achieve at Least a 4-point Reduction in AI-NRS.
Time frame: baseline and after two months of treatment
Number of Patients Who Achieve at Least a 30% Reduction in AI-NRS.
Time frame: baseline and after two months of treatment
Number of Patients Who Achieve at Least a 50% Reduction in AI-NRS.
Time frame: baseline and after two months of treatment
Weekly Worst Itch NRS
Time frame: baseline and week 1, 2, 3, 4, 5, 6, 7, and 8
Weekly AI-NRS
Time frame: baseline and week 1, 2, 3, 4, 5, 6, 7, and 8
Patient Global Impression of Change (PGIC)
Time frame: month 2
Change in Static Participant Assessment of Itch
Time frame: month 2
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Feb 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 31541747Chiou 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 patients with epidermolysis bullosa: A randomized clinical trial. J Am Acad Dermatol. 2020 Jun;82(6):1415-1421. doi: 10.1016/j.jaad.2019.09.014. Epub 2019 Sep 18.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Albert S Chiou, MD/MBA principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Serlopitant Tablet in people with Epidermolysis Bullosa, run by Stanford University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Dec 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Patients Who Achieve at Least a 3-point Reduction in AI-NRS. (time frame: baseline and after two months of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03836001, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Feb 2023.