An Open-Label and Long-Term Extension Study to Evaluate the Efficacy and Safety of Ustekinumab in the Treatment of Patients With Ichthyoses
|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. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04549792|
Recruitment Status : Active, not recruiting
First Posted : September 16, 2020
Last Update Posted : April 6, 2023
- Study Details
- Tabular View
- No Results Posted
- How to Read a Study Record
|Condition or disease||Intervention/treatment||Phase|
|Ichthyosis||Drug: Ustekinumab||Early Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||13 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label and Long-Term Extension Study to Evaluate the Efficacy and Safety of Ustekinumab in the Treatment of Patients With Ichthyoses.|
|Actual Study Start Date :||April 1, 2021|
|Estimated Primary Completion Date :||January 30, 2024|
|Estimated Study Completion Date :||June 30, 2024|
|Experimental: Open Label||
Each subject will receive ustekinumab at Baseline (Day 0) and Months 1, 3, 5, 7, 9, and 11. During the LTE, subjects will receive injections every 8 weeks for one year: Month 13, Month 15, Month 17, Month 19, Month 21, and Month 23. Subjects will come back in for a follow-up visit at Month 25 for an end of study visit (no drug administration).
- Reduction in Ichthyosis Severity Score [ Time Frame: 7 months after initiation of study drug ]To evaluate the efficacy of ustekinumab for ichthyosis, as measured by an at least 50% reduction in severity (using the Ichthyosis Severity Score (ISS) measure
- Occurence of bacterial and fungal infections [ Time Frame: 7 months after initiation of study drug ]To evaluate the safety of ustekinumab for ichthyosis, based on occurrence of bacterial and fungal infections
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||6 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Subject has provided informed consent; parental consent for patients under 18 years of age (plus assent for subjects age ≥ 12 and < 18).
- Subjects are at least 6 years of age or older at the time of screening.
Before screening visit, females must be:
Postmenopausal, defined as
≥ 45 years of age with amenorrhea for at least 18 months, OR
- ≥ 45 years of age with amenorrhea for at least 6 months and a serum FSH level > 40 IU/mL OR
- Of childbearing potential, in which case she must satisfy at least one of the below:
Surgically sterile (has had a hysterectomy or bilateral oophorectomy, tubal ligation, or otherwise be incapable of pregnancy), or
If heterosexually active, practicing a highly effective method of birth control, including hormonal prescription oral contraceptives, contraceptive injections, contraceptive patch, intrauterine device, double-barrier method (e.g., condoms, diaphragm, or cervical cap, with spermicidal foam, cream, film, gel or suppository), or male partner sterilization, consistent with local regulations regarding use of birth control methods for subjects participating in clinical trials, for a period of 16 weeks after the last administration of study agent,
Not heterosexually active. Abstinence is allowed as an acceptable form of contraception.
Note: If a woman participant's childbearing potential changes after start of the study (e.g., a premenarchal woman experiences menarche) or if women of childbearing potential who are not heterosexually active at screening become heterosexually active, they must agree to utilize a highly effective method of birth control, as described above.
Female participants of childbearing potential (menstrual and not surgically sterile), must have a negative serum beta-human chorionic gonadotropin (ᵦ-hCG) pregnancy test at screening and a negative urine pregnancy test at Week 0 (prior to screening visit) and agree not to donate eggs (ova, oocytes) for the purposes of assisted reproduction during the study and for a period of 16 weeks after the last administration of study agent.
• Male participants who are not surgically sterilized and are heterosexually active with a woman of childbearing potential, must agree to use a barrier method of contraception (e.g., condom with spermicidal foam/gel/film/cream/suppository) and to not donate sperm during the study and for 16 weeks after last receiving study agent. Note that barrier methods must also be used in all male subjects sexually active with pregnant partners for at least 16 weeks after last study agent administration.
- Subjects must have a confirmed clinical diagnosis of ichthyosis/ichthyotic disorder, and either have completed genotype or be willing to be genotyped (genotype results will not be required for entry into the study).
- Subjects must have at least moderate erythema (ISS-erythema score ≥ 2) related to his/her ichthyosis/ichthyotic disorder.
- Subjects must be clinically judged to be immunocompetent based on baseline laboratory testing (chemistry and hematology), medical history and physical examination.
- Subjects will have baseline negative QuantiFERON®-TB gold, Hepatitis B, Hepatitis C, and HIV laboratory testing.
- Subjects who are unable to provide informed consent or assent (or who do not have consent from a Legally Authorized Representative if < 18 years).
- Subjects with ichthyosis vulgaris or X-linked recessive ichthyosis.
- Subjects who have a known allergy to ustekinumab or its products.
- Female subjects who are pregnant or breastfeeding, or who are considering becoming pregnant.
- Subjects who have prior biologic use targeting IL-12/IL-23 monoclonal antibody.
- Subjects who have used a systemic retinoid or systemic anti-inflammatory agent within 4 weeks prior to baseline.
- Subjects who have used topical steroid in the previous week, retinoid or keratolytic agent in the previous 24 hours.
- Subjects with active infections or recent history of serious infections, malignancies or history of malignancies, recent immunizations with live vaccines, or any severe, progressive, or uncontrolled renal, hepatic, hematologic, endocrine, pulmonary, cardiac, neurologic, psychiatric, or cerebral disease, or signs or symptoms thereof
- Subjects who are under 6 years of age at the time of screening.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04549792
|United States, Illinois|
|Northwestern University/Lurie Children's Hospital|
|Chicago, Illinois, United States, 60611|
|Principal Investigator:||Amy Paller, MD||Northwestern University|
|Responsible Party:||Amy Paller, Principal Investigator, Northwestern University|
|Other Study ID Numbers:||
|First Posted:||September 16, 2020 Key Record Dates|
|Last Update Posted:||April 6, 2023|
|Last Verified:||April 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Infant, Newborn, Diseases
Ichthyosiform Erythroderma, Congenital
Skin Diseases, Genetic
Genetic Diseases, Inborn