The purpose of this study is to evaluate a drug (LUM001) that may help treat the liver and control itching in Alagille Syndrome. In this study, all children who are eligible to enrol will take study drug for 18 weeks, followed by a 4 week period where they will either take LUM001 or placebo. After this 4 week period, all patients will go back on active study drug treatment for the remaining 26 weeks.

Phase 1

• Conditions: Alagille Syndrome; MedDRA version: 18.1Level: PTClassification code 10053870Term: Alagille syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2013-005373-43-BE
• Lead Sponsor: umena Pharmaceuticals LLC

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-02-05
• Last Update Posted: 28 September 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

To participate in this study subjects must meet all of the following criteria:
1.Male or female between the ages of 12 months and 18 years inclusive.
2.Diagnosis of ALGS .
3.Evidence of cholestasis (one or more of the following):
a.Total serum bile acid > 3x ULN for age.
b.Conjugated bilirubin > 1 mg/dL.
c.Fat soluble vitamin deficiency otherwise unexplainable.
d.GGT > 3x ULN for age.
e.Intractable pruritus explainable only by liver disease.
4.Females of childbearing potential must have a negative serum pregnancy test during Screening.
5.Sexually active females must be prepared to use an effective method of contraception during the trial, such as:
a.Hormonal (e.g., contraceptive pill, patch, intramuscular implant or injection).
b.Barrier method, i.e., (a) condom (male or female) or (b) diaphragm, with spermicide.
c.Intrauterine device (IUD).
6.Subject is expected to have a consistent caregiver(s) for the duration of the study.
7.Informed consent and assent (per IRB/IEC) as appropriate.
8.Access to phone for scheduled calls from study site.
9.Caregivers (and age appropriate subjects) must be willing and able to use an eDiary device during the study.
10.Caregivers (and age appropriate subjects) must digitally accept the licensing agreement in the eDiary software.
11.Caregivers (and age appropriate subjects) must complete at least 10 eDiary reports (morning or evening) during each of two consecutive weeks of the screening period (maximum possible reports = 14 per week).
12.Average daily score >2 on the Itch Reported Outcome (ItchRO™) questionnaire (maximum possible daily score of 4) for two consecutive weeks in the screening period, prior to dosing. A daily score is the higher of the scores for the morning and evening ItchRO. The average daily score is the sum of all daily scores divided by the number of days the ItchRO was completed.

Eligible subjects for the 52-week follow-up period:
Subjects will be considered eligible for the optional 52-week follow-up treatment period if they have:
•Completed the protocol through the Week 48 visit with no safety concerns. Subjects who were discontinued due to safety reasons can be re-challenged if blood tests are back to relatively normal values for this patient population and subject does not meet any of the protocol’s stopping rules. The decision will be made by the investigator in consultation with the Sponsor Medical Monitor.
•Subjects who have undergone a surgical disruption of the enterohepatic circulation will not be eligible to enter into the follow up treatment period.
•Subjects who were discontinued for other reasons will be considered for the optional 52-week follow-up treatment period on an individual basis. The decision will be made by the investigator in consultation with the Sponsor Medical Monitor.

Are the trial subjects under 18? yes
Number of subjects for this age range: 30
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Subjects will be excluded from the study if they meet any of the following criteria:
1.Chronic diarrhea requiring ongoing intravenous fluid or nutritional intervention.
2.Surgical interruption of the enterohepatic circulation.
3.Previous liver transplant.
4.Decompensated cirrhosis [ALT >15 x ULN, INR >1.5 (unresponsive to vitamin K therapy), albumin < 3.0 gm/dL, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy].
5.History or presence of other concomitant liver disease.
6.History or presence of any other disease or condition known to interfere with the absorption, distribution, metabolism or excretion of drugs, including bile salt metabolism in the intestine (e.g., inflammatory bowel disease).
7.History or presence of gallstones or kidney stones
8.Known diagnosis of human immunodeficiency virus (HIV) infection.
9.Cancers except for in situ carcinoma, or cancers treated at least 5 years prior to Screening with no evidence of recurrence.
10.Recent medical history, or current status that suggests that the subject may be unable to complete the study.
11.Any female who is pregnant or lactating or who is planning to become pregnant during the study period.
12.Known history of alcohol or substance abuse.
123Administration of bile acid or lipid binding resins within 28 days prior to screening and throughout the trial.
14.Known hypersensitivity to LUM001 or any of its components
15.Receipt of investigational drug, biologic, or medical device within 28 days prior to Screening, or 5 half-lives of the study agent, whichever is longer.
16.History of non-adherence to medical regimens, unreliability, mental instability or incompetence that could compromise the validity of informed consent or lead to non-adherence with the study protocol based upon Investigator judgment.
17.Any other conditions or abnormalities which, in the opinion of the Investigator or Medical Monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study.
18.Subjects weighing over 50kg at screening

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified