A multi-center clinical study with LJPC-401 for the treatment of iron overload in adult patients with hereditary hemochromatosis
- Conditions
- Hereditary HemochromatosisMedDRA version: 20.0Level: SOCClassification code 10027433Term: Metabolism and nutrition disordersSystem Organ Class: 10027433 - Metabolism and nutrition disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2017-003598-33-GB
- Lead Sponsor
- a Jolla Pharmaceutical Company
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 76
1. Patients with a clinical diagnosis of hereditary hemochromatosis.
2. Patients who are prescribed therapeutic phlebotomy for the treatment of hereditary hemochromatosis.
3. Patients = 18 years of age.
4. Patients with a serum ferritin level from >100 ng/mL.
5. Patients with a TSAT level > 45%.
6. Female patients must be of non-childbearing potential, or using a highly effective method of contraception during participation in the study and for 30 days after the last dose of study drug. Highly effective methods of contraception are defined as those, alone or in combination, that result in a low failure rate when used consistently and correctly. Refer to Protocol Section 10.1.6.4.1
for the list of highly effective methods of contraception that can be used in this study.
7. Female patients of child bearing potential must have a negative serum pregnancy test at Screening (within 30 days prior to the first dose of study drug) and negative urine pregnancy test at Day 1 (ie, prior to initial dosing of study drug).
8. Male patients must be surgically sterile (vasectomy), or using a highly effective method of contraception during participation in the study and for 30 days after the last dose of study drug. Highly effective methods of contraception are defined as those, alone or in combination, that result in a low failure rate when used consistently and correctly. Refer to Protocol Section 10.1.6.4.1 for the list of highly effective methods of contraception that can be used in this study.
9. Patient must be willing and able to provide written informed consent.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 46
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 30
1. Patients receiving iron chelation therapy within 7 days prior to the
first dose of study drug.
2. Patients recently diagnosed, who have initiated phlebotomy treatments less than 3 months prior to the first dose of study drug.
3. Patients with a concomitant disease, disability or condition, including laboratory abnormality and ECG findings, which may interfere with the conduct of the study, or which would, in the opinion of the Investigator, pose an unacceptable risk to the patient in this study, including, but not limited to clinically significant arrhythmias, alcohol dependency or abuse, drug dependency or abuse, or psychiatric disease. Patients with depression are eligible if receiving a
stable dose of medication for at least 90 days prior to the first dose of
study drug.
4. Pregnant or lactating women.
5. Patients taking an immunosuppressive agent that has not been
approved for use by La Jolla (excluding topical over-the-counter steroids, and nonsteroidal anti-inflammatory drugs). Refer to Section 7.1 for the list of prohibited medications and procedures.
6. Patients participating in an unapproved investigational drug or investigational therapeutic device study within 30 days prior to study drug dosing, ie, there must be at least 30 days in between the last dose on a prior study and the first dose administration on this trial.
7. Patients who are unwilling or unable to comply with the study protocol requirements.
8. Patients with type 1 diabetes or type 2 diabetes with haemoglobin Alc greater than 8% within 2 months prior to randomisation.
9. Patients with uncontrolled active hepatitis B, hepatitis C, or human immunodeficiency virus (HIV) infection.
10. Patients with Child Pugh class C cirrhosis or liver failure.
11. Patients with severe congestive heart failure (NYHA = Class 4).
12. Patients who have advanced renal failure with an estimated glomerular filtration rate (eGFR) < 15 mL/min/1.73m2.
13. Patients with a history of allergic reaction to hepcidin or excipients.
14. Patients who have planned surgery (excluding dental surgery or simple dermatologic procedures) during participation in this study.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To compare the effect of weekly dosing of LJPC-401 versus placebo on Transferrin saturation (TSAT) in adult patients with hereditary hemochromatosis;Secondary Objective: - To compare the effect of LJPC-401 versus placebo on phlebotomy requirements<br>- To establish the safety and tolerability of LJPC-401 versus placebo in adult patients with hereditary hemochromatosis<br>- To compare the effect of LJPC-401 versus placebo on serum ferritin<br>;Primary end point(s): Change in TSAT defined as follows: <br>- For patients with 1 phlebotomy (the standard of care therapeutic<br>phlebotomy on Day 1 [predose]), change in TSAT from baseline to Week<br>16 (24 [± 4] hours postdose).<br>- For patients undergoing 2 or more phlebotomies, change in TSAT from baseline to the most recent postdose fasting TSAT observed prior to the second phlebotomy.;Timepoint(s) of evaluation of this end point: At screening, weekly from Week 1 to Week 16
- Secondary Outcome Measures
Name Time Method Secondary end point(s): • Number of phlebotomies Day 2 to End of Study (EOS)/30 (+ 3) days<br>after the last dose of study drug.<br>• Change in serum ferritin defined as follows: <br>- For patients with 1 phlebotomy (the standard of care therapeutic<br>phlebotomy on Day 1 [predose]), change in serum ferritin from baseline<br>to Week 16 (24 [± 4] hours postdose).<br>- For patients undergoing 2 or more phlebotomies, change in serum<br>ferritin from baseline to the most recent postdose serum ferritin<br>observed prior to the second phlebotomy.<br>• Adverse events (AEs) from consent to EOS<br>• Changes in clinical laboratory evaluations including serum iron parameters from baseline to EOS<br>• Changes in vital signs, electrocardiograms (ECGs), use of concomitant medications, and physical examinations from baseline to EOS<br>• Immunogenicity (anti-drug antibody)/pharmacokinetics (PK) from baseline to EOS<br>;Timepoint(s) of evaluation of this end point: At screening, weekly from Week 1 to Week 16 and at EOS