BridgeMD
A 24-Month, Multi-Centre, Open Label Phase IV Post Authorisation Efficacy Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in Patients with Partial Lipodystrophy
EU CTIS ID: 2022-502950-14-00
What this study is testing
To evaluate the efficacy of metreleptin treatment in patients with PL.
- Therapeutic use (Phase IV)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male and female patients aged ≥12 years of age at the time of signing the ICF and Child Assent Form (if applicable) prior to initiation of any study specific activities/procedures.
- Patient and/or his/her legal representative has/have been informed, has/have read and understood the patient ICF and Child Assent Form (if applicable), has/have given written informed consent and is/are willing to comply with the protocol requirements. If the child is too young or unable to read, then the Child Assent Form must be explained to the child.
- Confirmed diagnosis of familial or acquired PL.
- Confirmation by the Investigator that the potential differential diagnosis of LD has been excluded (e.g., Cushing’s syndrome, anorexia nervosa, cachexia, diencephalic syndrome, Rabson-Mendenhall syndrome, leprechaunism, SHORT syndrome, mandibuloacral dysplasia, progeroid syndromes, localised scleroderma, lichen sclerosus et atrophicus, annular lipodystrophy, semi-circular lipoatrophy, local panniculitis due to connective tissue diseases and autoimmune disorders, intradermal or subcutaneous related lipoatrophy [e.g., insulin, acupuncture, recombinant growth hormone], progressive hemifacial atrophy [Parry- Romberg syndrome])
- Patients must have: a) HbA1c level ≥6.5% and/or b) Fasting serum TG levels ≥500 mg/dL (5.65 mmol/L)
- Standard treatments have failed to achieve adequate metabolic control: a) Patients with HbA1c level ≥6.5% must be on stable dose of anti-diabetic therapy for at least 90 days prior to screening (diet and/or antidiabetic medications) and their diet (as reported by the patients) should be stable and in line with medical recommendations. b) Patients with fasting serum TG levels ≥500 mg/dL (5.65 mmol/L) must be on stable dose of lipid-lowering agents for at least 6 weeks prior to screening (unless these medications were not tolerated or are contra-indicated) and their diet (as reported by the patients) should be stable and in line with medical recommendations.
You likely can't join if
- Known to have tested positive for human immunodeficiency virus (HIV) or known to be diagnosed with HIV-related LD
- Any condition where, in the opinion of the Investigator, participation in this study may pose a significant risk to the patient or could render the patient unable to successfully complete the study (e.g., life expectancy <12 months).
- Known history of severe hypersensitivity reactions to any of the metreleptin product components.
- Known history of drug or alcohol abuse within 1 year prior to Screening as assessed according to the Investigator’s judgment.
- Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m2 calculated by Bedside Schwartz for patients <18 years and CKD-EPI for patients ≥18 years.
- Patients whose anti-diabetic/lipid-lowering therapies and/or other concomitant medications that may affect the primary endpoint results (such as appetite suppressing medications) are not stable at Screening.
See the full eligibility criteria
Who can join
- Male and female patients aged ≥12 years of age at the time of signing the ICF and Child Assent Form (if applicable) prior to initiation of any study specific activities/procedures.
- Patient and/or his/her legal representative has/have been informed, has/have read and understood the patient ICF and Child Assent Form (if applicable), has/have given written informed consent and is/are willing to comply with the protocol requirements. If the child is too young or unable to read, then the Child Assent Form must be explained to the child.
- Confirmed diagnosis of familial or acquired PL.
- Confirmation by the Investigator that the potential differential diagnosis of LD has been excluded (e.g., Cushing’s syndrome, anorexia nervosa, cachexia, diencephalic syndrome, Rabson-Mendenhall syndrome, leprechaunism, SHORT syndrome, mandibuloacral dysplasia, progeroid syndromes, localised scleroderma, lichen sclerosus et atrophicus, annular lipodystrophy, semi-circular lipoatrophy, local panniculitis due to connective tissue diseases and autoimmune disorders, intradermal or subcutaneous related lipoatrophy [e.g., insulin, acupuncture, recombinant growth hormone], progressive hemifacial atrophy [Parry- Romberg syndrome])
- Patients must have: a) HbA1c level ≥6.5% and/or b) Fasting serum TG levels ≥500 mg/dL (5.65 mmol/L)
- Standard treatments have failed to achieve adequate metabolic control: a) Patients with HbA1c level ≥6.5% must be on stable dose of anti-diabetic therapy for at least 90 days prior to screening (diet and/or antidiabetic medications) and their diet (as reported by the patients) should be stable and in line with medical recommendations. b) Patients with fasting serum TG levels ≥500 mg/dL (5.65 mmol/L) must be on stable dose of lipid-lowering agents for at least 6 weeks prior to screening (unless these medications were not tolerated or are contra-indicated) and their diet (as reported by the patients) should be stable and in line with medical recommendations.
- Patients receiving antidiabetic and/or lipid-lowering therapy prior to the beginning of the study must be kept stable on optimised treatment based on the Investigator’s judgement and stable during the screening period. For patients on insulin, a stable dose is defined as no more than 20% change in total daily insulin dose. For all other therapies, a stable dose is defined as no dose change.
- Patients are willing to follow the dietary restrictions recommended by the Investigator.
- Metreleptin naïve and planned to receive commercial supply of metreleptin or currently treated with commercially supplied metreleptin. Patients currently treated with metreleptin can only be enrolled subject to medical monitor approval and should meet all the following criteria: a) Initiated treatment within 6 months prior to Screening b) Have retained serum samples taken prior to initiation of metreleptin, that can be used to evaluate leptin levels and immunogenicity c) Have documented HbA1c and TG levels prior to initiation of metreleptin (unless these can be assessed based on the retained serum samples).
- Females of childbearing potential must be postmenopausal (defined as cessation of menses for at least 1 year), surgically sterile (hysterectomy, bilateral oophorectomy, or tubal ligation), or willing to use an effective method of contraception (such methods include combined [estrogen and progestogen containing] hormonal contraception: oral / intravaginal; transdermal / progestogen-only hormonal contraception: oral / injectable; implantable / intrauterine device [IUD] / intrauterine hormone-releasing system [IUS] / bilateral tubal occlusion / vasectomised partner/ sexual abstinence / condoms) for the duration of the study (from the time they sign an ICF and Child Assent Form (if applicable), until 4 weeks after the last dose of study drug). Hormonal contraception alone, including oral, injectable, transdermal, and implantable forms, is not acceptable; an additional barrier method must be used. Intravaginal hormonal contraception or IUS alone are allowed per the Investigator’s discretion. Patients on oral contraceptives will not be required to discontinue medication. Female patients will not be permitted to commence oral contraceptives while taking study treatment during the study.
What rules you out
- Known to have tested positive for human immunodeficiency virus (HIV) or known to be diagnosed with HIV-related LD
- Any condition where, in the opinion of the Investigator, participation in this study may pose a significant risk to the patient or could render the patient unable to successfully complete the study (e.g., life expectancy <12 months).
- Known history of severe hypersensitivity reactions to any of the metreleptin product components.
- Known history of drug or alcohol abuse within 1 year prior to Screening as assessed according to the Investigator’s judgment.
- Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m2 calculated by Bedside Schwartz for patients <18 years and CKD-EPI for patients ≥18 years.
- Patients whose anti-diabetic/lipid-lowering therapies and/or other concomitant medications that may affect the primary endpoint results (such as appetite suppressing medications) are not stable at Screening.
- Treatment with any Investigational Medicinal Product (IMP) within 6 months or 5 times the terminal half-life of the corresponding IMP, whichever is longer, before the screening visit.
- For females only - currently pregnant (confirmed with positive pregnancy test) or breastfeeding.
- Positive pregnancy test (urine or serum) for females of childbearing potential.
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.