MAP Cohort CLNP023B12004M to Provide Access to Iptacopan for C3 Glomerulopathy
Study Details
Study Description
Brief Summary
The purpose of this Managed Access Program (MAP) Cohort Treatment Plan is to provide guidance to the Physician for the treatment and monitoring of patients in the Cohort MAP.
The Physician should follow the suggested treatment guidelines. Furthermore, the Physician must comply with the MAP Agreement Letter and applicable local laws and regulations.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
Study Design
Outcome Measures
Primary Outcome Measures
Eligibility Criteria
Criteria
Inclusion Criteria:
Patients eligible for inclusion in this Treatment Plan have to meet all of the following criteria for his category:
A) Patients with native kidneys:
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Adult male and female (≥ 18 years) who are able and willing to provide written informed consent.
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C3 Glomerulopathy confirmed by kidney biopsy within last five years.
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Estimated GFR (using the CKD-EPI formula) or measured GFR ≥30 mL/min per 1.73m2.
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UPCR ≥ 1.0 g/g.
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On supportive care, including a maximally tolerated dose of ACEi or ARB for at least 90 days.
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Vaccination against Neisseria meningitidis and Streptococcus pneumoniae is required prior to the start of treatment. If the patient has not been previously vaccinated or if a booster is required, the vaccine should be given according to local regulations at least 2 weeks prior to the first administration of iptacopan. If iptacopan has to start earlier than 2 weeks post vaccination, prophylactic antibiotic treatment must be initiated per local standard of care.
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Vaccination against Haemophilus influenzae (or a booster, if required) should be given, if available and according to local regulations, at least 2 weeks prior to first dosing.
B) Patient with transplanted kidney:
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Adult male and female (≥ 18 years) who are able and willing to provide written informed consent.
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C3G recurrence after kidney transplantation confirmed by kidney biopsy.
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Estimated GFR (using the CKD-EPI formula) or measured GFR ≥30 mL/min/1.73m2
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Vaccination against Neisseria meningitidis and Streptococcus pneumoniae is required prior to the start of treatment. If the patient has not been previously vaccinated or if a booster is required, the vaccine should be given according to local regulations at least 2 weeks prior to the first administration of iptacopan. If iptacopan has to start earlier than 2 weeks post vaccination, prophylactic antibiotic treatment must be initiated.
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Vaccination against Haemophilus influenzae (or a booster, if required) should be given, if available and according to local regulations, at least 2 weeks prior to first dosing.
Exclusion Criteria:
Patients eligible for this Treatment Plan must not meet any of the following criteria:
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History of hypersensitivity to any drugs or metabolites of similar chemical classes as the product.
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Renal biopsy showing interstitial fibrosis/tubular atrophy (IF/TA) or chronic allograft nephropathy of more than 50%.
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Monoclonal gammopathy of undetermined significance (MGUS) confirmed by the measurement of serum free light chains or other investigation as per local standard of care.
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Patients with an active systemic bacterial, viral or fungal infection within 14 days prior to iptacopan administration.
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The presence of fever ≥ 38°C (100.4°F) within 7 days prior to iptacopan administration.
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A history of recurrent invasive infections caused by encapsulated organisms, e.g., N. meningitidis and S. pneumoniae.
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Human immunodeficiency virus (HIV) infection
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Liver disease, such as active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection defined as HBsAg positive or HCV RNA positive, or liver injury as indicated by abnormal liver function tests prior to enrolling as defined below:
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Any single parameter of alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma-glutamyl transferase (GGT) or alkaline phosphatase must not exceed 3 x upper limit of normal (ULN)
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Serum bilirubin must not exceed 2 x upper limit of normal (ULN) (with the exception of those patients with a known confirmed diagnosis of Gilbert syndrome).
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History or current diagnosis of ECG abnormalities indicating significant risk of safety, such as clinically significant cardiac arrhythmias, e.g., sustained ventricular tachycardia and clinically significant second- or third-degree atrioventricular block (AV block) without a pacemaker.
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History of malignancy of any organ system (other than localized basal cell carcinoma of the skin or in situ cervical cancer), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases.
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The use of inhibitors of complement factors (e.g., Factor B, Factor D, C3 inhibitors, anti C5 antibodies, C5a receptor antagonists) within 6 months prior to the start of the medication.
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Participation in any investigational drug trial or use of investigational drugs at the time of iptacopan first dose, or within 5 elimination half-lives or within 30 days before the iptacopan first dose, whichever is longer; or longer if required by local regulations.
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Female patients who are pregnant or breastfeeding or intending to conceive during the course of treatment.
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Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception for the duration of the treatment and for 1 week after stopping iptacopan. Women are considered post-menopausal and not of childbearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g., age appropriate, history of vasomotor symptoms)
Effective contraception methods include:
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Total abstinence (when this is in line with the preferred and usual lifestyle). Periodic abstinence (e.g., calendar, ovulation, symptothermal, post ovulation methods) and withdrawal are not acceptable methods of contraception.
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Female sterilization (bilateral oophorectomy), total hysterectomy or tubal ligation at least six weeks before starting iptacopan. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment.
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Male sterilization (at least 6 months prior to enrollment). For female patients, the vasectomized male partner should be the sole partner.
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Barrier methods of contraception: Condom or Occlusive cap (diaphragm or cervical/vault caps).
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Oral (estrogen and progesterone), injected or implanted hormonal methods of contraception or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example hormone vaginal ring or transdermal hormone contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS). In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking investigational drug.
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Use of drugs that are strong inhibitors of cytochrome P450 enzyme (CYP2C8), that metabolizes iptacopan, are prohibited (e.g., clopidogrel).
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Use of drugs which inhibit both CYP2C8 enzyme and OATP1B1 and OATP1B3 drug transporters are prohibited (e.g., gemfibrozil).
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Severe concurrent comorbidities including but not limited to advanced cardiac disease (e.g., NYHA class IV), severe pulmonary disease (e.g., severe pulmonary hypertension (WHO class IV)) or any other condition that in the opinion of the treating physician precludes the patient's to receive iptacopan and participate in the program.
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Novartis Pharmaceuticals
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- CLNP023B12004M