Nomacopan (rVA576) in Transplant Associated Thrombotic Microangiopathy
Study Details
Study Description
Brief Summary
Multicentre Study of nomacopan in Paediatric Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
Detailed Description
This is an open-label, multi-centre study of two-parts, Part A and B, includes 24 weeks of treatment, safety follow up after 30 days.
Part A: dose algorithm, safety and efficacy
Part B: safety and efficacy
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: nomacopan (rVA576) The study population will consist of paediatric patients who have undergone allogeneic or autologous HSCT and develop HSCT-TMA within 100 days of HSCT |
Drug: nomacopan (rVA576)
The study population will consist of paediatric patients who have undergone allogeneic or autologous HSCT and develop HSCT-TMA within 100 days of HSCT
|
Outcome Measures
Primary Outcome Measures
- RBC transfusion independence for ≥ 28 days immediately prior to any scheduled clinical visit up to Week 24 [24 weeks]
Transfusion independence is defined as no RBC or platelet transfusion attributable to, or required to manage, thrombotic microangiopathy (TMA). Transfusions required for causes other than TMA will not be considered within the evaluation of the primary efficacy endpoints.
- Urine protein creatinine ratio ≤ 2 mg/mg [24 weeks]
Urine protein creatinine ratio ≤ 2 mg/mg
Secondary Outcome Measures
- Renal Function Improvement [24 weeks]
Percentage of patients who achieve the primary endpoint of urine protein creatinine ratio ≤ 2 mg/mg (the nephrotic threshold) for ≥ 28 days
- Platelet transfusion independence [24 weeks]
Platelet transfusion independence for ≥ 28 days within the 24 week timeframe
- Normalisation of lab parameters [24 weeks]
Serum sC5b-9 ≤ ULN
- Normalisation of lab parameters [24 weeks]
Lactate dehydrogenase (LDH) ≤ULN
- Normalisation of lab parameters [24 weeks]
Normalization of haptoglobin
- Safety and tolerability of nomacopan [28 weeks]
New or worsening AEs after dosing of investigational product will be recorded in the eCRF.
- Safety and tolerability of nomacopan [28 weeks]
Listings of subjects who have an SAE.
- Safety and tolerability of nomacopan [28 weeks]
Listings of subjects who discontinue from the study due to an AE.
- Safety and tolerability of nomacopan [28 weeks]
Occurrence of significant laboratory abnormalities will be summarized.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Aged ≥ 0.5 and < 18 years at the time of diagnosis of TMA.
-
Undergone allogeneic or autologous HSCT.
-
TMA diagnosis within 100 days of their first allogeneic or autologous HSCT.
-
Clinical or histological diagnosis of TMA
-
Provision of written informed consent.
-
Provision of informed assent
Exclusion Criteria:
-
Patients weighing less than 5 kg.
-
Patients with a positive direct Coomb's test.
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Patients who do not receive nomacopan within 14 days of the initial diagnosis of TMA.
-
Patients having an active systemic or organ system bacterial or fungal infection or progressive severe infection
-
Grade 4 Acute GVHD
-
Received eculizumab or any other complement blocker therapy at any time.
-
Known hypersensitivity to the active ingredient or excipients
-
A positive ADAMTS13 test (<10%),
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Duke University Medical Center, Children's Health Center | Durham | North Carolina | United States | 27710 |
Sponsors and Collaborators
- AKARI Therapeutics
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- AK901