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NXT007 · 4 trials · 2 indications
The vital signs that will be assessed are body temperature, pulse rate, respiratory rate, and systolic and diastolic blood pressure.
The ECG parameters that will be assessed are heart rate, PR interval, QRS interval, QT interval, and QTcF inteval.
| Arm | Type | Description |
|---|---|---|
| Main Study Treatment Period: NXT007 Prophylaxis | EXPERIMENTAL | Participants randomized to this arm will receive NXT007 prophylaxis for the main study treatment period. |
| Main Study Treatment Period: FVIII SOC Prophylaxis | ACTIVE_COMPARATOR | Participants randomized to this arm will receive FVIII standard of care (SOC) prophylaxis for the main study treatment period. |
| Open-Label Extension Period: NXT007 Prophylaxis | EXPERIMENTAL | After the main study treatment period, participants in the NXT007 arm will be able to continue with NXT007 dosing, and participants in the FVIII arm will be able to switch to NXT007, in the open-label extension period. |
| Main Study Treatment Period: Emicizumab Prophylaxis | ACTIVE_COMPARATOR | Participants randomized to this arm will receive emicizumab prophylaxis for the main study treatment period at 3 mg/kg once weekly (QW) for 4 weeks as loading doses, followed by maintenance dosing of either 1.5 mg/kg QW, 3 mg/kg once every 2 weeks (Q2W), or 6 mg/kg once every 4 weeks (Q4W). Loading doses are not required for participants who were taking emicizumab prior to study start. |
| A: Single NXT007 SC Injection Into Abdomen | EXPERIMENTAL | - |
| B: Single NXT007 SC Injection Into Upper Arm | EXPERIMENTAL | - |
| C: Single NXT007 SC Injection Into Thigh | EXPERIMENTAL | - |
| D: Single NXT007 IV Infusion | EXPERIMENTAL | - |
| Part 1: Cohort 1 - NXT007 Dose Level 1 (Low) | EXPERIMENTAL | - |
| Part 1: Cohort 2 - NXT007 Dose Level 2 | EXPERIMENTAL | - |
| Part 1: Cohort 3 - NXT007 Dose Level 3 | EXPERIMENTAL | - |
| Part 1: Cohort 4 - NXT007 Dose Level 4 | EXPERIMENTAL | - |
| Part 1: Cohort 5 - NXT007 Dose Level 5 (High) | EXPERIMENTAL | - |
| Part 2: Cohort A - NXT007 | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| NXT007 | COMBINATION_PRODUCT | NXT007 will be administered subcutaneously (SC) using an integrated drug-device combination product. |
| Human Coagulation Factor VIII | DRUG | Factor VIII (FVIII) prophylaxis standard of care (SOC) will be administered at the dose and frequency as stated in the local labels and per local country practice. |
| Emicizumab | DRUG | Emicizumab will be administered subcutaneously (SC) using vial and syringe. |
Inclusion Criteria: * Diagnosis of severe (FVIII:C \<1 IU/dL \[International Unit per decilitre\]) or moderate (FVIII:C between ≥1 IU/dL and ≤5 IU/dL) congenital hemophilia A without inhibitors against FVIII * No documented inhibitor (i.e., \<0.6 BU/mL \[Bethesda unit per millilitre\]), FVIII half-...
NXT007 is an investigational small molecule being studied for the treatment of hemophilia A. It is being evaluated in clinical trials for people with severe or moderate hemophilia A, including those aged 12 years and older. It is also being studied in healthy male participants to assess its safety and how the body processes it.
NXT007 is being developed by Roche Holding AG, which trades under the ticker RHHBY. Roche is conducting clinical trials of NXT007 in multiple countries, including the United States, Canada, Japan, and several European nations.
NXT007 is in Phase 1 clinical development. One Phase 1 trial in healthy male participants has been completed, and another Phase 1 trial in people with hemophilia A is currently recruiting. NXT007 is investigational and has not been approved by regulatory authorities.
NXT007 is being studied in several clinical trials. NCT05987449 is a Phase 1 trial in people with severe or moderate hemophilia A. NCT06189508 is a completed Phase 1 trial in healthy male participants. NCT07416526 and NCT07416604 are Phase 3 trials comparing NXT007 to factor VIII prophylaxis and emicizumab prophylaxis, respectively, in people with hemophilia A.
Yes, one clinical trial of NXT007 includes children. NCT05987449, a Phase 1 study in people with hemophilia A, enrolls participants as young as 2 years old. Other trials of NXT007 enroll participants aged 12 years and older.
In clinical trials, NXT007 is given as a subcutaneous injection. One completed study evaluated single subcutaneous doses of NXT007 administered at different injection sites, including the abdomen, upper arm, and thigh, in healthy male participants.