Hemophilia APhase 3NCT03947320
Safety,Efficacy and Pharmacokinetics Evaluation of SCT800 in Previously Treated Paediatric Patients With Severe Haemophilia A.
A Phase 3 study testing Recombinant Human Coagulation FVIII in people with Hemophilia A, run by Sinocelltech Ltd..
Trial summary
This study is a multicenter phase III uncontrolled open-label trial to evaluate the efficacy,safety and pharmacokinetics of SCT800 in regular prophylaxis and perioperative treatment in patients (<12 years old) with severe hemophilia A who have been previously treated with coagulation factor VIII(FVIII) . Read more about this study
- Who is it for?
- Ages Up to 12 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Recombinant Human Coagulation FVIII
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- No sites listed yet
- Study dates
- 20 Dec 2019to 20 Sep 2020
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 12 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Aged <12 years ;
- Male severe (central laboratory tested FVIII:C <1%) hemophilia A patients;
- Previously received FVIII treatment (prophylactic or bleeding treatment), have the relevant records and are verified to have accumulated EDs ≥150 days（6≤age<12years old）and EDs >50 days（age <6 years old）;
- The bleeding treatment records of at least 3 months before screening can be obtained;
- Negative FVIII inhibitor assay results (laboratory tested Nijmegen-Bethesda assay result <0.6 BU/mL);
- HIV negative; if HIV positive, the viral load <200 particles/uL or <400,000 copies/mL, and HIV patients must satisfy CD4+ count >200/μL;
- The patient or his guardian voluntarily signed the Informed Consent Form.
Who cannot join
- Known allergy to any coagulation factor VIII or any excipient; known allergy to bovine, rodent or hamster bovine;
- Has a history or family history of blood coagulation factor VIII inhibitor;
- Platelet count <100 × 109/L;
- Clinical liver function test ((glutamic-pyruvic transaminase, glutamic-pyruvic transaminase) ≥ five times the upper limit of normal (ULN) or clinical kidney function test （creatinine) ≥ two times the ULN;
- International normalized ratio (INR) >1.5;
- Patients with other coagulation dysfunction diseases in addition to hemophilia A;
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- Patients who used any anticoagulant or anti-platelet treatment (including non-steroidal anti-inflammatory drugs [NSAIDs]) within 1 weeks before the first drug administration or who regularly (e.g., daily, every other day) use anticoagulant or anti-platelet treatment within the clinical trial period;
- Patients who used immunomodulator(e.g., immunoglobulin, corticosteroids,alpha-interferon, prednisone [>10 mg/day and >7 days], or comparable drugs, other than anti-retroviral chemotherapy) within two weeks before the first administration of the study drug or during the clinical trial period;
- Administration of any cryoprecipitate, whole blood or plasma within 30 days prior to administration of study drug.
- Patients with other clinically significant diseases, alcoholism, drug abuse, mental disorders or intellectual disabilities;
- Patients with other severe or clinical significant diseases verified by the investigator to be unable to benefit from the clinical study;
- Patients who participated in other clinical studies within one month before the first drug administration (except FVIII trials) and patients who participated in other FVIII clinical trials after signing the Informed Consent Form;
About this study
Official title: A Multicenter Phase III Uncontrolled Open-label Trial to Evaluate Safety and Efficacy and Pharmacokinetics of Recombinant Human Coagulation Factor VIII (SCT800) in Previously Treated Paediatric Patients With Severe Haemophilia A.
This study is a multicenter phase III uncontrolled open-label trial to evaluate the efficacy,safety and pharmacokinetics of SCT800 in regular prophylaxis and perioperative treatment in patients (<12 years old) with severe hemophilia A who have been previously treated with coagulation factor VIII(FVIII) . This study includes two phases: the screening period and prophylaxis period.Prophylaxis with 25 - 50 IU/kg of SCT800 shall be administered once every other day or three times per week starting from Visit 1 and prophylaxis with SCT800 shall continue for 24 consecutive weeks.
- Conditions
- Hemophilia A
- Being tested
- Recombinant Human Coagulation FVIII
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant Human Coagulation FVIIIDrug
Also called SCT800
Participant received SCT800 for prophylaxis with 25 - 50 IU/kg injection once every other day or three times per week for 6 months.
| Study group | Role | What they receive |
|---|---|---|
| Recombinant Human Coagulation FVIII | Experimental | Participant receivedSCT800 for prophylaxis with 25 - 50 IU/kg injection once every other day or three times per week for 6 months. Receives: Drug: Recombinant Human Coagulation FVIII |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Bleeding Rate
Annualized Bleeding Rate（ABR） can be calculated using the following formula: Number of bleeding events in efficacy evaluation period/(number of days in treatment period/365.25)
Measured: up to 24 weeks
Secondary outcomes (5)
Annualized joint bleeding rate
Annualized joint bleeding rate（AJBR） can be calculated using the following formula: Number of joint bleeding events during efficacy evaluation period/(number of days in treatment period/365.25).
Measured: up to 24 weeks
FVIII incremental in-vivo recovery
Incremental recovery is determined as the peak factor level recorded in the first hour after infusion and is reported as [IU/ml]/[IU/kg]
Measured: Predose within 30 min，15 min±2 min
Bleeding event treatment efficacy
The investigator shall evaluate the hemostatic effect after the treatment of every bleeding event of subjects based on a four-point scale(excellent, good, moderate, not relieved).
Measured: up to 24 weeks
Elimination Half Life
t1/2; Chromogenic Assay
Measured: Predose within 30 min，15 min±2 min、1 hour±5 min，10 hours±30 min，24 hours±1hours and 48 hours±2 hours post-dose
Clearance
CL; Chromogenic Assay
Measured: Predose within 30 min，15 min±2 min、1 hour±5 min，10 hours±30 min，24 hours±1hours and 48 hours±2 hours post-dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2019
- Study start20 Dec 2019 (estimated)
- Primary completion20 Sep 2020 (estimated)
- Study completion20 Sep 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by Sinocelltech Ltd.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sinocelltech Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03947320
- Sponsor's protocol ID
- SCT800-A303
- Status last verified
- May 2019
- First submitted
- 9 May 2019
- Last updated
- 13 May 2019
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Phase III Uncontrolled Open-label Trial to Evaluate Safety and Efficacy and Pharmacokinetics of Recombinant Human Coagulation Factor VIII (SCT800) in Previously Treated Paediatric Patients With Severe Haemophilia A.
- ClinicalTrials.gov ID
- NCT03947320
- Acronym / sponsor ID
- SCT800-A303
- Conditions
- Hemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- Sinocelltech Ltd.Industry (pharma, biotech, devices)
- Study start
- 20 Dec 2019 (estimated)
- Primary completion
- 20 Sep 2020 (estimated)
- Study completion
- 20 Sep 2020 (estimated)
- First posted
- 13 May 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is a multicenter phase III uncontrolled open-label trial to evaluate the efficacy,safety and pharmacokinetics of SCT800 in regular prophylaxis and perioperative treatment in patients (<12 years old) with severe hemophilia A who have been previously treated with coagulation factor VIII(FVIII) . This study includes two phases: the screening period and prophylaxis period.Prophylaxis with 25 - 50 IU/kg of SCT800 shall be administered once every other day or three times per week starting from Visit 1 and prophylaxis with SCT800 shall continue for 24 consecutive weeks.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Recombinant Human Coagulation FVIII | Experimental | Drug: Recombinant Human Coagulation FVIIIParticipant receivedSCT800 for prophylaxis with 25 - 50 IU/kg injection once every other day or three times per week for 6 months. |
Who may qualify
- Ages
- Up to 12 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Aged <12 years ;
- Male severe (central laboratory tested FVIII:C <1%) hemophilia A patients;
- Previously received FVIII treatment (prophylactic or bleeding treatment), have the relevant records and are verified to have accumulated EDs ≥150 days（6≤age<12years old）and EDs >50 days（age <6 years old）;
- The bleeding treatment records of at least 3 months before screening can be obtained;
- Negative FVIII inhibitor assay results (laboratory tested Nijmegen-Bethesda assay result <0.6 BU/mL);
Key exclusion criteria (12 listed)
- Known allergy to any coagulation factor VIII or any excipient; known allergy to bovine, rodent or hamster bovine;
- Has a history or family history of blood coagulation factor VIII inhibitor;
- Platelet count <100 × 109/L;
- Clinical liver function test ((glutamic-pyruvic transaminase, glutamic-pyruvic transaminase) ≥ five times the upper limit of normal (ULN) or clinical kidney function test （creatinine) ≥ two times the ULN;
- International normalized ratio (INR) >1.5;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Recombinant Human Coagulation FVIIIAlso: SCT800Participant received SCT800 for prophylaxis with 25 - 50 IU/kg injection once every other day or three times per week for 6 months. | Drug | Recombinant Human Coagulation FVIII |
Primary outcome
Annualized Bleeding Rate
Time frame: up to 24 weeks
Annualized Bleeding Rate（ABR） can be calculated using the following formula: Number of bleeding events in efficacy evaluation period/(number of days in treatment period/365.25)
5 secondary outcomes
Annualized joint bleeding rate
Time frame: up to 24 weeks
FVIII incremental in-vivo recovery
Time frame: Predose within 30 min，15 min±2 min
Bleeding event treatment efficacy
Time frame: up to 24 weeks
Elimination Half Life
Time frame: Predose within 30 min，15 min±2 min、1 hour±5 min，10 hours±30 min，24 hours±1hours and 48 hours±2 hours post-dose
Clearance
Time frame: Predose within 30 min，15 min±2 min、1 hour±5 min，10 hours±30 min，24 hours±1hours and 48 hours±2 hours post-dose
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 May 2019.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Recombinant Human Coagulation FVIII in people with Hemophilia A, run by Sinocelltech Ltd..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 13 May 2019. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized Bleeding Rate (time frame: up to 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 May 2019.