Hemophilia APhase 4NCT03947567
Safety and Efficacy of Long-term Treatment With SCT800 in Previously Treated Hemophilia A Patients.
A Phase 4 study testing Recombinant Human Coagulation FVIII in people with Hemophilia A, run by Sinocelltech Ltd. at 1 site in China.
Trial summary
This study is a multi-center, open-label, extension trial to evaluate the safety, efficacy of SCT800 in long term prophylaxis and on-demand treatment in patients with severe hemophilia A who have been previously treated with coagulation factor VIII(FVIII) . Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Recombinant Human Coagulation FVIII
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 240 participants
- Where
- 1 siteChina
- Study dates
- 26 Jul 2019to 1 Apr 2025
- Phase
- Phase 4
- 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 Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male patients who are diagnosed with severe (laboratory tested FVIII:C <2%) hemophilia A;
- Patients Who Completed trial of SCT800-A302 or SCT800-A 303 and New PTP(previously treated patients)(Previously received FVIII prophylactic or bleeding treatment, have the relevant records and are verified to have accumulated exposures days( EDs) ≥150 days;
- Negative FVIII inhibitor assay results (laboratory tested Nijmegen-Bethesda assay result <0.6 BU(Bethesda unit)/mL);
- The treatment records of at least 50EDs before screening can be obtained;
- 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;
- Patients with other coagulation dysfunction diseases in addition to hemophilia A;
- 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;
Trial locations and contacts
1 site in 1 country.
Feng Xu contact
China1 site, 1 recruiting
- Recruiting
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
Tianjin
Renchi Yang
About this study
Official title: A Multicenter, Open, Extension Trial to Evaluate Safety and Efficacy of Recombinant Human Coagulation Factor VIII (SCT800) During Long Term Treatment in Previously Treated Patients With Severe Haemophilia A .
This study is a multi-center, open-label, extension trial to evaluate the safety, efficacy of SCT800 in long term prophylaxis and on-demand treatment in patients 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 V1, on-demand treatment is given when bleeding episodes occur. The study period is 120 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
Participants received SCT800 for prophylaxis and/or on-demand treatment for 120 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Recombinant Human Coagulation FVIII | Experimental | Not described Receives: Drug: Recombinant Human Coagulation FVIII |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of FVIII inhibitors
The Nijmegen-Bethesda assay shall be used to monitor the production of FVIII inhibitors during the trial.
Measured: up to 120 weeks
Secondary outcomes (3)
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 120 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: up to 120 weeks
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 120 weeks
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 start26 Jul 2019
- Primary completion1 Apr 2025 (estimated)
- Study completion1 Apr 2025 (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 4
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 240 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sinocelltech Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Renchi Yang, Principal investigator, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03947567
- Sponsor's protocol ID
- SCT800-A401
- Status last verified
- May 2019
- First submitted
- 9 May 2019
- Last updated
- 28 Apr 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open, Extension Trial to Evaluate Safety and Efficacy of Recombinant Human Coagulation Factor VIII (SCT800) During Long Term Treatment in Previously Treated Patients With Severe Haemophilia A .
- ClinicalTrials.gov ID
- NCT03947567
- Acronym / sponsor ID
- SCT800-A401
- Conditions
- Hemophilia A
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 240 participants (estimated)
- Lead sponsor
- Sinocelltech Ltd.Industry (pharma, biotech, devices)
- Study start
- 26 Jul 2019 (actual)
- Primary completion
- 1 Apr 2025 (estimated)
- Study completion
- 1 Apr 2025 (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 multi-center, open-label, extension trial to evaluate the safety, efficacy of SCT800 in long term prophylaxis and on-demand treatment in patients 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 V1, on-demand treatment is given when bleeding episodes occur. The study period is 120 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 FVIII |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Male patients who are diagnosed with severe (laboratory tested FVIII:C <2%) hemophilia A;
- Patients Who Completed trial of SCT800-A302 or SCT800-A 303 and New PTP(previously treated patients)(Previously received FVIII prophylactic or bleeding treatment, have the relevant records and are verified to have accumulated exposures days( EDs) ≥150 days;
- Negative FVIII inhibitor assay results (laboratory tested Nijmegen-Bethesda assay result <0.6 BU(Bethesda unit)/mL);
- The treatment records of at least 50EDs before screening can be obtained;
- 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;
Key exclusion criteria (6 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;
- Patients with other coagulation dysfunction diseases in addition to hemophilia A;
- 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;
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: SCT800Participants received SCT800 for prophylaxis and/or on-demand treatment for 120 weeks. | Drug | Recombinant Human Coagulation FVIII |
Primary outcome
Incidence of FVIII inhibitors
Time frame: up to 120 weeks
The Nijmegen-Bethesda assay shall be used to monitor the production of FVIII inhibitors during the trial.
3 secondary outcomes
Annualized Bleeding Rate
Time frame: up to 120 weeks
FVIII incremental in-vivo recovery
Time frame: up to 120 weeks
Bleeding event treatment efficacy
Time frame: up to 120 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2020.
Related trials for Hemophilia A
RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
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.
Feng Xu contact
Renchi Yang principal investigator
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Recombinant Human Coagulation FVIII in people with Hemophilia A, run by Sinocelltech Ltd. at 1 site in China.
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 6 inclusion and 6 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 28 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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?
Contact Renchi Yang (13512078851, rcyang65@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of FVIII inhibitors (time frame: up to 120 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 28 Apr 2020.