Dose-Ranging Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 in Subjects With Severe Hemophilia A
NCT03061201
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Adult Trials: 18+ Years
- Male ≥18 years of age
- Severe hemophilia A (past evidence of circulating FVIII activity of < 1% normal)
- Treated or exposed to FVIII concentrates or cryoprecipitate for at least 150 exposure days
- ≥12 bleeding episodes if receiving on-demand therapy over the preceding 12 months
- Agree to use double barrier contraceptive until at least 3 consecutive semen samples are negative for AAV 2/6 after SB-525 infusion
- Presence of neutralizing antibodies
- Current inhibitor, or history of FVIII inhibitor (except for transient low titer
inhibitor detected in childhood)
- History of hypersensitivity response to FVIII
- History of Hepatitis B or HIV-1/2 infection
- History of Hepatitis C, unless viral assays in two samples, collected at least 6
months apart, are negative
- Evidence of any bleeding disorder in addition to hemophilia A
- Markers of hepatic inflammation or overt or occult cirrhosis
- History of chronic renal disease or creatinine ≥ 1.5 mg/dL
- Presence of liver mass on magnetic resonance imaging (MRI), or, positive alpha
fetoprotein
- Presence of > grade 2 liver fibrosis on elastography for subjects with history of
treated Hepatitis C or suspicion of chronic liver disease
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| Descriptive Information | |||||||
|---|---|---|---|---|---|---|---|
| Brief Title ICMJE | Dose-Ranging Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 in Subjects With Severe Hemophilia A | ||||||
| Official Title ICMJE | A PHASE 1/2, OPEN-LABEL, ADAPTIVE, DOSE-RANGING STUDY TO ASSESS THE SAFETY AND TOLERABILITY OF SB-525 (PF-07055480) (RECOMBINANT AAV2/6 HUMAN FACTOR 8 GENE THERAPY) IN ADULT SUBJECTS WITH SEVERE HEMOPHILIA A | ||||||
| Brief Summary | The purpose of the study is to evaluate the safety, tolerability and time-course profile of FVIII activity levels with adaptive doses of SB-525. | ||||||
| Detailed Description | The objective of the study is to reduce or eliminate the need for FVIII replacement therapy. The proposed SB-525 clinical study uses a recombinant adeno-associated virus 2/6 (AAV2/6) vector encoding the cDNA for the B-domain deleted human F8 (hF8). The secreted FVIII has the same amino acid sequence as approved recombinant anti hemophilic factors (Refacto® and Xyntha®). The SB-525 vector encodes a liver-specific promotor module and AAV2/6 exhibits liver tropism, thus providing the potential for long-term hepatic production of FVIII in hemophilia A subjects. The constant production of FVIII after a single SB-525 administration may provide potential benefit in durable protection against bleeding and the complications thereof without lifelong repetitive IV factor replacement administration. | ||||||
| Study Type ICMJE | Interventional | ||||||
| Study Phase ICMJE | Phase 2 | ||||||
| Study Design ICMJE | Intervention Model: Sequential Assignment Intervention Model Description: Dose selection based on safety and observed FVIII levels. Masking: None (Open Label)Masking Description: Open Label Primary Purpose: Treatment
| ||||||
| Condition ICMJE | Hemophilia A | ||||||
| Intervention ICMJE | Biological: SB-525
Single dose of investigational product SB-525 | ||||||
| Study Arms ICMJE | Experimental: Sequential dose escalation
SB-525 is administered as a single infusion Intervention: Biological: SB-525 | ||||||
| Publications * | Not Provided | ||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. | |||||||
| Recruitment Information | |||||||
| Recruitment Status ICMJE | Recruiting | ||||||
| Estimated Enrollment ICMJE | 13 | ||||||
| Original Estimated Enrollment ICMJE | 20 | ||||||
| Estimated Study Completion Date ICMJE | July 23, 2024 | ||||||
| Estimated Primary Completion Date | July 23, 2024 (Final data collection date for primary outcome measure) | ||||||
| Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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| Sex/Gender ICMJE |
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| Ages ICMJE | 18 Years and older (Adult, Older Adult) | ||||||
| Accepts Healthy Volunteers ICMJE | No | ||||||
| Contacts ICMJE |
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| Listed Location Countries ICMJE | United States | ||||||
| Removed Location Countries | |||||||
| Administrative Information | |||||||
| NCT Number ICMJE | NCT03061201 | ||||||
| Other Study ID Numbers ICMJE | SB-525-1603 C3731001 ( Other Identifier: Alias Study Number ) | ||||||
| Has Data Monitoring Committee | Yes | ||||||
| U.S. FDA-regulated Product |
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| IPD Sharing Statement ICMJE |
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| Responsible Party | Pfizer | ||||||
| Study Sponsor ICMJE | Pfizer | ||||||
| Collaborators ICMJE | Not Provided | ||||||
| Investigators ICMJE |
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| PRS Account | Pfizer | ||||||
| Verification Date | August 2020 | ||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP | |||||||