A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT03061201 |
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Recruitment Status :
Active, not recruiting
First Posted : February 23, 2017
Last Update Posted : June 1, 2023
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Hemophilia A | Biological: SB-525 (PF-07055480) | Phase 2 |
The proposed 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 (PF-07055480) 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 (PF-07055480) administration may provide potential benefit in durable protection against bleeding and the complications thereof without lifelong repetitive IV factor replacement administration.
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 11 participants |
| Allocation: | N/A |
| Intervention Model: | Sequential Assignment |
| Intervention Model Description: | Dose selection based on safety and kinetics of circulating FVIII levels observed in previously dosed participants. |
| Masking: | None (Open Label) |
| Masking Description: | Open Label |
| Primary Purpose: | Treatment |
| Official Title: | 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 |
| Actual Study Start Date : | June 21, 2017 |
| Estimated Primary Completion Date : | July 25, 2024 |
| Estimated Study Completion Date : | July 25, 2024 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: Sequential dose escalation
SB-525 (PF-07055480) is administered as a single infusion
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Biological: SB-525 (PF-07055480)
Single dose of investigational product SB-525 (PF-07055480) |
- Incidence of adverse events and serious adverse events [ Time Frame: Up to 5 years after SB-525 (PF-07055480) infusion ]
- Changes in circulating FVIII activity [ Time Frame: Up to 5 years after SB-525 (PF-07055480) infusion ]
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | Male |
| Gender Based Eligibility: | Yes |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03061201
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| Study Director: | Pfizer CT.gov Call Center | Pfizer |
| Responsible Party: | Pfizer |
| ClinicalTrials.gov Identifier: | NCT03061201 |
| Other Study ID Numbers: |
SB-525-1603 C3731001 ( Other Identifier: Alias Study Number ) |
| First Posted: | February 23, 2017 Key Record Dates |
| Last Update Posted: | June 1, 2023 |
| Last Verified: | May 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Yes |
| Plan Description: | Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests. |
| URL: | https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
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Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn |