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| ID | Type | Description | Link |
|---|---|---|---|
| NCT03362502 | Registry Identifier | ClinicalTrials.gov |
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All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in 1 combined study: C3391003
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This is a first-in-human/first-in-patient, multi-center, open-label, non-randomized, ascending dose, safety and tolerability study of a single intravenous infusion of PF-06939926 in ambulatory and non-ambulatory subjects with Duchenne muscular dystrophy (DMD). Other objectives include measurement of dystrophin expression and distribution, and assessments of muscle strength, quality, and function.
A total of approximately 22 subjects will receive PF-06939926, and these will include both ambulatory and non-ambulatory subjects. Up to 13 subjects may be included in a cohort that includes the concomitant medication, sirolimus. In order to mitigate unanticipated risks to subject safety, enrollment will be staggered within and between two planned dose-levels and will include a formal review by an external data monitoring committee (E-DMC) prior to dose progression.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| PF-06939926 | Experimental |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PF-06939926 | Genetic | Recombinant adeno-associated virus, serotype 9 (AAV9) carrying a truncated human dystrophin gene (mini-dystrophin) under the control of a muscle-specific promoter. Subjects will receive a single intravenous infusion of one of 2 dose levels. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs) for 1-Year Follow-Up | An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a treatment-emergent AE (TEAE). A serious AE (SAE) was an untoward medical occurrence that resulted in death, was life-threatening (immediate risk of death), required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in congenital anomaly/birth defect, or was considered to be an important medical event. Severe AE=an event that prevents normal everyday activities. | Baseline up to 1 year post dose of PF-06939926 |
| Number of Participants With All-Causality TEAEs by System Organ Class (SOC) and Preferred Term (PT) for 1-Year Follow-Up | An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. For this outcome measure, TEAEs were reported according to Medical Dictionary for Regulatory Activities (MedDRA) version 25.0 SOC and PT. | Baseline up to 1 year post dose of PF-06939926 |
| Number of Participants With Treatment-Related TEAEs for 1-Year Follow-Up | An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. An SAE was an untoward medical occurrence that resulted in death, was life-threatening (immediate risk of death), required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in congenital anomaly/birth defect, or was considered to be an important medical event. Severe AE=an event that prevents normal everyday activities. Treatment-related AE was any untoward medical occurrence attributed to study treatment in a participant who received study treatment. Treatment-related AEs and SAEs were determined by the investigator. |
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Inclusion Criteria:
Age as follows, based on ambulatory status:
Diagnosis of Duchenne muscular dystrophy confirmed by medical history and genetic testing;
Receipt of glucocorticoids for 6 months and a stable daily dose for at least 3 months prior to study entry;
Ability to tolerate magnetic resonance imaging (MRI) without sedation and with no contraindications to these procedures;
Ability to tolerate muscle biopsies under anesthesia with no contraindications to these procedures;
Body weights as follows, based on ambulatory status:
Functional performance as follows, based on ambulatory status:
Exclusion Criteria:
Receipt of live attenuated vaccination within 3 months prior to receiving PF-06939926 or exposure to an influenza (or other inactivated) vaccination or systemic antiviral and/or interferon therapy within 30 days prior to receipt of PF-06939926;
Prior exposure to any gene therapy agent, including exon-skipping agents;
Exposure to other investigational drugs within 30 days or 5 half-lives, whichever is longer;
Neutralizing antibodies (NAb) against adeno-associated virus, serotype 9 (AAV9);
Compromised cardiac function as indicated by left ventricular ejection fraction on cardiac MRI, as follows, based on ambulatory status:
Inadequate hepatic or renal function or risk factors for autoimmune disease on screening laboratory assessments.
The following genetic abnormalities in the dystrophin gene as confirmed by the investigator based on the review of the DMD genetic testing:
Sirolimus Cohort
Inclusion Criteria
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| MRI Research Center | Los Angeles | California | 90095 | United States | ||
| Reed Neurological Research Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40753271 | Derived | Walsh J, Palandra J, Duriga N, Beidler D, McIntosh A, Binks M, Neubert H. Dystrophin/mini-dystrophin expression analysis by immunoaffinity liquid chromatography-tandem mass spectrometry after gene therapy for DMD. Gene Ther. 2025 Dec;32(6):573-580. doi: 10.1038/s41434-025-00554-5. Epub 2025 Aug 2. | |
| 40583275 | Derived |
| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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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.
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One participant was enrolled but not dosed due to a pause in enrollment. The participant was considered as an early termination due to screening window running out. The participant was subsequently re-screened and assigned to study intervention with another participant ID. Therefore, a total of 22 unique participants were enrolled as of 30 Sep 2022.
The study reached its primary completion date (PCD) on 28 Mar 2022. The data cutoff for PCD was on 30 Sep 2022. As of 30 Sep 2022, a total of 22 participants, including 19 ambulatory participants and 3 non-ambulatory participants, were enrolled and assigned to treatment.
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| ID | Title | Description |
|---|---|---|
| FG000 | PF-06939926: Low Dose, Ambulatory Cohort | Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. |
| FG001 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 7, 2022 | Mar 27, 2023 |
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| Baseline up to 1 year post dose of PF-06939926 |
| Number of Participants With Treatment-Related TEAEs by SOC and PT for 1-Year Follow-Up | An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. For this outcome measure, TEAEs were reported according to MedDRA version 25.0 SOC and PT. Treatment-related AE was any untoward medical occurrence attributed to study treatment in a participant who received study treatment. Treatment-related AEs were determined by the investigator. | Baseline up to 1 year post dose of PF-06939926 |
| Number of Participants With All-Causality SAEs by SOC and PT for 1-Year Follow-Up | An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. An SAE was an untoward medical occurrence that resulted in death, was life-threatening (immediate risk of death), required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in congenital anomaly/birth defect, or was considered to be an important medical event. For this outcome measure, TEAEs were reported according to MedDRA version 25.0 SOC and PT. | Baseline up to 1 year post dose of PF-06939926 |
| Number of Participants With Treatment-Related SAEs by SOC and PT for 1-Year Follow-Up | An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. An SAE was an untoward medical occurrence that resulted in death, was life-threatening (immediate risk of death), required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in congenital anomaly/birth defect, or was considered to be an important medical event. Treatment-related AE was any untoward medical occurrence attributed to study treatment in a participant who received study treatment. Treatment-related AEs and SAEs were determined by the investigator. For this outcome measure, TEAEs were reported according to MedDRA version 25.0 SOC and PT. | Baseline up to 1 year post dose of PF-06939926 |
| Number of Participants With Laboratory Test Abnormalities Without Regard to Baseline Abnormality for 1-Year Follow-Up - Hematology | Following hematologic parameters were analyzed for laboratory examination: hemoglobin, hematocrit, red blood cell (RBC) count, platelet count, white blood cell count, total neutrophils, absolute neutrophils (ANC), eosinophils, monocytes, basophils, lymphocytes, red blood cell indices, and haptoglobin. Laboratory abnormalities with occurrence in at least 1 participant are reported for this outcome measure. Baseline was defined as the last pre-dose measurement. | Baseline up to 1 year post dose of PF-06939926 |
| Number of Participants With Laboratory Test Abnormalities Without Regard to Baseline Abnormality for 1-Year Follow-Up - Clinical Chemistry | Following parameters for clinical chemistry were analyzed for laboratory examination: blood urea nitrogen (BUN) and creatinine, glucose, calcium, sodium, potassium, chloride, total CO2 (bicarbonate), aspartate aminotransferase (AST), alanine aminotransferase (ALT), total bilirubin (direct and indirect bilirubin), alkaline phosphatase, uric acid, albumin, total protein, serum phosphorus, cystatin C, creatine kinase, creatine kinase myocardial b fraction (CK-MB), amylase, lipase, gamma-glutamyl transferase (GGT), C-reactive protein (CRP), and cardiac troponin I. Laboratory abnormalities with occurrence in at least 1 participant are reported for this outcome measure. Baseline was defined as the last pre-dose measurement. | Baseline up to 1 year post dose of PF-06939926 |
| Number of Participants With Laboratory Test Abnormalities Without Regard to Baseline Abnormality for 1-Year Follow-Up - Urinalysis | Following parameters for urinalysis were analyzed for laboratory examination: pH, glucose, protein, blood, ketones, nitrites, leukocyte esterase, and microscopy. Laboratory abnormalities with occurrence in at least 1 participant are reported for this outcome measure. Baseline was defined as the last pre-dose measurement. | Baseline up to 1 year post dose of PF-06939926 |
| Change From Baseline on Left Ventricular Ejection Fraction as Measured by Cardiac Magnetic Resonance Imaging (MRI) for 1-Year Follow-Up | Cardiac MRI was performed after thigh and upper limb MRI or on separate days within the required visit window. Change in LVEF is presented as percentages, and was calculated as [(stroke volume) / (end-diastolic volume)]*100%. Baseline is defined as the last pre-dose measurement. Change from baseline on Day 360 (ie, 1 year post dose) was reported for this outcome measure. | Baseline, 1 year post dose of PF-06839926 (Day 360) |
| Number of Participants With Electrocardiograms (ECGs) Meeting the Pre-Defined Categorical Criteria for 1-Year Follow-Up | Triplicate ECG was performed after the participant had rested quietly for at least 10 minutes in a supine position. The pre-defined categorical criteria include PR interval aggregate (msec): value>=300, baseline>200 and percent change>=25%, baseline<=200 and percent change>=50%; QRS duration aggregate (msec): value>=140, percent change>=50%; QTcF interval aggregate (msec): 450<=value<480, 480<=value<500, value>=500, 30<=change<60, change>=60. Only the category(ies) with at least 1 participant meeting the pre-defined criterion is/are reported for this outcome measure. Baseline for ECG was defined as the measurement before the Day 1 study drug administration. | Baseline up to 1 year post dose of PF-06939926 |
| Number of Participants With Positive Responses on Columbia-Suicide Severity Rating Scale (C-SSRS) According to the Columbia Classification Algorithm of Suicide Assessment (C-CASA) Categories | C-SSRS is a participant rated questionnaire to assess whether participant experienced the following: completed suicide(1), suicide attempt(2)("Yes" on "actual attempt"), preparatory acts toward imminent suicidal behavior(3)("Yes" on "preparatory acts or behavior"), suicidal ideation(4)("Yes" on "wish to be dead","non-specific active suicidal thoughts","active suicidal ideation with methods without intent to act or some intent to act, without specific plan or with specific plan and intent), any suicidal behavior or ideation, self-injurious behavior(7)("Yes" on "Has subject engaged in non-suicidal self-injurious behavior"). Yes/No responses are mapped to C-CASA categories. C-SSRS was conducted with the participant's care giver/legal guardian on the participant's behalf throughout the study for participants<=12 years of age at screening. Baseline is defined as the last predose measurement. Number of participants with responses of "Yes" is reported for this outcome measure. | Baseline, Day 7, Day 14, Day 180, and Day 360 |
| Number of Participants With Vital Signs Meeting the Pre-Defined Categorical Criteria for 1-Year Follow-Up | Vital signs consisted of blood pressure, respiratory rate, and heart rate, was obtained with these measurements at the following timepoints: within 30 minutes before and approximately 30 minutes, 1, 2, 4, 8, and 24 hours after the start of the infusion, and on Days 4, 7, 10, 14, 30, 90, 180, and 360. Baseline was defined as the last pre-dose recording. Number of participants who had at least 1 vital sign measurement at the specified timepoints meeting the pre-defined criteria from baseline up to the end of 1-year follow-up is reported for this outcome measure. | Baseline up to 1 year post dose of PF-06939926 |
| Los Angeles |
| California |
| 90095 |
| United States |
| Ronald Reagan UCLA Medical Center (Investigational Drug Section) | Los Angeles | California | 90095 | United States |
| Ronald Reagan UCLA Medical Center - Interventional Radiology | Los Angeles | California | 90095 | United States |
| Ronald Reagan UCLA Medical Center Drug Information Center | Los Angeles | California | 90095 | United States |
| UCLA (David Geffen School of Medicine) | Los Angeles | California | 90095 | United States |
| UCLA Children's Heart Center | Los Angeles | California | 90095 | United States |
| UCLA Mattel Children's Hospital | Los Angeles | California | 90095 | United States |
| UCLA Medical Center | Los Angeles | California | 90095 | United States |
| UCLA Outpatient Surgery Center | Los Angeles | California | 90095 | United States |
| Duke Neurology | Durham | North Carolina | 27705 | United States |
| Duke University Medical Center, Lenox Baker Children's Hospital | Durham | North Carolina | 27705 | United States |
| Biospecimen Repository & Processing Core - BPRC | Durham | North Carolina | 27710 | United States |
| Duke Cardiovascular Magnetic Resonance Center | Durham | North Carolina | 27710 | United States |
| Duke Children's Hospital & Health Center | Durham | North Carolina | 27710 | United States |
| Duke University Hospital Investigational Drug Services (IDS) Pharmacy | Durham | North Carolina | 27710 | United States |
| CCTS Clinical Research Center | Salt Lake City | Utah | 84108 | United States |
| University of Utah Imaging and Neurosciences Center | Salt Lake City | Utah | 84108 | United States |
| University of Utah Hospital & Clinics Investigational Drug Services | Salt Lake City | Utah | 84112 | United States |
| University of Utah Hospital | Salt Lake City | Utah | 84112 | United States |
| Primary Children's Hospital | Salt Lake City | Utah | 84113 | United States |
| University of Utah Clinical Neurosciences Center | Salt Lake City | Utah | 84132 | United States |
| Byrne BJ, Butterfield RJ, Shieh PB, Smith EC, Licht C, Binks M, Casinghino S, Delnomdedieu M, Ravindra KC, McDonnell T, Ryan K, Schulz M, Shen Q, Shi H, Sirivelu MP, Vaidya VS, Whiteley L, Levy DI. Complement activation in a phase Ib study of fordadistrogene movaparvovec for Duchenne muscular dystrophy. Mol Ther. 2025 Sep 3;33(9):4226-4238. doi: 10.1016/j.ymthe.2025.06.032. Epub 2025 Jun 28. |
| 40583273 | Derived | Sherlock SP, Levy DI, McIntosh A, Shieh PB, Smith EC, McDonnell TG, Ryan KA, Delnomdedieu M, Binks M, Lal AK, Butterfield RJ. Cardiac safety of fordadistrogene movaparvovec gene therapy in Duchenne muscular dystrophy: Initial observations from a phase 1b trial. Mol Ther. 2025 Sep 3;33(9):4216-4225. doi: 10.1016/j.ymthe.2025.06.031. Epub 2025 Jun 28. |
| 40579547 | Derived | Butterfield RJ, Shieh PB, Li H, Binks M, McDonnell TG, Ryan KA, Delnomdedieu M, Belluscio BA, Neelakantan S, Levy DI, Schwartz PF, Smith EC. AAV mini-dystrophin gene therapy for Duchenne muscular dystrophy: a phase 1b trial. Nat Med. 2025 Aug;31(8):2712-2721. doi: 10.1038/s41591-025-03750-3. Epub 2025 Jun 27. |
| PF-06939926: High Dose, Ambulatory Cohort |
Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
| FG002 | PF-06939926: High Dose, Non-Ambulatory Cohort | Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
| COMPLETED | Completed indicates participants completed the 1-year follow-up as of data cutoff on 30 Sep 2022. |
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| NOT COMPLETED |
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Includes all enrolled participants who received PF-06939926 as of the PCD data cutoff (30 Sep 2022). Participants were analyzed according to the intervention they actually received.
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| ID | Title | Description |
|---|---|---|
| BG000 | PF-06939926: Low Dose, Ambulatory Cohort | Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. |
| BG001 | PF-06939926: High Dose, Ambulatory Cohort | Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
| BG002 | PF-06939926: High Dose, Non-Ambulatory Cohort | Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Count of Participants | Participants |
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| Age, Customized | Median | Full Range | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Mutation Type | Confirmation by genetic testing of the diagnosis of duchenne muscular dystrophy (DMD) was reported from an appropriate regulated laboratory using a clinically validated genetic test. | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||
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| Primary | Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs) for 1-Year Follow-Up | An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a treatment-emergent AE (TEAE). A serious AE (SAE) was an untoward medical occurrence that resulted in death, was life-threatening (immediate risk of death), required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in congenital anomaly/birth defect, or was considered to be an important medical event. Severe AE=an event that prevents normal everyday activities. | Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. | Posted | Count of Participants | Participants | Baseline up to 1 year post dose of PF-06939926 |
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| Primary | Number of Participants With All-Causality TEAEs by System Organ Class (SOC) and Preferred Term (PT) for 1-Year Follow-Up | An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. For this outcome measure, TEAEs were reported according to Medical Dictionary for Regulatory Activities (MedDRA) version 25.0 SOC and PT. | Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. | Posted | Count of Participants | Participants | Baseline up to 1 year post dose of PF-06939926 |
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| Primary | Number of Participants With Treatment-Related TEAEs for 1-Year Follow-Up | An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. An SAE was an untoward medical occurrence that resulted in death, was life-threatening (immediate risk of death), required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in congenital anomaly/birth defect, or was considered to be an important medical event. Severe AE=an event that prevents normal everyday activities. Treatment-related AE was any untoward medical occurrence attributed to study treatment in a participant who received study treatment. Treatment-related AEs and SAEs were determined by the investigator. | Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. | Posted | Count of Participants | Participants | Baseline up to 1 year post dose of PF-06939926 |
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| Primary | Number of Participants With Treatment-Related TEAEs by SOC and PT for 1-Year Follow-Up | An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. For this outcome measure, TEAEs were reported according to MedDRA version 25.0 SOC and PT. Treatment-related AE was any untoward medical occurrence attributed to study treatment in a participant who received study treatment. Treatment-related AEs were determined by the investigator. | Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. | Posted | Count of Participants | Participants | Baseline up to 1 year post dose of PF-06939926 |
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| Primary | Number of Participants With All-Causality SAEs by SOC and PT for 1-Year Follow-Up | An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. An SAE was an untoward medical occurrence that resulted in death, was life-threatening (immediate risk of death), required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in congenital anomaly/birth defect, or was considered to be an important medical event. For this outcome measure, TEAEs were reported according to MedDRA version 25.0 SOC and PT. | Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. | Posted | Count of Participants | Participants | Baseline up to 1 year post dose of PF-06939926 |
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| Primary | Number of Participants With Treatment-Related SAEs by SOC and PT for 1-Year Follow-Up | An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. An SAE was an untoward medical occurrence that resulted in death, was life-threatening (immediate risk of death), required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in congenital anomaly/birth defect, or was considered to be an important medical event. Treatment-related AE was any untoward medical occurrence attributed to study treatment in a participant who received study treatment. Treatment-related AEs and SAEs were determined by the investigator. For this outcome measure, TEAEs were reported according to MedDRA version 25.0 SOC and PT. | Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. | Posted | Count of Participants | Participants | Baseline up to 1 year post dose of PF-06939926 |
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| Primary | Number of Participants With Laboratory Test Abnormalities Without Regard to Baseline Abnormality for 1-Year Follow-Up - Hematology | Following hematologic parameters were analyzed for laboratory examination: hemoglobin, hematocrit, red blood cell (RBC) count, platelet count, white blood cell count, total neutrophils, absolute neutrophils (ANC), eosinophils, monocytes, basophils, lymphocytes, red blood cell indices, and haptoglobin. Laboratory abnormalities with occurrence in at least 1 participant are reported for this outcome measure. Baseline was defined as the last pre-dose measurement. | Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. Number of participants analyzed represents the total number of participants in each treatment group in the analysis population for this outcome measure. Number analyzed represents the number of participants who had reportable data for this outcome measure. | Posted | Count of Participants | Participants | Baseline up to 1 year post dose of PF-06939926 |
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| Primary | Number of Participants With Laboratory Test Abnormalities Without Regard to Baseline Abnormality for 1-Year Follow-Up - Clinical Chemistry | Following parameters for clinical chemistry were analyzed for laboratory examination: blood urea nitrogen (BUN) and creatinine, glucose, calcium, sodium, potassium, chloride, total CO2 (bicarbonate), aspartate aminotransferase (AST), alanine aminotransferase (ALT), total bilirubin (direct and indirect bilirubin), alkaline phosphatase, uric acid, albumin, total protein, serum phosphorus, cystatin C, creatine kinase, creatine kinase myocardial b fraction (CK-MB), amylase, lipase, gamma-glutamyl transferase (GGT), C-reactive protein (CRP), and cardiac troponin I. Laboratory abnormalities with occurrence in at least 1 participant are reported for this outcome measure. Baseline was defined as the last pre-dose measurement. | Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. Number of participants analyzed represents the total number of participants in each treatment group in the analysis population for this outcome measure. Number analyzed represents the number of participants who had reportable data for this outcome measure. | Posted | Count of Participants | Participants | Baseline up to 1 year post dose of PF-06939926 |
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| Primary | Number of Participants With Laboratory Test Abnormalities Without Regard to Baseline Abnormality for 1-Year Follow-Up - Urinalysis | Following parameters for urinalysis were analyzed for laboratory examination: pH, glucose, protein, blood, ketones, nitrites, leukocyte esterase, and microscopy. Laboratory abnormalities with occurrence in at least 1 participant are reported for this outcome measure. Baseline was defined as the last pre-dose measurement. | Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. Number of participants analyzed represents the total number of participants in each treatment group in the analysis population for this outcome measure. Number analyzed represents the number of participants who had reportable data for this outcome measure. | Posted | Count of Participants | Participants | Baseline up to 1 year post dose of PF-06939926 |
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| Primary | Change From Baseline on Left Ventricular Ejection Fraction as Measured by Cardiac Magnetic Resonance Imaging (MRI) for 1-Year Follow-Up | Cardiac MRI was performed after thigh and upper limb MRI or on separate days within the required visit window. Change in LVEF is presented as percentages, and was calculated as [(stroke volume) / (end-diastolic volume)]*100%. Baseline is defined as the last pre-dose measurement. Change from baseline on Day 360 (ie, 1 year post dose) was reported for this outcome measure. | Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. Number of participants analyzed represents the total number of participants in each treatment group in the analysis population and had reportable data for this outcome measure. | Posted | Mean | Standard Deviation | Percentage | Baseline, 1 year post dose of PF-06839926 (Day 360) |
| ||||||||||||||||||||||||||||||||||||
| Primary | Number of Participants With Electrocardiograms (ECGs) Meeting the Pre-Defined Categorical Criteria for 1-Year Follow-Up | Triplicate ECG was performed after the participant had rested quietly for at least 10 minutes in a supine position. The pre-defined categorical criteria include PR interval aggregate (msec): value>=300, baseline>200 and percent change>=25%, baseline<=200 and percent change>=50%; QRS duration aggregate (msec): value>=140, percent change>=50%; QTcF interval aggregate (msec): 450<=value<480, 480<=value<500, value>=500, 30<=change<60, change>=60. Only the category(ies) with at least 1 participant meeting the pre-defined criterion is/are reported for this outcome measure. Baseline for ECG was defined as the measurement before the Day 1 study drug administration. | Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. | Posted | Count of Participants | Participants | Baseline up to 1 year post dose of PF-06939926 |
| |||||||||||||||||||||||||||||||||||||
| Primary | Number of Participants With Positive Responses on Columbia-Suicide Severity Rating Scale (C-SSRS) According to the Columbia Classification Algorithm of Suicide Assessment (C-CASA) Categories | C-SSRS is a participant rated questionnaire to assess whether participant experienced the following: completed suicide(1), suicide attempt(2)("Yes" on "actual attempt"), preparatory acts toward imminent suicidal behavior(3)("Yes" on "preparatory acts or behavior"), suicidal ideation(4)("Yes" on "wish to be dead","non-specific active suicidal thoughts","active suicidal ideation with methods without intent to act or some intent to act, without specific plan or with specific plan and intent), any suicidal behavior or ideation, self-injurious behavior(7)("Yes" on "Has subject engaged in non-suicidal self-injurious behavior"). Yes/No responses are mapped to C-CASA categories. C-SSRS was conducted with the participant's care giver/legal guardian on the participant's behalf throughout the study for participants<=12 years of age at screening. Baseline is defined as the last predose measurement. Number of participants with responses of "Yes" is reported for this outcome measure. | Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. Number of participants analyzed represents the total number of participants in each treatment group in the analysis population for this outcome measure. Number analyzed represents the number of participants who had reportable data for this outcome measure. | Posted | Count of Participants | Participants | Baseline, Day 7, Day 14, Day 180, and Day 360 |
| |||||||||||||||||||||||||||||||||||||
| Primary | Number of Participants With Vital Signs Meeting the Pre-Defined Categorical Criteria for 1-Year Follow-Up | Vital signs consisted of blood pressure, respiratory rate, and heart rate, was obtained with these measurements at the following timepoints: within 30 minutes before and approximately 30 minutes, 1, 2, 4, 8, and 24 hours after the start of the infusion, and on Days 4, 7, 10, 14, 30, 90, 180, and 360. Baseline was defined as the last pre-dose recording. Number of participants who had at least 1 vital sign measurement at the specified timepoints meeting the pre-defined criteria from baseline up to the end of 1-year follow-up is reported for this outcome measure. | Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. Number of participants analyzed represents the total number of participants in each treatment group in the analysis population for this outcome measure. Number analyzed represents the number of participants who had reportable data for this outcome measure. | Posted | Count of Participants | Participants | Baseline up to 1 year post dose of PF-06939926 |
|
Baseline up to the end of 1-year follow-up (approximately 360 days)
The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for SAEs or AEs.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | PF-06939926: Low Dose, Ambulatory Cohort | Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. | 0 | 3 | 0 | 3 | 3 | 3 |
| EG001 | PF-06939926: High Dose, Ambulatory Cohort | Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. | 0 | 16 | 3 | 16 | 16 | 16 |
| EG002 | PF-06939926: High Dose, Non-Ambulatory Cohort | Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. | 1 | 3 | 2 | 3 | 3 | 3 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Haemolytic uraemic syndrome | Blood and lymphatic system disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Cardiogenic shock | Cardiac disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA v25.1 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Haemolysis | Blood and lymphatic system disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Lymphopenia | Blood and lymphatic system disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Cardiomyopathy | Cardiac disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Androgen deficiency | Endocrine disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Lacrimation increased | Eye disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Abdominal pain lower | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Inguinal hernia | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Chest pain | General disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Infusion site reaction | General disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Mass | General disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Liver injury | Hepatobiliary disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Molluscum contagiosum | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Parainfluenzae virus infection | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Tooth abscess | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA v25.1 | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA v25.1 | Non-systematic Assessment |
| |
| Joint dislocation | Injury, poisoning and procedural complications | MedDRA v25.1 | Non-systematic Assessment |
| |
| Joint injury | Injury, poisoning and procedural complications | MedDRA v25.1 | Non-systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA v25.1 | Non-systematic Assessment |
| |
| Post procedural contusion | Injury, poisoning and procedural complications | MedDRA v25.1 | Non-systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA v25.1 | Non-systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA v25.1 | Non-systematic Assessment |
| |
| Amylase increased | Investigations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Complement factor C4 decreased | Investigations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Cystatin C increased | Investigations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Glutamate dehydrogenase increased | Investigations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Lipase increased | Investigations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Troponin increased | Investigations | MedDRA v25.1 | Non-systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Osteopenia | Musculoskeletal and connective tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Abnormal behaviour | Psychiatric disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Affect lability | Psychiatric disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Irritability | Psychiatric disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Mood altered | Psychiatric disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Chronic kidney disease | Renal and urinary disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Glycosuria | Renal and urinary disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Ketonuria | Renal and urinary disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA v25.1 | Non-systematic Assessment |
|
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 27, 2022 | Mar 27, 2023 | SAP_001.pdf |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| 7-<8 years |
|
| 8-<9 years |
|
| 9-<10 years |
|
| 10-<11 years |
|
| 11-<12 years |
|
| >=12 years |
|
| Male |
|
| Black or African American |
|
| Asian |
|
| Not Reported |
|
| Exon Deletion |
|
| Exon Duplication |
|
| Insertion |
|
| Point Mutation |
|
| Cryptic Splice Donor Site |
|
| Title | Measurements |
|---|---|
|
| Severe AEs |
|
| OG002 | PF-06939926: High Dose, Non-Ambulatory Cohort | Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
|
|
Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
| OG002 | PF-06939926: High Dose, Non-Ambulatory Cohort | Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
|
|
| OG002 | PF-06939926: High Dose, Non-Ambulatory Cohort | Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
|
|
| OG002 | PF-06939926: High Dose, Non-Ambulatory Cohort | Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
|
|
Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
| OG002 | PF-06939926: High Dose, Non-Ambulatory Cohort | Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
|
|
Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.
| OG002 | PF-06939926: High Dose, Non-Ambulatory Cohort | Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
|
|
| OG001 | PF-06939926: High Dose, Ambulatory Cohort | Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
| OG002 | PF-06939926: High Dose, Non-Ambulatory Cohort | Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
|
|
| OG002 | PF-06939926: High Dose, Non-Ambulatory Cohort | Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
|
|
| OG002 | PF-06939926: High Dose, Non-Ambulatory Cohort | Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
|
|
| OG002 | PF-06939926: High Dose, Non-Ambulatory Cohort | Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
|
|
Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years.
| OG001 | PF-06939926: High Dose, Ambulatory Cohort | Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
| OG002 | PF-06939926: High Dose, Non-Ambulatory Cohort | Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
|
|
Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
| OG002 | PF-06939926: High Dose, Non-Ambulatory Cohort | Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method. |
|
|