Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A (GENEr8-3)

• ClinicalTrials.gov Identifier: NCT04323098
• Recruitment Status: Active, not recruiting
• First Posted: March 26, 2020
• Results First Posted: April 4, 2024
• Last Update Posted: April 4, 2024
• Sponsor: BioMarin Pharmaceutical
• Information provided by (Responsible Party): BioMarin Pharmaceutical

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Hemophilia A
• Intervention: Biological: valoctocogene roxaparvovec
• Enrollment: 22

Participant Flow

Recruitment Details	This study was conducted by 12 principal investigators at 12 study centers in 4 countries (United States, Australia, Brazil, and Taiwan)
Pre-assignment Details	Total of 34 subjects were screened. Of these, 12 subjects failed screening and remaining 22 subject was enrolled in the study. Of the 22 enrolled participants, all 22 completed the Week 52 visit, and all 22 remained enrolled in the study as on data cut-off date 27January2023. While up to 25% of patients could be AAV5+, no such patients were enrolled, i.e., everyone was AAV5 negative.

Arm/Group Title	BMN 270 (Valoctocogene Roxaparvovec)
Arm/Group Description	Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII, with Prophylactic Corticosteroids in Hemophilia A subjects
Period Title: Overall Study
Started	22
Continuing in the Study	22
Completed Week 52 Visit	22
Completed [1]	0
Not Completed	22
[1] Study is still ongoing.

Baseline Characteristics

Arm/Group Title		BMN 270 (Valoctocogene Roxaparvovec)
Arm/Group Description		Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII, with Prophylactic Corticosteroids in Hemophilia A subjects
Overall Number of Baseline Participants		22
Baseline Analysis Population Description		Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
Age, Continuous [1]; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	22 participants
		28.0 (7.4)
		[1] Measure Description: Age at enrollment, years
Age, Customized [1]; Measure Type: Count of Participants; Unit of measure: Participants	Number Analyzed	22 participants
18 to < 30 years		13 59.1%
30 to < 50 years		9 40.9%
>= 50 years		0 0.0%
		[1] Measure Description: Age at enrollment: n(%)
Sex: Female, Male [1]; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	22 participants
	Female	0 0.0%
	Male	22 100.0%
		[1] Measure Description: Percentages were calculated using the total number of participants (N) in each analysis population as the denominator.
Race/Ethnicity, Customized [1]; Measure Type: Count of Participants; Unit of measure: Participants	Number Analyzed	22 participants
Asian		2 9.1%
Black or African-American		2 9.1%
White		18 81.8%
		[1] Measure Description: n(%)
Baseline annualized FVIII usage [1]; Mean (Standard Deviation); Unit of measure: IU/kg/year
	Number Analyzed	22 participants
		4342.74 (3184.01)
		[1] Measure Description: The annualized utilization (IU/kg/year) of exogenous FVIII replacement therapy is defined as Sum of FVIII use (IU/kg) during calculation period/Total number of days during the calculation period ×365.25.
Baseline annualized number of FVIII infusions [1]; Mean (Standard Deviation); Unit of measure: Infusions/year
	Number Analyzed	22 participants
		149.13 (72.74)
		[1] Measure Description: Annualized FVIII infusion rate (count/yr) = (sum(Number of FVIII replacement infusions during calculation period) /sum(follow-up days of the period))*365.25.
Baseline ABR (treated bleeds) [1]; Mean (Standard Deviation); Unit of measure: Bleeds/year
	Number Analyzed	22 participants
		5.61 (11.29)
		[1] Measure Description: Bleeds that were treated with FVIII replacement therapy (recorded as "treatment for bleed") within 72 hours and were not associated with surgery or a procedure were included. Annualized bleed rate (episodes/yr) = (sum (number of bleed episodes during calculation period))/(sum (follow-up days of the period)) *365.25
Baseline ABR (treated bleeds) [1]; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	22 participants
	0 bleeds/year	5 22.7%
	>0 to 4 bleeds/year	10 45.5%
	>4 to 10 bleeds/year	5 22.7%
	>10 bleeds/year	2 9.1%
		[1] Measure Description: Bleeds that were treated with FVIII replacement therapy (recorded as "treatment for bleed") within 72 hours and were not associated with surgery or a procedure were included. Annualized bleed rate (episodes/yr) = (sum (number of bleed episodes during calculation period))/(sum (follow-up days of the period)) *365.25
Baseline ABR (all bleeds) [1]; Mean (Standard Deviation); Unit of measure: Bleeds/year
	Number Analyzed	22 participants
		8.20 (17.86)
		[1] Measure Description: All bleeds comprises both treated and non-treated bleeds. In this definition, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. All bleeds are any reported bleeding events regardless of the use of FVIII or other treatments. Annualized bleed rate (episodes/yr) = (sum (number of bleed episodes during calculation period))/(sum (follow-up days of the period)) *365.25
Baseline ABR (all bleeds) [1]; Measure Type: Count of Participants; Unit of measure: Participants	Number Analyzed	22 participants
0 bleeds/year		5 22.7%
>0 to 4		10 45.5%
>4 to 10		4 18.2%
>10		3 13.6%
		[1] Measure Description: All bleeds comprises both treated and non-treated bleeds. In this definition, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. All bleeds are any reported bleeding events regardless of the use of FVIII or other treatments. Annualized bleed rate (episodes/yr) = (sum (number of bleed episodes during calculation period))/(sum (follow-up days of the period)) *365.25
History of previous diseases [1]; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	22 participants
	Hepatitis B	0 0.0%
	Hepatitis C	3 13.6%
	HIV	0 0.0%
	No History of exposure to Hepatitis B/Hepatitis C/HIV	19 86.4%
		[1] Measure Analysis Population Description: n(%)
Number of target joints [1]; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	22 participants
	0	16 72.7%
	1	3 13.6%
	2	2 9.1%
	3	1 4.5%
		[1] Measure Description: n(%) Target joints are defined as joints with >= 3 spontaneous treated bleeds in the same joint location within any consecutive 6-month (180 days) period in the Baseline period.
Adeno-associated virus Type 5 (AAV5) Total Antibody Titers and Positivity [1]; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	22 participants
	Detected	0 0.0%
	Not detected	22 100.0%
		[1] Measure Description: Percentages were calculated using the number of subjects with non-missing assessments (n) for each analysis population as the denominator.

Outcome Measures

1. Primary Outcome

Title	Change From Baseline in FVIII Activity as Measured by Chromogenic Substrate Assay at Week 52.
Description	The change from baseline (assuming no treatment for severe hemophilia A) in FVIII activity, as measured by chromogenic substrate assay (CSA), at Week 52 (during Weeks 49 - 52) post-BMN 270 infusion. Each participant's FVIII activity level at Week 52 is defined as the median of the values obtained within the analysis window at Weeks 49-52. The baseline value will be imputed as 1 IU/dL, since there will be no washout of severe hemophilia A participants' usual FVIII prophylaxis (in order to avoid increasing the risk of bleeding) prior to BMN 270 infusion. Post-BMN 270 infusion values for FVIII activity will be excluded from analysis if obtained within 72 hours (or 3 calendar days if time is not available) since the last infusion of exogenous FVIII replacement therapy. Baseline: prior to BMN 270 infusion while receiving FVIII prophylaxis.
Time Frame	Baseline to Week 52

Outcome Measure Data

Analysis Population Description

Modified intention-to-treat (mITT) population (N=21) - all participants who received BMN 270 infusion in 270-303 and were on adequate prophylactic hemophilia therapy for at least 12 months (adequate prophylaxis was defined as >= 52 doses of FVIII replacement therapy in 12 months if on FVIII prophylaxis) prior to BMN 270 infusion.

Arm/Group Title	BMN 270 (Valoctocogene Roxaparvovec)
Arm/Group Description:	Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A subjects
Overall Number of Participants Analyzed	21
Mean (Standard Deviation); Unit of Measure: IU /dL
	15.13 (22.38)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	BMN 270 (Valoctocogene Roxaparvovec)
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.0057
	Comments	P-value was based on two-sided t-test against 0.
	Method	t-test, 2 sided
	Comments	[Not Specified]

2. Secondary Outcome

Title	Change From Baseline in Annualized Utilization of Exogenous FVIII Replacement Therapy in EEP
Description	The change from baseline (prior to BMN 270 infusion while receiving FVIII prophylaxis) in the annualized utilization (IU/kg/year) of exogenous FVIII replacement therapy in the efficacy evaluation period ("Post-FVIII Prophylaxis period"). The annualized utilization (IU/kg/year) of exogenous FVIII replacement therapy is defined as Sum of FVIII use (IU/kg) during calculation period/Total number of days during the calculation period ×365.25. Baseline: prior to BMN 270 infusion while receiving FVIII prophylaxis EEP: From Week 5 post-BMN 270 infusion (Study Day 33) or the end of FVIII prophylaxis plus the washout period (3 days for products of standard half-life or plasma-derived and 5 days for products of extended half-life), whichever is later, to last visit by the data cut-off for the analysis, hereafter referred to as "Post FVIII Prophylaxis to Last Visit").
Time Frame	Baseline to efficacy evaluation period (EEP)

Outcome Measure Data

Analysis Population Description

mITT population

Arm/Group Title	BMN 270 (Valoctocogene Roxaparvovec)
Arm/Group Description:	Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A subjects
Overall Number of Participants Analyzed	21
Mean (Standard Deviation); Unit of Measure: IU/kg/yr
	-4150.09 (3208.50)

3. Secondary Outcome

Title	Change From Baseline in the Annualized Number of Bleeding Episodes Irrespective of Exogenous FVIII Replacement Treatment (Annualized Bleeding Rate, ABR for All Bleeds) in EEP
Description	All bleeds comprises both treated and non-treated bleeds. In this definition, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. All bleeds are any reported bleeding events regardless of the use of FVIII or other treatments. ABR for all bleeds= Number of bleeding episodes for all bleeds during the calculation period / total number of days during the calculation period * 365.25. Baseline: prior to BMN 270 infusion while receiving FVIII prophylaxis. EEP: From Week 5 post-BMN 270 infusion (Study Day 33) or the end of FVIII prophylaxis plus the washout period (3 days for products of standard half-life or plasma-derived and 5 days for products of extended half-life), whichever is later, to last visit by the data cut-off for analysis, hereafter referred to as "Post FVIII Prophylaxis to Last Visit").
Time Frame	Baseline to efficacy evaluation period (EEP)

Outcome Measure Data

Analysis Population Description

mITT population

Arm/Group Title	BMN 270 (Valoctocogene Roxaparvovec)
Arm/Group Description:	Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A subjects
Overall Number of Participants Analyzed	21
Mean (Standard Deviation); Unit of Measure: bleeds/year
	-6.15 (17.24)

4. Secondary Outcome

Title	Change From Baseline in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Treatment (ABR for Treated Bleeds) in the EEP.
Description	ABR for treated bleeds=Number of bleeding episodes for treated bleeds during the calculation period/total number of days during the calculation period * 365.25 Bleeds that were treated with FVIII replacement therapy (recorded as "treatment for bleed") within 72 hours and were not associated with surgery or a procedure were included. Baseline: prior to BMN 270 infusion while receiving FVIII prophylaxis. EEP: From Week 5 post-BMN 270 infusion (Study Day 33) or the end of FVIII prophylaxis plus the washout period (3 days for products of standard half-life or plasma-derived and 5 days for products of extended half-life), whichever is later, to last visit by the data cut-off for analysis, hereafter referred to as "Post FVIII Prophylaxis to Last Visit").
Time Frame	Baseline to EEP

Outcome Measure Data

Analysis Population Description

mITT population

Arm/Group Title	BMN 270 (Valoctocogene Roxaparvovec)
Arm/Group Description:	Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A subjects
Overall Number of Participants Analyzed	21
Mean (Standard Deviation); Unit of Measure: bleeds/year
	-3.78 (10.14)

5. Secondary Outcome

Title	Change From Baseline in Haemo-QoL-A Quality of Life: Total Score at Week 52
Description	The change from baseline(assuming no treatment for severe hemophilia A) in Haemo-Qol-A score, at wk52 post-BMN 270 infusion.The Haemo-Qol-A questionnaire is a fit for purpose hemophilia-specific health related quality of life(HRQoL)questionnaire for adults consisting of 41 items covering 6 domains(Physical Functioning, Role Functioning,Worry,Consequences of Bleeding,Emotional Impact &Treatment Concerns). The Haemo-Qol-A items are answered on a 6-point Likert scale ranging from 0(none of the time)to 5 (all of the time).The recall period for the Haemo-Qol-A is one month (4-weeks). The Haemo-QoL-A domain(physical functioning, role functioning, worry, consequences of bleeding, emotional impact, treatment concern) scores range from 0 to 5 and the total score is derived by summing each domain score (range, 0 to 30). Domain and total scores are transformed to a 0 (minimum) to 100 (maximum) scale with higher scores indicating a better or less impaired haemophilia related quality of life.
Time Frame	Baseline to Week 52

Outcome Measure Data

Analysis Population Description

mITT population

Arm/Group Title	BMN 270 (Valoctocogene Roxaparvovec)
Arm/Group Description:	Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A subjects
Overall Number of Participants Analyzed	19
Mean (Standard Deviation); Unit of Measure: score on a scale
	6.70 (8.96)

6. Secondary Outcome

Title	Change From Baseline in Haemo-QoL-A Quality of Life: Physical Functioning Domain Score, at Week 52
Description	The change from baseline (assuming no treatment for severe hemophilia A) in Haemo-Qol-A score, at week 52 post BMN 270 infusion. The Haemo-Qol-A questionnaire is a fit for purpose hemophilia-specific health related quality of life (HRQoL) questionnaire for adults consisting of 41 items covering six domains (Physical Functioning, Role Functioning, Worry, Consequences of Bleeding, Emotional Impact and Treatment Concerns).The Haemo-Qol-A items are answered on a 6-point Likert scale ranging from 0 (none of the time) to 5 (all of the time).The recall period for the Haemo-Qol-A is one month (4-weeks). The Haemo-Qol-A physical functioning domain score is an average of each item value within a domain.The range of domain scores is 0 to 5; higher scores mean better HRQoL or less impairment for the domain. The physical functioning domain score is transformed to a 0 (minimum) to 100 (maximum) scale with higher scores indicating a better or less impaired haemophilia-related physical functioning
Time Frame	Baseline to Week 52

Outcome Measure Data

Analysis Population Description

mITT population

Arm/Group Title	BMN 270 (Valoctocogene Roxaparvovec)
Arm/Group Description:	Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A subjects
Overall Number of Participants Analyzed	19
Mean (Standard Deviation); Unit of Measure: score on a scale
	6.43 (8.51)

7. Secondary Outcome

Title	Change From Baseline in Haemo-QoL-A Quality of Life: Consequences of Bleeding Domain Score, at Week 52
Description	The change from baseline(assuming no treatment for severe hemophilia A)in Haemo-Qol-A score, at week 52 post-BMN 270 infusion. The Haemo-Qol-A questionnaire is a fit for purpose hemophilia-specific health related quality of life(HRQoL)questionnaire for adults consisting of 41 items covering 6 domains(Physical Functioning,Role Functioning,Worry,Consequences of Bleeding,Emotional Impact and Treatment Concerns). The Haemo-Qol-A items are answered on a 6-point Likert scale ranging from 0(none of the time) to 5(all of the time). The recall period for the Haemo-Qol-A is one month (4-wks). The Haemo-Qol-A consequences of bleeding domain score is an average of each item value within a domain. The range of domain scores is 0 to 5; higher scores mean better HRQoL or less impairment for the domain. The consequences of bleeding domain score is transformed to a 0 (minimum) to 100 (maximum) scale with higher scores indicating a better or less impaired haemophilia-related consequences of bleeding.
Time Frame	Baseline to Week 52

Outcome Measure Data

Analysis Population Description

mITT population

Arm/Group Title	BMN 270 (Valoctocogene Roxaparvovec)
Arm/Group Description:	Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A subjects
Overall Number of Participants Analyzed	19
Mean (Standard Deviation); Unit of Measure: score on a scale
	10.68 (16.33)

8. Secondary Outcome

Title	Change From Baseline in Haemo-QoL-A Quality of Life: Role Functioning Domain Score, at Week 52
Description	The change from baseline (assuming no treatment for severe hemophilia A) in Haemo-Qol-A score, at week 52 post BMN 270 infusion. The Haemo-Qol-A questionnaire is a fit for purpose hemophilia-specific health related quality of life (HRQoL) questionnaire for adults consisting of 41 items covering six domains (Physical Functioning, Role Functioning, Worry, Consequences of Bleeding, Emotional Impact and Treatment Concerns).The Haemo-Qol-A items are answered on a 6-point Likert scale ranging from 0 (none of the time) to 5 (all of the time). The recall period for the Haemo-Qol-A is one month (4-weeks). The Haemo-Qol-A role functioning domain score is an average of each item value within a domain. The range of domain scores is 0 to 5; higher scores mean better HRQoL or less impairment for the domain. The role functioning domain score is transformed to a 0 (minimum) to 100 (maximum) scale with higher scores indicating a better or less impaired haemophilia-related role functioning.
Time Frame	Baseline to Week 52

Outcome Measure Data

Analysis Population Description

mITT population

Arm/Group Title	BMN 270 (Valoctocogene Roxaparvovec)
Arm/Group Description:	Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A subjects
Overall Number of Participants Analyzed	19
Mean (Standard Deviation); Unit of Measure: Score on a scale
	6.03 (10.29)

Adverse Events

Time Frame	Up to 112 weeks
Adverse Event Reporting Description	Intention-to-treat (ITT) population (N=22) - all participants who received BMN 270 infusion in 270-303 by the time of the data cutoff
Arm/Group Title	BMN 270 (Valoctocogene Roxaparvovec)
Arm/Group Description	Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids valoctocogene roxaparvovec: Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A
All-Cause Mortality
	BMN 270 (Valoctocogene Roxaparvovec)
	Affected / at Risk (%)
Total	0/22 (0.00%)
Serious Adverse Events
	BMN 270 (Valoctocogene Roxaparvovec)
	Affected / at Risk (%)	# Events
Total	1/22 (4.55%)
Injury, poisoning and procedural complications
Head injury † 1	1/22 (4.55%)	1
1 Term from vocabulary, MedDRA 24.0; † Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	BMN 270 (Valoctocogene Roxaparvovec)
	Affected / at Risk (%)	# Events
Total	22/22 (100.00%)
Blood and lymphatic system disorders
Anaemia † 1	2/22 (9.09%)	3
Iron deficiency anaemia † 1	2/22 (9.09%)	2
Gastrointestinal disorders
Diarrhoea † 1	4/22 (18.18%)	4
Dyspepsia † 1	3/22 (13.64%)	3
Gastrooesophageal reflux disease † 1	3/22 (13.64%)	3
Nausea † 1	3/22 (13.64%)	6
Vomiting † 1	4/22 (18.18%)	9
General disorders
Fatigue † 1	3/22 (13.64%)	3
Influenza like illness † 1	3/22 (13.64%)	4
Pyrexia † 1	2/22 (9.09%)	2
Infections and infestations
COVID-19 † 1	12/22 (54.55%)	12
Upper respiratory tract infection † 1	5/22 (22.73%)	8
Influenza † 1	4/22 (18.18%)	4
Rash pustular † 1	2/22 (9.09%)	2
Sinusitis † 1	2/22 (9.09%)	2
Injury, poisoning and procedural complications
Infusion related reaction † 1	3/22 (13.64%)	3
Road traffic accident † 1	2/22 (9.09%)	2
Investigations
Alanine aminotransferase increased † 1	20/22 (90.91%)	50
Aspartate aminotransferase increased † 1	4/22 (18.18%)	9
Gamma-glutamyltransferase increased † 1	2/22 (9.09%)	3
Musculoskeletal and connective tissue disorders
Arthralgia † 1	4/22 (18.18%)	4
Myalgia † 1	4/22 (18.18%)	4
Arthropathy † 1	3/22 (13.64%)	3
Muscle spasms † 1	3/22 (13.64%)	3
Back pain † 1	2/22 (9.09%)	2
Nervous system disorders
Headache † 1	7/22 (31.82%)	11
Tremor † 1	2/22 (9.09%)	2
Psychiatric disorders
Insomnia † 1	5/22 (22.73%)	6
Agitation † 1	2/22 (9.09%)	2
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain † 1	4/22 (18.18%)	4
Rhinorrhoea † 1	3/22 (13.64%)	3
Skin and subcutaneous tissue disorders
Acne † 1	6/22 (27.27%)	7
Dermatitis acneiform † 1	3/22 (13.64%)	4
Rash maculo-papular † 1	3/22 (13.64%)	3
Hyperhidrosis † 1	2/22 (9.09%)	2
Social circumstances
Pregnancy of partner † 1	2/22 (9.09%)	2
1 Term from vocabulary, MedDRA 24.0; † Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

• Name/Title: Tara M Robinson, MD, PhD, Senior Medical Director, Late-Stage Clinical Development
• Organization: BioMarin Pharmaceutical Inc.
• Phone: 415-455-7931
• EMail: tara.robinson@bmrn.com

• Responsible Party: BioMarin Pharmaceutical
• ClinicalTrials.gov Identifier: NCT04323098
• Other Study ID Numbers: 270-303
• First Submitted: March 24, 2020
• First Posted: March 26, 2020
• Results First Submitted: January 24, 2024
• Results First Posted: April 4, 2024
• Last Update Posted: April 4, 2024