Trial Outcomes & Findings for Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD (NCT NCT04632940)

Last Updated: 2024-08-26

Results Overview

The NSAA consisted of 17 activities, each scored as 0 (activity could not be performed), 1 (modified method but achieved goal without physical assistance from another), or 2 (normal, achieved goal without assistance). The sum of these 17 scores was used to form a total score ranging from 0 (worst) to 34 (fully independent function). If fewer than 15 of the 17 activities were performed, the total score was considered missing. If 15 to 16 activities were performed, the total score was calculated by multiplying the sum of the scores in the x activities that were performed by 17/x. If an activity could not be performed due to disease progression/loss of ambulation, a score of 0 was assigned. Higher scores indicated better functioning. Least square (LS) mean and standard error (SE) were analyzed using a mixed model for repeated measure (MMRM).

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

73 participants

Primary outcome timeframe

Baseline, Week 52

Results posted on

2024-08-26

Participant Flow

The study included 2 periods: a Double-blind (DB) period and an Open-label extension (OLE) period.

Participant milestones

Participant milestones
Measure	Pamrevlumab Participants received pamrevlumab 35 milligrams (mg)/kilogram (kg) by intravenous (IV) infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period and continued to receive the same dose of pamrevlumab for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.	Placebo Participants received placebo matched to pamrevlumab by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period. Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.
DB Period (52 Weeks) STARTED	37	36
DB Period (52 Weeks) Received at Least 1 Dose of Study Drug	36	36
DB Period (52 Weeks) COMPLETED	35	36
DB Period (52 Weeks) NOT COMPLETED	2	0
OLE Period (52 Weeks) STARTED	34	35
OLE Period (52 Weeks) Received at Least 1 Dose of Study Drug	34	34
OLE Period (52 Weeks) COMPLETED	0	0
OLE Period (52 Weeks) NOT COMPLETED	34	35

Reasons for withdrawal

Reasons for withdrawal
Measure	Pamrevlumab Participants received pamrevlumab 35 milligrams (mg)/kilogram (kg) by intravenous (IV) infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period and continued to receive the same dose of pamrevlumab for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.	Placebo Participants received placebo matched to pamrevlumab by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period. Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.
DB Period (52 Weeks) Participant/Legal Guardian Decision	2	0
OLE Period (52 Weeks) Adverse Event	1	0
OLE Period (52 Weeks) Lost to Follow-up	0	1
OLE Period (52 Weeks) Physician Decision	1	0
OLE Period (52 Weeks) Participant/Legal Guardian Decision	11	13
OLE Period (52 Weeks) Sponsor Decision to Terminate Study	21	19
OLE Period (52 Weeks) Other than specified	0	1
OLE Period (52 Weeks) Entered into OLE but not treated	0	1

Baseline Characteristics

Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Pamrevlumab n=37 Participants Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period and continued to receive the same dose of pamrevlumab for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.	Placebo n=36 Participants Participants received placebo matched to pamrevlumab by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period. Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.	Total n=73 Participants Total of all reporting groups
Age, Continuous	9.1 years STANDARD_DEVIATION 1.46 • n=5 Participants	9.0 years STANDARD_DEVIATION 1.56 • n=7 Participants	9.0 years STANDARD_DEVIATION 1.50 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Male	37 Participants n=5 Participants	36 Participants n=7 Participants	73 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	5 Participants n=7 Participants	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	34 Participants n=5 Participants	28 Participants n=7 Participants	62 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	10 Participants n=5 Participants	9 Participants n=7 Participants	19 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) White	23 Participants n=5 Participants	22 Participants n=7 Participants	45 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants	4 Participants n=7 Participants	7 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Week 52

Population: The ITT set included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Pamrevlumab n=36 Participants Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period and continued to receive the same dose of pamrevlumab for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.	Placebo n=36 Participants Participants received placebo matched to pamrevlumab by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period. Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.
Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52	-3.022 units on a scale Standard Error 0.5505	-2.494 units on a scale Standard Error 0.6962

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: The ITT set included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

The 4SCV (centimeters \[cm\]/second \[sec\]) was calculated as the ratio of the total height (cm) of stairs climbed divided by the number of seconds taken to complete the 4-stair climb.

Outcome measures

Outcome measures
Measure	Pamrevlumab n=36 Participants Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period and continued to receive the same dose of pamrevlumab for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.	Placebo n=36 Participants Participants received placebo matched to pamrevlumab by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period. Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.
Change From Baseline in 4-Stair Climb Velocity (4SCV) Assessment at Week 52	-1.858 cm/sec Standard Deviation 4.5459	-3.797 cm/sec Standard Deviation 5.1899

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: The ITT set included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

The time (in sec) required for a participant to run or walk a distance of 10 meters as quickly as possible was calculated as velocity (meters/sec).

Outcome measures

Outcome measures
Measure	Pamrevlumab n=36 Participants Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period and continued to receive the same dose of pamrevlumab for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.	Placebo n=36 Participants Participants received placebo matched to pamrevlumab by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period. Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.
Change From Baseline in the 10-Meter Walk/Run Test at Week 52	-0.176 meters/sec Standard Deviation 0.2193	-0.196 meters/sec Standard Deviation 0.3552

SECONDARY outcome

Timeframe: Baseline, Week 52

Population: The ITT set included all randomized participants. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

The time (in sec) required for a participant to stand from supine position has been reported. A longer time taken reflected a worse outcome.

Outcome measures

Outcome measures
Measure	Pamrevlumab n=36 Participants Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period and continued to receive the same dose of pamrevlumab for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.	Placebo n=31 Participants Participants received placebo matched to pamrevlumab by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period. Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.
Change From Baseline in Time to Stand (TTSTAND) at Week 52	2.24 sec Standard Deviation 3.353	1.94 sec Standard Deviation 3.688

SECONDARY outcome

Timeframe: Baseline to Week 52

Population: The ITT set included all randomized participants.

Time (days) to LoA was defined as the number of days from randomization to the date of LoA, or all-cause death based on observed data, whichever occurred earlier during the on-study period. Median time (days) to LoA was calculated using Kaplan Meier Survival Estimates.

Outcome measures

Outcome measures
Measure	Pamrevlumab n=37 Participants Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period and continued to receive the same dose of pamrevlumab for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.	Placebo n=36 Participants Participants received placebo matched to pamrevlumab by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period. Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.
Time to Loss of Ambulation (LoA) From Baseline to Week 52	NA days Due to insufficient number of participants with an event of LoA or death, median and 95% confidence interval (CI) could not be calculated.	NA days Due to insufficient number of participants with an event of LoA or death, median and 95% CI could not be calculated.

Adverse Events

DB Period: Pamrevlumab

Serious events: 3 serious events

Other events: 35 other events

Deaths: 0 deaths

DB Period: Placebo

Serious events: 1 serious events

Other events: 33 other events

Deaths: 0 deaths

OLE Period: Pamrevlumab

Serious events: 0 serious events

Other events: 45 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	DB Period: Pamrevlumab n=36 participants at risk Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period.	DB Period: Placebo n=36 participants at risk Participants received placebo matched to pamrevlumab by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the DB period.	OLE Period: Pamrevlumab n=68 participants at risk Participants received pamrevlumab 35 mg/kg by IV infusion every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks in the OLE period or until pamrevlumab was commercially available, or the sponsor decided to end the study, whichever occurred first.
Cardiac disorders Myocarditis	2.8% 1/36 • Baseline up to Week 129 As pre-specified, All-cause mortality data were collected and reported for all enrolled participants; and Serious and Non-serious adverse events data were collected and reported for all participants who received any dose of study medication.	0.00% 0/36 • Baseline up to Week 129 As pre-specified, All-cause mortality data were collected and reported for all enrolled participants; and Serious and Non-serious adverse events data were collected and reported for all participants who received any dose of study medication.	0.00% 0/68 • Baseline up to Week 129 As pre-specified, All-cause mortality data were collected and reported for all enrolled participants; and Serious and Non-serious adverse events data were collected and reported for all participants who received any dose of study medication.
Infections and infestations Upper respiratory tract infection	2.8% 1/36 • Baseline up to Week 129 As pre-specified, All-cause mortality data were collected and reported for all enrolled participants; and Serious and Non-serious adverse events data were collected and reported for all participants who received any dose of study medication.	0.00% 0/36 • Baseline up to Week 129 As pre-specified, All-cause mortality data were collected and reported for all enrolled participants; and Serious and Non-serious adverse events data were collected and reported for all participants who received any dose of study medication.	0.00% 0/68 • Baseline up to Week 129 As pre-specified, All-cause mortality data were collected and reported for all enrolled participants; and Serious and Non-serious adverse events data were collected and reported for all participants who received any dose of study medication.
Musculoskeletal and connective tissue disorders Knee deformity	2.8% 1/36 • Baseline up to Week 129 As pre-specified, All-cause mortality data were collected and reported for all enrolled participants; and Serious and Non-serious adverse events data were collected and reported for all participants who received any dose of study medication.	0.00% 0/36 • Baseline up to Week 129 As pre-specified, All-cause mortality data were collected and reported for all enrolled participants; and Serious and Non-serious adverse events data were collected and reported for all participants who received any dose of study medication.	0.00% 0/68 • Baseline up to Week 129 As pre-specified, All-cause mortality data were collected and reported for all enrolled participants; and Serious and Non-serious adverse events data were collected and reported for all participants who received any dose of study medication.
Musculoskeletal and connective tissue disorders Rhabdomyolysis	0.00% 0/36 • Baseline up to Week 129 As pre-specified, All-cause mortality data were collected and reported for all enrolled participants; and Serious and Non-serious adverse events data were collected and reported for all participants who received any dose of study medication.	2.8% 1/36 • Baseline up to Week 129 As pre-specified, All-cause mortality data were collected and reported for all enrolled participants; and Serious and Non-serious adverse events data were collected and reported for all participants who received any dose of study medication.	0.00% 0/68 • Baseline up to Week 129 As pre-specified, All-cause mortality data were collected and reported for all enrolled participants; and Serious and Non-serious adverse events data were collected and reported for all participants who received any dose of study medication.

Other adverse events

Additional Information

Clinical Trial Information Desk

FibroGen, Inc.

Phone: 415-978-1441

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The multisite consortium can publish any time after the data is collected and analyzed by FibroGen. The investigator can only publish after the multisite consortium publishes (or tries to publish and fails). FibroGen has 60 days to review a publication and can extend the embargo up to an additional 120 days (or 180 total).
Publication restrictions are in place

Restriction type: OTHER