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Corticosteroid
Pamrevlumab + Corticosteroids for Duchenne Muscular Dystrophy (LELANTOS-2 Trial)
Phase 3
Waitlist Available
Research Sponsored by FibroGen
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Medical history includes diagnosis of DMD and confirmed Duchenne mutation, including status of exon 44 using a validated genetic test
Able to complete 6-minute walking distance (6MWD) test with a distance of at least 270 meters but no more than 450 meters on two occasions within 3 months prior to randomization with ≤10% variation between these two tests
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, week 52
Awards & highlights
LELANTOS-2 Trial Summary
This trial is testing a new drug, pamrevlumab, to see if it is effective and safe to use in combination with systemic corticosteroids in young boys with Duchenne muscular dystrophy.
Who is the study for?
Boys aged 6 to under 12 with Duchenne muscular dystrophy (DMD) can join this trial. They must have a specific genetic mutation, be able to walk and rise from the floor quickly, and have been on stable corticosteroid treatment for at least 6 months. Those with severe allergies to certain antibodies, kidney issues, or other conditions that could interfere with the study cannot participate.Check my eligibility
What is being tested?
The trial is testing pamrevlumab in combination with systemic corticosteroids against a placebo plus steroids in boys with DMD. The goal is to see if pamrevlumab helps improve lung function and muscle strength when given every two weeks.See study design
What are the potential side effects?
Pamrevlumab may cause reactions related to infusion, potential allergic responses due to its antibody nature, and possibly affect organ functions like liver or kidneys based on previous similar treatments.
LELANTOS-2 Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have been diagnosed with DMD and have a confirmed Duchenne mutation.
Select...
I can walk between 270 and 450 meters in 6 minutes, and I did this consistently in two tests.
Select...
I can stand up from the floor in less than 10 seconds without help.
Select...
I can have an MRI scan of my leg muscles.
Select...
My kidney function is good, with cystatin C levels at or below 1.4 mg/L.
Select...
My blood counts and electrolyte levels are within the normal range.
Select...
My liver functions well, with normal GGT and bilirubin levels.
Select...
I am a boy between 6 and 11 years old.
LELANTOS-2 Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline, week 52
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, week 52
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52
Secondary outcome measures
Change From Baseline in 4-Stair Climb Velocity (4SCV) Assessment at Week 52
Change From Baseline in Time to Stand (TTSTAND) at Week 52
Change From Baseline in the 10-Meter Walk/Run Test at Week 52
+1 moreSide effects data
From 2017 Phase 2 trial • 160 Patients • NCT0189026530%
Respiratory tract infection
28%
Cough
26%
Dyspnoea
20%
Fatigue
20%
Idiopathic pulmonary fibrosis
20%
Urinary tract infection
18%
Nasopharyngitis
16%
Diarrhoea
16%
Sinusitis
14%
Nausea
10%
Arthralgia
10%
Back pain
8%
Headache
8%
Pain
8%
Upper-airway cough syndrome
8%
Abdominal pain upper
8%
Chest pain
8%
Oedema peripheral
8%
Dizziness
8%
Insomnia
6%
Sleep apnoea syndrome
6%
Myalgia
6%
Chest discomfort
6%
Heart sounds abnormal
6%
Decreased appetite
6%
Musculoskeletal pain
6%
Anxiety
6%
Pulmonary hypertension
6%
Sinus congestion
6%
Flushing
6%
Hypertension
4%
Bronchitis
4%
Constipation
4%
Interstitial lung disease
4%
Pulmonary embolism
4%
Contusion
2%
Immune thrombocytopenic purpura
2%
Autoimmune haemolytic anaemia
2%
Non-cardiac chest pain
2%
Acute respiratory failure
2%
Respiratory failure
2%
Angina pectoris
2%
Sepsis
2%
Femoral neck fracture
2%
Humerus fracture
2%
Musculoskeletal chest pain
2%
Squamous cell carcinoma of the tongue
2%
Peripheral ischaemia
2%
Throat irritation
100%
80%
60%
40%
20%
0%
Study treatment Arm
Pamrevlumab
Placebo
Sub-Study: Pamrevlumab+Pirfenidone
Sub-Study: Placebo+Pirfenidone
Sub-Study: Pamrevlumab+Nintedanib
Sub-Study: Placebo+Nintedanib
LELANTOS-2 Trial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: PamrevlumabExperimental Treatment2 Interventions
Pamrevlumab 35 milligrams (mg)/kilogram (kg) intravenously (IV) every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks
Group II: PlaceboPlacebo Group2 Interventions
Matching placebo IV every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Pamrevlumab
2013
Completed Phase 3
~210
Corticosteroids
2003
Completed Phase 4
~8270
Find a Location
Who is running the clinical trial?
FibroGenLead Sponsor
57 Previous Clinical Trials
15,234 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have any other important heart problem.Your lung function, as measured by forced vital capacity, is above 45% of what is expected for someone your age and size.I have asthma or lung disease that is not well-managed.I have been diagnosed with DMD and have a confirmed Duchenne mutation.I've been on a steady dose of corticosteroids for at least 6 months.I can walk between 270 and 450 meters in 6 minutes, and I did this consistently in two tests.I can stand up from the floor in less than 10 seconds without help.I can have an MRI scan of my leg muscles.I agree to get yearly flu shots during the study.My kidney function is good, with cystatin C levels at or below 1.4 mg/L.I haven't taken any experimental drugs or certain approved treatments recently, except for systemic corticosteroids.My blood counts and electrolyte levels are within the normal range.I was hospitalized for breathing failure within the last 8 weeks.My liver functions well, with normal GGT and bilirubin levels.I am a boy between 6 and 11 years old.You need to use a ventilator for at least 16 hours straight.My kidney function is low or I have signs of kidney injury.Your body mass index (BMI) is 40 or higher, or you weigh more than 117 kilograms.You have had a severe allergic reaction to certain types of medications called monoclonal antibodies.I am able to fully participate and complete the study.I have a health condition besides DMD that affects my muscle strength or movement.I have been treated with pamrevlumab before.I do not have severe heart failure or serious kidney problems.I am on medication for an irregular heartbeat.
Research Study Groups:
This trial has the following groups:- Group 1: Pamrevlumab
- Group 2: Placebo
Awards:
This trial has 1 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Could you detail the possible side effects of Pamrevlumab?
"Pamrevlumab has received a score of 3 for safety. This is due to Phase 3 trials having data supporting both efficacy and multiple rounds of safety testing."
Answered by AI
Does this experiment include elderly participants?
"The age bracket that is eligible for participation in this clinical trial is 6 to 11 years old."
Answered by AI
Could you please provide a list of all clinical trials that have utilized Pamrevlumab?
"The first clinical trials for pamrevlumab were completed in 2016 at The Children's Hospital of Philadelphia. Out of the 18283 total completed studies, there are only 6 that are actively recruiting patients. A Hershey, Pennsylvania medical center is hosting many of these active trials."
Answered by AI
To what type of patient population is this clinical trial open?
"Currently, this trial is enrolling 70 children aged 6-11 that have muscular dystrophy. The most crucial requirements for candidates are as follows: Written consent from a legal guardian, being on a stable dose of systemic corticosteroids for at least 6 months with no substantial changes in the last 3 months, receiving annual influenza vaccinations, and the ability to rise from the floor unassisted within 10 seconds."
Answered by AI
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