Muscular Dystrophy, Duchenne
Conditions
Keywords
Muscular Dystrophies, Musculoskeletal Diseases, Nervous System Diseases, Neuromuscular Diseases, Duchenne muscular dystrophy, DMD, dystrophin, dystrophy, Duchenne
Brief summary
The MoveDMD study is a 3-part, Phase 1/2, multi-site study to evaluate the safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of edasalonexent (also known as CAT-1004) in pediatric patients with a genetically confirmed diagnosis of DMD. Male patients from ≥4 to \<8 years of age will be enrolled. Edasalonexent is an orally administered small molecule targeted to inhibit activated NF-κB, a molecule that is activated from infancy in DMD and which is central to causing muscle damage and preventing muscle regeneration. Data on magnetic resonance imaging of the lower and upper leg muscles, physical function (including timed function tests) and muscle strength will be studied.
Detailed description
Part A was a 1-week, open-label study to assess safety, tolerability, pharmacokinetics and biomarkers for three dose levels of edasalonexent and is now complete. Part B was a randomized, double-blind, placebo-controlled, multiple dose study to evaluate the safety, efficacy, PK, and PD of edasalonexent over 12 weeks. Patients who participated in Part A also participated in Part B, along with newly enrolled patients. Patients received either edasalonexent 67 mg/kg/day, edasalonexent 100 mg/kg/day, or placebo in Part B. Part B is now complete. Following completion of Part B, patients receive edasalonexent for 138 weeks in Part C, the open-label portion of the MoveDMD study. Patients on the 67 mg/kg/day treatment moved to the 100 mg/kg/day treatment. Patients on the 100 mg/kg/day treatment remained on the 100 mg/kg/day treatment. If clinically indicated, concomitant treatment with eteplirsen (Exondys 51™) may be acceptable in patients with amenable gene mutations during Part C after the patient has been exposed to edasalonexent for 6 months. \*\*Following completion of MoveDMD Part C, access to edasalonexent for trial participants will continue through the open-label extension study, GalaxyDMD.\*\*
Interventions
DRUGEdasalonexent
DRUGPlacebo
Sponsors
Catabasis Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
MALE
Age
4 Years to 7 Years
Healthy volunteers
No
Inclusion criteria
* Written informed consent from parent or legal guardian prior to participation and, for patients who are 7 years of age, written assent from patient * Diagnosis of DMD based on a clinical phenotype with increased serum CK and the presence of a mutation in the dystrophin gene known to be associated with a DMD phenotype * Ability to walk independently (assistive devices are permitted) * Adequate immunization for influenza and varicella
Exclusion criteria
* Use of corticosteroids within prior 6 months of treatment initiation or planning to initiate steroid therapy within the next 6 months * Other prior or ongoing significant medical conditions * Exposure to another investigational drug (such as eteplirsen or idebenone) within 28 days prior to start of study treatment or ongoing participation in any other therapeutic clinical trial * Note: There are separate criteria for patients who participated in Part A versus newly enrolling patients. New patients must meet all of the Part A entry criteria to participate in Part B. Patients who participated in Part A must meet the following criteria to participate in Part B: * Completed Part A * Continue to meet all of the Part A entry criteria, including an absence of safety concerns (however, patients may be ≥8 years of age) There are no entry criteria for Part C; all patients who complete Part B will automatically continue in Part C
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B | Baseline to Week 12 | The LLC5-T2 calculated from the unweighted average of the T2 relaxation times of all 5 lower leg muscles for each patient at each evaluation (the medial gastrocnemius, peroneals, soleus, tibialis anterior, and tibialis posterior muscles). Increases in LLC5-T2 relaxation time indicate muscle damage, inflammation, edema, and fat infiltration and are highly correlated with muscle fat |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in the Speed of Completing the 10-meter Walk/Run Test (10MWT) at Week 12 - Part B and Part C | Baseline to Week 12 | The 10MWT determines the speed to walk a distance of 10 meters. The initial measurement was made in seconds and the speed of completing the test (i.e., calculated as the reciprocals of the time to complete; speed=1/time) is provided as the measure. For patients who are not able to complete the test, the speed of 0 will be imputed. |
| Change From Baseline in the Speed of Completing the 4-Stairs Climb Task at Week 12 - Part B and Part C | Baseline to Week 12 | The 4-stair climb test determines the speed to climb 4 standard steps. The initial measurement was made in seconds and the speed of completing the test (i.e., calculated as the reciprocals of the time to complete; speed=1/time) is provided as the measure. For patients who are not able to complete the test, the speed of 0 will be imputed. |
| Change From Baseline in the Speed of Completing the Stand From Supine Task at Week 12 - Part B and Part C | Baseline to Week 12 | The stand from supine test determines the speed to stand from a supine position. The initial measurement was made in seconds and the speed of completing the test (i.e., calculated as the reciprocals of the time to complete; speed=1/time) is provided as the measure. For patients who are not able to complete the test, the speed of 0 will be imputed. |
| Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Screening to Week 152 | A TEAE was any adverse event (AE) that started during or after the first dose of IP through the end of the safety follow-up period. Part B TEAEs were those that started on or after the first dose date in Part B through the date of Week 12 clinic dose. TEAEs for the Part C (Active B or C) analysis were those that started on or after the first dose date of active treatment in Part B or Part C. Drug related AEs included those marked as Related or Possibly Related to the study treatment. |
Countries
United States
Participant flow
Recruitment details
This was a 3-part, Phase 1/2, multi-centre study conducted at 5 study centers in United States.
Pre-assignment details
A total of 32 patients were treated with edasalonexent in this study. 17 patients were enrolled in Part A and 16 completed 7 days of dosing, 1 patient screen failed due to inability to comply with study procedures and did not continue to Part B. A total of 31 patients (16 subjects from Part A and 15 New subjects who entered Part B) were enrolled in Part B, and all completed the 12 weeks of dosing. All 31 patients included in Part B enrolled in the open-label long-term extension phase, Part C.
Participants by arm
| Arm | Count |
|---|---|
| Part A -CAT-1004 33 mg/kg/Day Edasalonexent 33 mg/kg/day. Capsules taken by mouth two times per day
Edasalonexent | 5 |
| Part A - CAT-1004 67 mg/kg/Day Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day
Edasalonexent | 6 |
| Part A - CAT-1004 100 mg/kg/Day Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day
Edasalonexent | 6 |
| Part B - CAT-1004 67 mg/kg/Day Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day
Edasalonexent | 10 |
| Part B - CAT-1004 100 mg/kg/Day Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day
Edasalonexent | 10 |
| Part B: Overall Placebo Placebo1:Placebo 67 mg/kg/day Capsules taken by mouth two times per day Placebo2: Placebo 100 mg/kg/day Capsules taken by mouth three times per day | 11 |
| Total | 48 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 | FG004 | FG005 | FG006 | FG007 | FG008 |
|---|---|---|---|---|---|---|---|---|---|---|
| PartA(7 Day Open-Label Treatment Period) | Screen failure | 1 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Part C (138-week, Open-label Treatment) | Lost to Follow-up | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 2 |
| Part C (138-week, Open-label Treatment) | Progressive disease | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 3 |
| Part C (138-week, Open-label Treatment) | Withdrawal by Parent or Guardian | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 5 | 6 |
| Part C (138-week, Open-label Treatment) | Withdrawal by Subject | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 0 |
Baseline characteristics
| Characteristic | Part A -CAT-1004 33 mg/kg/Day | Part A - CAT-1004 67 mg/kg/Day | Part A - CAT-1004 100 mg/kg/Day | Total | Part B - CAT-1004 67 mg/kg/Day | Part B - CAT-1004 100 mg/kg/Day | Part B: Overall Placebo |
|---|---|---|---|---|---|---|---|
| Age, Continuous Part A | 5.2 years STANDARD_DEVIATION 1.1 | 5.5 years STANDARD_DEVIATION 0.55 | 5.7 years STANDARD_DEVIATION 1.21 | 5.5 years STANDARD_DEVIATION 0.94 | — | — | — |
| Age, Continuous Part B | — | — | — | 6.11 years STANDARD_DEVIATION 1.086 | 5.97 years STANDARD_DEVIATION 1.137 | 6.00 years STANDARD_DEVIATION 1.165 | 6.34 years STANDARD_DEVIATION 1.032 |
| Ethnicity (NIH/OMB) Part A Hispanic or Latino | 0 Participants | 0 Participants | 1 Participants | 1 Participants | — | — | — |
| Ethnicity (NIH/OMB) Part A Not Hispanic or Latino | 4 Participants | 6 Participants | 5 Participants | 15 Participants | — | — | — |
| Ethnicity (NIH/OMB) Part A Unknown or Not Reported | 1 Participants | 0 Participants | 0 Participants | 1 Participants | — | — | — |
| Ethnicity (NIH/OMB) Part B Hispanic or Latino | — | — | — | 3 Participants | 2 Participants | 1 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Part B Not Hispanic or Latino | — | — | — | 28 Participants | 8 Participants | 9 Participants | 11 Participants |
| Ethnicity (NIH/OMB) Part B Unknown or Not Reported | — | — | — | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Part A American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants | — | — | — |
| Race (NIH/OMB) Part A Asian | 0 Participants | 0 Participants | 0 Participants | 0 Participants | — | — | — |
| Race (NIH/OMB) Part A Black or African American | 0 Participants | 0 Participants | 0 Participants | 0 Participants | — | — | — |
| Race (NIH/OMB) Part A More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants | — | — | — |
| Race (NIH/OMB) Part A Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants | — | — | — |
| Race (NIH/OMB) Part A Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | — | — | — |
| Race (NIH/OMB) Part A White | 5 Participants | 6 Participants | 6 Participants | 17 Participants | — | — | — |
| Race (NIH/OMB) Part B American Indian or Alaska Native | — | — | — | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Part B Asian | — | — | — | 1 Participants | 1 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Part B Black or African American | — | — | — | 1 Participants | 0 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) Part B More than one race | — | — | — | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Part B Native Hawaiian or Other Pacific Islander | — | — | — | 1 Participants | 0 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) Part B Unknown or Not Reported | — | — | — | 1 Participants | 0 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) Part B White | — | — | — | 27 Participants | 9 Participants | 9 Participants | 9 Participants |
| Region of Enrollment United States Part A | 5 Participants | 6 Participants | 6 Participants | 17 Participants | 0 Participants | 0 Participants | 0 Participants |
| Region of Enrollment United States Part B | 0 Participants | 0 Participants | 0 Participants | 31 Participants | 10 Participants | 10 Participants | 11 Participants |
| Sex: Female, Male Part A Female | 0 Participants | 0 Participants | 0 Participants | 0 Participants | — | — | — |
| Sex: Female, Male Part A Male | 5 Participants | 6 Participants | 6 Participants | 17 Participants | — | — | — |
| Sex: Female, Male Part B Female | — | — | — | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Part B Male | — | — | — | 31 Participants | 10 Participants | 10 Participants | 11 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk | EG006 affected / at risk | EG007 affected / at risk |
|---|---|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 5 | 0 / 6 | 0 / 6 | 0 / 10 | 0 / 10 | 0 / 11 | 0 / 15 | 0 / 16 |
| other Total, other adverse events | 2 / 5 | 5 / 6 | 5 / 6 | 9 / 10 | 8 / 10 | 9 / 11 | 15 / 15 | 16 / 16 |
| serious Total, serious adverse events | 0 / 5 | 0 / 6 | 0 / 6 | 0 / 10 | 1 / 10 | 0 / 11 | 0 / 15 | 0 / 16 |
Outcome results
Primary
Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B
The LLC5-T2 calculated from the unweighted average of the T2 relaxation times of all 5 lower leg muscles for each patient at each evaluation (the medial gastrocnemius, peroneals, soleus, tibialis anterior, and tibialis posterior muscles). Increases in LLC5-T2 relaxation time indicate muscle damage, inflammation, edema, and fat infiltration and are highly correlated with muscle fat
Time frame: Baseline to Week 12
Population: Part B Full Analysis Population: The Full Analysis Population was a modified intent-to-treat population and consisted of all patients who received at least 1 dose of investigational product (IP) in Part B and had a valid baseline and at least 1 post baseline timed function test (TFT) or MRI efficacy assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Part B - CAT-1004 67 mg/kg/Day | Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B | Baseline to Week 12 Endpoint - Tibialis Posterior | 0.62 msec | Standard Deviation 1.107 |
| Part B - CAT-1004 67 mg/kg/Day | Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B | Baseline to Week 12 Endpoint - Tibialis Anterior | -0.25 msec | Standard Deviation 2.558 |
| Part B - CAT-1004 67 mg/kg/Day | Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B | Baseline to Week 12 Endpoint - Soleus | 2.04 msec | Standard Deviation 3.179 |
| Part B - CAT-1004 67 mg/kg/Day | Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B | Baseline to Week 12 Endpoint - Medial Gastrocnemius | 0.35 msec | Standard Deviation 2.41 |
| Part B - CAT-1004 67 mg/kg/Day | Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B | Baseline to Week 12 Endpoint - Peroneal | 0.28 msec | Standard Deviation 2.764 |
| Part B - CAT-1004 100 mg/kg/Day | Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B | Baseline to Week 12 Endpoint - Tibialis Anterior | 0.01 msec | Standard Deviation 1.433 |
| Part B - CAT-1004 100 mg/kg/Day | Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B | Baseline to Week 12 Endpoint - Soleus | 1.26 msec | Standard Deviation 2.913 |
| Part B - CAT-1004 100 mg/kg/Day | Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B | Baseline to Week 12 Endpoint - Medial Gastrocnemius | -0.16 msec | Standard Deviation 2.003 |
| Part B - CAT-1004 100 mg/kg/Day | Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B | Baseline to Week 12 Endpoint - Tibialis Posterior | -1.05 msec | Standard Deviation 2.447 |
| Part B - CAT-1004 100 mg/kg/Day | Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B | Baseline to Week 12 Endpoint - Peroneal | 0.63 msec | Standard Deviation 2.29 |
| Part B - Placebo (Overall) | Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B | Baseline to Week 12 Endpoint - Peroneal | 1.27 msec | Standard Deviation 1.63 |
| Part B - Placebo (Overall) | Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B | Baseline to Week 12 Endpoint - Tibialis Posterior | -0.42 msec | Standard Deviation 2.716 |
| Part B - Placebo (Overall) | Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B | Baseline to Week 12 Endpoint - Soleus | 0.34 msec | Standard Deviation 2.38 |
| Part B - Placebo (Overall) | Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B | Baseline to Week 12 Endpoint - Tibialis Anterior | 0.00 msec | Standard Deviation 1.878 |
| Part B - Placebo (Overall) | Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B | Baseline to Week 12 Endpoint - Medial Gastrocnemius | 1.16 msec | Standard Deviation 2.517 |
Secondary
Change From Baseline in the Speed of Completing the 10-meter Walk/Run Test (10MWT) at Week 12 - Part B and Part C
The 10MWT determines the speed to walk a distance of 10 meters. The initial measurement was made in seconds and the speed of completing the test (i.e., calculated as the reciprocals of the time to complete; speed=1/time) is provided as the measure. For patients who are not able to complete the test, the speed of 0 will be imputed.
Time frame: Baseline to Week 12
Population: The Active B or C Full Analysis Population consisted of the pooled patient populations of Parts B and C that received treatment in either part and had valid baseline and efficacy measurements.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Part B - CAT-1004 67 mg/kg/Day | Change From Baseline in the Speed of Completing the 10-meter Walk/Run Test (10MWT) at Week 12 - Part B and Part C | -0.0008 10 meters/sec | Standard Deviation 0.01742 |
| Part B - CAT-1004 100 mg/kg/Day | Change From Baseline in the Speed of Completing the 10-meter Walk/Run Test (10MWT) at Week 12 - Part B and Part C | -0.0041 10 meters/sec | Standard Deviation 0.01584 |
| Part B - Placebo (Overall) | Change From Baseline in the Speed of Completing the 10-meter Walk/Run Test (10MWT) at Week 12 - Part B and Part C | -0.0100 10 meters/sec | Standard Deviation 0.01627 |
Secondary
Change From Baseline in the Speed of Completing the 4-Stairs Climb Task at Week 12 - Part B and Part C
The 4-stair climb test determines the speed to climb 4 standard steps. The initial measurement was made in seconds and the speed of completing the test (i.e., calculated as the reciprocals of the time to complete; speed=1/time) is provided as the measure. For patients who are not able to complete the test, the speed of 0 will be imputed.
Time frame: Baseline to Week 12
Population: The Active B or C Full Analysis Population consisted of the pooled patient populations of Parts B and C that received treatment in either part and had valid baseline and efficacy measurements.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Part B - CAT-1004 67 mg/kg/Day | Change From Baseline in the Speed of Completing the 4-Stairs Climb Task at Week 12 - Part B and Part C | -0.0032 4 Stairs/Sec | Standard Deviation 0.07487 |
| Part B - CAT-1004 100 mg/kg/Day | Change From Baseline in the Speed of Completing the 4-Stairs Climb Task at Week 12 - Part B and Part C | -0.0046 4 Stairs/Sec | Standard Deviation 0.06223 |
| Part B - Placebo (Overall) | Change From Baseline in the Speed of Completing the 4-Stairs Climb Task at Week 12 - Part B and Part C | -0.0121 4 Stairs/Sec | Standard Deviation 0.06647 |
Secondary
Change From Baseline in the Speed of Completing the Stand From Supine Task at Week 12 - Part B and Part C
The stand from supine test determines the speed to stand from a supine position. The initial measurement was made in seconds and the speed of completing the test (i.e., calculated as the reciprocals of the time to complete; speed=1/time) is provided as the measure. For patients who are not able to complete the test, the speed of 0 will be imputed.
Time frame: Baseline to Week 12
Population: Active B or C Full Analysis Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Part B - CAT-1004 67 mg/kg/Day | Change From Baseline in the Speed of Completing the Stand From Supine Task at Week 12 - Part B and Part C | -0.0293 /Sec | Standard Deviation 0.0493 |
| Part B - CAT-1004 100 mg/kg/Day | Change From Baseline in the Speed of Completing the Stand From Supine Task at Week 12 - Part B and Part C | -0.0042 /Sec | Standard Deviation 0.0389 |
| Part B - Placebo (Overall) | Change From Baseline in the Speed of Completing the Stand From Supine Task at Week 12 - Part B and Part C | -0.0007 /Sec | Standard Deviation 0.05449 |
Secondary
Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs).
A TEAE was any adverse event (AE) that started during or after the first dose of IP through the end of the safety follow-up period. Part B TEAEs were those that started on or after the first dose date in Part B through the date of Week 12 clinic dose. TEAEs for the Part C (Active B or C) analysis were those that started on or after the first dose date of active treatment in Part B or Part C. Drug related AEs included those marked as Related or Possibly Related to the study treatment.
Time frame: Screening to Week 152
Population: Part A Safety Population (referred to in tables as All Dosed Patients) The Safety Population was all patients who received at least 1 dose of IP in Part A.~Part B Safety Population: The Safety Population was all patients who received at least 1 dose of IP in Part B.~Active B or C Safety Population: The Safety Population was all patients who received at least 1 dose of active treatment in Part B and all patients who received placebo in Part B and at least 1 dose of active treatment in Part C.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Part B - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any SAE | 0 Participants |
| Part B - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AE related to study drug | 2 Participants |
| Part B - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AEs after first dose | 2 Participants |
| Part B - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AE leading to discontinuation from study | 0 Participants |
| Part B - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any TEAE | 0 Participants |
| Part B - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any drug-related TEAE | 0 Participants |
| Part B - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any TEAE leading to study drug discontinuation | 0 Participants |
| Part B - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with drug-related TEAEs leading to study drug discontinuation | 0 Participants |
| Part B - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any TEAE | 0 Participants |
| Part B - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any SAE | 0 Participants |
| Part B - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any drug-related TEAE | 0 Participants |
| Part B - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any TEAE leading to study drug discontinuation | 0 Participants |
| Part B - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AEs after first dose | 5 Participants |
| Part B - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AE leading to discontinuation from study | 0 Participants |
| Part B - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AE related to study drug | 2 Participants |
| Part B - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with drug-related TEAEs leading to study drug discontinuation | 0 Participants |
| Part B - Placebo (Overall) | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any TEAE leading to study drug discontinuation | 0 Participants |
| Part B - Placebo (Overall) | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any TEAE | 0 Participants |
| Part B - Placebo (Overall) | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with drug-related TEAEs leading to study drug discontinuation | 0 Participants |
| Part B - Placebo (Overall) | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any SAE | 0 Participants |
| Part B - Placebo (Overall) | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any drug-related TEAE | 0 Participants |
| Part B - Placebo (Overall) | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AE leading to discontinuation from study | 0 Participants |
| Part B - Placebo (Overall) | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AEs after first dose | 5 Participants |
| Part B - Placebo (Overall) | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AE related to study drug | 5 Participants |
| Part B - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with drug-related TEAEs leading to study drug discontinuation | 0 Participants |
| Part B - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AE related to study drug | 0 Participants |
| Part B - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AEs after first dose | 0 Participants |
| Part B - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AE leading to discontinuation from study | 0 Participants |
| Part B - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any SAE | 0 Participants |
| Part B - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any TEAE | 9 Participants |
| Part B - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any drug-related TEAE | 5 Participants |
| Part B - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any TEAE leading to study drug discontinuation | 0 Participants |
| Part B - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AE leading to discontinuation from study | 0 Participants |
| Part B - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any TEAE leading to study drug discontinuation | 0 Participants |
| Part B - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any SAE | 0 Participants |
| Part B - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AEs after first dose | 0 Participants |
| Part B - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with drug-related TEAEs leading to study drug discontinuation | 0 Participants |
| Part B - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any TEAE | 8 Participants |
| Part B - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any drug-related TEAE | 7 Participants |
| Part B - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AE related to study drug | 0 Participants |
| Part B - Overall Placebo | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with drug-related TEAEs leading to study drug discontinuation | 0 Participants |
| Part B - Overall Placebo | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AE related to study drug | 0 Participants |
| Part B - Overall Placebo | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any SAE | 1 Participants |
| Part B - Overall Placebo | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AEs after first dose | 0 Participants |
| Part B - Overall Placebo | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any TEAE leading to study drug discontinuation | 0 Participants |
| Part B - Overall Placebo | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any drug-related TEAE | 1 Participants |
| Part B - Overall Placebo | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any TEAE | 10 Participants |
| Part B - Overall Placebo | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AE leading to discontinuation from study | 0 Participants |
| Part C - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any SAE | 0 Participants |
| Part C - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AE related to study drug | 0 Participants |
| Part C - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AE leading to discontinuation from study | 0 Participants |
| Part C - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any TEAE | 15 Participants |
| Part C - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any drug-related TEAE | 8 Participants |
| Part C - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with drug-related TEAEs leading to study drug discontinuation | 0 Participants |
| Part C - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any TEAE leading to study drug discontinuation | 0 Participants |
| Part C - CAT-1004 67 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AEs after first dose | 0 Participants |
| Part C - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with drug-related TEAEs leading to study drug discontinuation | 0 Participants |
| Part C - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any drug-related TEAE | 11 Participants |
| Part C - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any TEAE | 16 Participants |
| Part C - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AE leading to discontinuation from study | 0 Participants |
| Part C - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any SAE | 1 Participants |
| Part C - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AE related to study drug | 0 Participants |
| Part C - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any AEs after first dose | 0 Participants |
| Part C - CAT-1004 100 mg/kg/Day | Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs). | Patients with any TEAE leading to study drug discontinuation | 1 Participants |