Trial Outcomes & Findings for Open-label Extension of the HOPE-2 Trial (NCT NCT04428476)

Last Updated: 2025-11-03

Results Overview

Adverse event (AE) is defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of study drug, whether or not considered related to the study drug. Serious AE: an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. TEAE: AE with onset after start of treatment or with onset date before the treatment start date but worsening after the treatment start date. TEAEs included both serious and non-serious TEAEs.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

13 participants

Primary outcome timeframe

Baseline up to Month 12

Results posted on

2025-11-03

Participant Flow

Participant milestones

Participant milestones
Measure	Deramiocel Participants will receive an intravenous (IV) infusion of deramiocel (150 million Cardiosphere-Derived Cells (CDCs) per infusion) every 3 months for total of 20 IV infusions.
Overall Study STARTED	13
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	13

Reasons for withdrawal

Reasons for withdrawal
Measure	Deramiocel Participants will receive an intravenous (IV) infusion of deramiocel (150 million Cardiosphere-Derived Cells (CDCs) per infusion) every 3 months for total of 20 IV infusions.
Overall Study Withdrawal by Subject	1
Overall Study Still on treatment	12

Baseline Characteristics

Open-label Extension of the HOPE-2 Trial

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Deramiocel n=13 Participants Participants will receive an intravenous (IV) infusion of deramiocel (150 million Cardiosphere-Derived Cells (CDCs) per infusion) every 3 months for a total of 20 IV infusions.
Age, Continuous	16.5 years STANDARD_DEVIATION 3.76 • n=3 Participants
Sex: Female, Male Female	0 Participants n=3 Participants
Sex: Female, Male Male	13 Participants n=3 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=3 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	11 Participants n=3 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=3 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=3 Participants
Race (NIH/OMB) Asian	2 Participants n=3 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=3 Participants
Race (NIH/OMB) Black or African American	0 Participants n=3 Participants
Race (NIH/OMB) White	11 Participants n=3 Participants
Race (NIH/OMB) More than one race	0 Participants n=3 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=3 Participants

PRIMARY outcome

Timeframe: Baseline up to Month 12

Population: Safety population included all participants who received investigational product.

Outcome measures

Outcome measures
Measure	Deramiocel n=13 Participants Participants will receive an intravenous (IV) infusion of deramiocel (150 million Cardiosphere-Derived Cells (CDCs) per infusion) every 3 months for a total of 20 IV infusions.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) From Baseline Through Month 12	11 Participants

PRIMARY outcome

Timeframe: Baseline up to Month 12

Population: Safety population included all participants who received investigational product.

Severity of adverse events (AE) were assessed by the investigator as Grade 1 = Mild (Transient or mild discomfort; no limitation in activity; no medical intervention/therapy required), Grade 2 = Moderate (Mild to moderate limitation in activity - some assistance may be needed; no or minimal medical intervention/therapy required), Grade 3 = Severe (Marked limitation in activity, some assistance usually required; medical intervention/therapy required and often requiring hospitalization or prolongation of hospitalization), Grade 4 = Life-threatening (Extreme limitation in activity, significant assistance required; significant medical intervention/therapy required; hospitalization, prolongation of hospitalization, or hospice care) and Grade 5 = Death.

Outcome measures

Outcome measures
Measure	Deramiocel n=13 Participants Participants will receive an intravenous (IV) infusion of deramiocel (150 million Cardiosphere-Derived Cells (CDCs) per infusion) every 3 months for a total of 20 IV infusions.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Severity From Baseline Through Month 12 Grade 3	2 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Severity From Baseline Through Month 12 Grade 1	6 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Severity From Baseline Through Month 12 Grade 2	3 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Severity From Baseline Through Month 12 Grade 4	0 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Severity From Baseline Through Month 12 Grade 5	0 Participants

PRIMARY outcome

Timeframe: Baseline, Month 12

Population: Safety population included all participants who received investigational product.

PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function.

Outcome measures

Outcome measures
Measure	Deramiocel n=13 Participants Participants will receive an intravenous (IV) infusion of deramiocel (150 million Cardiosphere-Derived Cells (CDCs) per infusion) every 3 months for a total of 20 IV infusions.
Change From Baseline in Functional Capacity as Assessed by Performance of the Upper Limb Test, Version 2 (PUL 2.0) Total Score.	-1.8 units on a scale Standard Deviation 3.07

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Deramiocel n=13 participants at risk Participants will receive an intravenous (IV) infusion of deramiocel (150 million Cardiosphere-Derived Cells (CDCs) per infusion) every 3 months for a total of 20 IV infusions.
Cardiac disorders Cardiac Dysfunction	15.4% 2/13 • Up to Month 12
Cardiac disorders Supraventricular Extrasystoles	7.7% 1/13 • Up to Month 12
Cardiac disorders Tachycardia	7.7% 1/13 • Up to Month 12
Cardiac disorders Ventricular Extrasystoles	7.7% 1/13 • Up to Month 12
Endocrine disorders Secondary Hypogonadism	7.7% 1/13 • Up to Month 12
Gastrointestinal disorders Nausea	15.4% 2/13 • Up to Month 12
General disorders Pyrexia	23.1% 3/13 • Up to Month 12
Infections and infestations Eyelid Infection	7.7% 1/13 • Up to Month 12
Infections and infestations Nasopharyngitis	7.7% 1/13 • Up to Month 12
Infections and infestations Pharyngitis Streptococcal	15.4% 2/13 • Up to Month 12
Injury, poisoning and procedural complications Contusion	15.4% 2/13 • Up to Month 12
Injury, poisoning and procedural complications Ligament Injury	7.7% 1/13 • Up to Month 12
Injury, poisoning and procedural complications Lower Limb Fracture	7.7% 1/13 • Up to Month 12
Injury, poisoning and procedural complications Lumbar Vertebral Fracture	7.7% 1/13 • Up to Month 12
Injury, poisoning and procedural complications Procedural Pain	7.7% 1/13 • Up to Month 12
Investigations Heart Rate Increased	7.7% 1/13 • Up to Month 12
Investigations Respiratory Rate Increased	7.7% 1/13 • Up to Month 12
Metabolism and nutrition disorders Decreased Appetite	7.7% 1/13 • Up to Month 12
Musculoskeletal and connective tissue disorders Arthralgia	7.7% 1/13 • Up to Month 12
Musculoskeletal and connective tissue disorders Deformity Thorax	7.7% 1/13 • Up to Month 12
Musculoskeletal and connective tissue disorders Muscular Weakness	7.7% 1/13 • Up to Month 12
Musculoskeletal and connective tissue disorders Musculoskeletal Pain	7.7% 1/13 • Up to Month 12
Musculoskeletal and connective tissue disorders Scoliosis	15.4% 2/13 • Up to Month 12
Nervous system disorders Dysgeusia	7.7% 1/13 • Up to Month 12
Nervous system disorders Headache	30.8% 4/13 • Up to Month 12
Nervous system disorders Paraesthesia	7.7% 1/13 • Up to Month 12
Psychiatric disorders Anxiety	7.7% 1/13 • Up to Month 12
Respiratory, thoracic and mediastinal disorders Cough	23.1% 3/13 • Up to Month 12
Respiratory, thoracic and mediastinal disorders Dysphonia	7.7% 1/13 • Up to Month 12
Respiratory, thoracic and mediastinal disorders Epistaxis	7.7% 1/13 • Up to Month 12
Respiratory, thoracic and mediastinal disorders Nasal Congestion	7.7% 1/13 • Up to Month 12
Respiratory, thoracic and mediastinal disorders Tachypnoea	7.7% 1/13 • Up to Month 12
Respiratory, thoracic and mediastinal disorders Throat Irritation	15.4% 2/13 • Up to Month 12
Skin and subcutaneous tissue disorders Mechanical Urticaria	7.7% 1/13 • Up to Month 12
Skin and subcutaneous tissue disorders Rash	7.7% 1/13 • Up to Month 12
Skin and subcutaneous tissue disorders Skin Disorder	7.7% 1/13 • Up to Month 12
Vascular disorders Deep Vein Thrombosis	7.7% 1/13 • Up to Month 12
Vascular disorders Flushing	23.1% 3/13 • Up to Month 12

Additional Information

Mark Awadalla

Capricor, Inc.

Phone: 858-727-1755

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place