Inherited Metabolic Diseases Case Study

Cmed is currently working on a Phase II trial with a US Pharmaceutical company involving pediatric patients with selected inherited metabolic disorders undergoing stem cell transplantation. The study aims to magnify the necessary cell population within a UCB to quicken the time to reach transplant effectiveness and ultimately aims to positively impact the transplant success and survival rate.

 

Inherited metabolic diseases

Inherited metabolic diseases (IMD), including Hurler syndrome, cerebral adrenoleukodystrophy (cALD), globoid cell leukodystrophy (GLD or Krabbe) and metachromatic leukodystrophy (MLD), are a group of rare, inherited disorders that result in a progressive loss of neuromotor and cognitive abilities. Untreated, these disorders are lethal.

Current therapeutic solutions

The only accepted therapy for Hurler syndrome, MLD, GLD, and cALD is allogeneic hematopoietic stem cell transplantation (HSCT), after which immature cells of hematopoietic origin migrate to tissues, including the central nervous system.

 

 

Challenges for patients

HSCT has a high risk of complications and death. Deaths from transplant-related complications occur in 20% to 30% of patients.

Unrelated Umbilical Cord Blood (UCB) donor programs are most often utilized as treatment. The amount of necessary cells (stem and progenitor) within a UCB unit is infinitely small and therefore takes a very long time to be effective for the patient, a primary factor of the deaths observed.

Cmed Solutions

Enrolment: Changes have been made to the protocol to extend the inclusion criteria; motivational visits to site for support have been conducted; additional sites have been activated; the Sponsor website has been updated to support parents researching the study.

Recurring IDMC meetings: Scheduled and ad hoc IDMCs have been supported by encapsia EDC, enabling committee members to assess real-time data.

IMP supply: Manufacturing complexities have been managed plus transparent and timely communication with the site has enabled the process to run as smoothly as possible.

Medical Monitoring: Medical monitors are medical science experts who answer any questions the clinical team and sites may have, review the data in encapsia (Cmed’s proprietary EDC and analytics platform) in real-time, and respond to any safety findings that require the sponsor to convene an ad hoc IDMC meeting.

Case Study Key Facts    
Disease/indication Inherited metabolic diseases (IMD), including Hurler syndrome, cerebral adrenoleukodystrophy (cALD), globoid cell leukodystrophy (GLD or Krabbe) and metachromatic leukodystrophy (MLD)
Type of study Phase 2 trial with pediatric patients
Size of study 9 patients
Client type Mid Size Biotech
Client base East Coast USA
Project timeline 2017 - ongoing
Other background Stem Cell Therapy

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