Rare and Orphan Diseases
We leverage our experience working closely with patients, advocacy groups, and key opinion leaders to successfully recruit, retain and empower patients.
In the realm of rare diseases, traditional large-scale study approaches can fall short, necessitating a blend of deep scientific expertise and innovative thinking. Our team excels in designing unique programs that maximise the impact of even the smallest patient cohorts.
Common areas in which we have expertise include:
- Huntington’s Disease
- Usher Syndrome
- Ehlers-Danlos Syndrome (EDS)
- Sickle Cell
- Cystic Fibrosis
- Duchenne Muscular Dystrophy (DMD)
- Haemophilia
- Gaucher Disease
- Cutaneous T-Cell Lymphoma
- Glioblastoma Multiforme
Disciplined,
Cost-Efficient Execution
while maintaining the Highest Quality
Local Trials,
Global Approvals
Access FDA Regulatory Expertise
Our Clinical Team has over
120
years
of Combined Clinical Trial Experience
Rare and Orphan Diseases Clinical Trial Resources
Rett Syndrome
Lessons from Successes and Failures in Clinical Trials.
Phelan-McDermid Syndrome
Navigating Diagnostic Criteria, Trial Endpoints, and Drug Development Challenges.
Glioblastoma
Navigating the Complexities of Glioblastoma Clinical Trials: From Diagnosis to Enhanced Protocols.
How iNGENū CRO Addresses Key Pain Points in Respiratory Clinical Trials
Complexity in Respiratory Assessments
Problem:
Respiratory trials rely heavily on pulmonary function tests (PFTs), imaging, and biomarkers, all of which can vary widely depending on technique, equipment, and patient effort.
iNGENū CRO addresses this pain point with Standardised Respiratory Assessments:
iNGENū ensures consistency through centralised training of clinical staff, standardised protocols for spirometry and imaging (e.g., HRCT, PET), and the use of advanced biomarker analysis to ensure reliable, high-quality data collection across all trial sites.
Placebo Effects and Patient Variability
Problem:
Respiratory trials, particularly for conditions like asthma and COPD, are highly susceptible to placebo effects and variability in symptom severity, complicating data interpretation.
iNGENū CRO addresses this pain point with Robust Trial Design and Advanced Analytics:
Our trials are carefully designed with robust blinding methods and include run-in periods and crossover designs where appropriate. Advanced statistical methodologies are employed to account for variability and placebo effects, ensuring precise and credible results.
Patient Recruitment and Retention
Problem:
Recruiting patients with chronic respiratory conditions can be challenging, as these populations often have comorbidities and varying degrees of disease severity, impacting their ability and willingness to participate.
iNGENū CRO addresses this pain point with Targeted Recruitment Strategies:
iNGENū leverages extensive patient databases, collaborations with respiratory specialists, and connections with patient advocacy groups. Our patient-centric approach includes flexible visit schedules, remote monitoring options, and robust patient support systems to maximise recruitment and retention.
Safety Monitoring
Problem:
Respiratory medications often have systemic effects or require inhalation or infusion methods, increasing the importance of vigilant monitoring for adverse events and patient safety.
iNGENū CRO addresses this pain point with Comprehensive Safety Protocols:
Our safety monitoring systems are state-of-the-art, providing real-time surveillance and prompt intervention for adverse events. A dedicated safety oversight committee regularly reviews safety data, ensuring rapid response and transparent communication with all stakeholders to prioritise patient well-being.
Regulatory Complexities
Problem:
Trials involving novel biologics, inhalation devices, or advanced therapies in pulmonology face complex regulatory landscapes requiring meticulous documentation and compliance.
iNGENū CRO addresses this pain point with Regulatory Expertise:
iNGENū’s regulatory affairs specialists are highly experienced in pulmonology-specific submissions, including INDs, NDAs, and device approvals. We provide end-to-end support to navigate regulatory pathways efficiently, reducing delays and accelerating trial progression.
The iNGENū CRO Difference
Local Trials, Global Approvals
We uniquely combine Australian expertise with direct FDA submission capabilities.
Disciplined, Cost-Efficient Execution
iNGENū CRO takes a highly strategic approach to clinical research.
Australian-Headquartered, Asia-Pacific Reach
We provide sponsors with comprehensive trial capabilities.
We have been using iNGENū for a Phase 1, first-in-human trial of our investigational drug. We have been very happy with all aspects of the management of the clinical trial.
Ready to discuss your Rare and Orphan Diseases Clinical Trial?