Proof-of-Concept Clinical Trials
Early-Phase Clinical Expertise in Proof-Of-Concept Studies
Proof-of-concept provides a critical bridge between Phase I heathy volunteer studies and Phase II patient clinical trials, making an experienced CRO partner like Altasciences vital to achieving optimal outcomes for early-phase clinical research.
Whether you are expanding a first-in-human (FIH) study with patient cohorts or conducting a standalone POC study, our role is to help you detect early signals of clinical efficacy through the right biomarkers, endpoints, and patient populations. Our integrated early-phase expertise ensures your POC study generates the actionable insight needed to support go/no-go decisions and Phase II/III strategy.
Early-Phase Clinical Expertise in Proof-Of-Concept Studies
Altasciences is the premier organization for early-phase clinical research with extensive expertise in conducting POC studies. We have designed, managed, conducted, analyzed, and reported on hundreds of POC studies, with varying degrees of complexity.
First-in-Human Studies
We focus on early-phase development, so your FIH trial will never compete for resources with larger, later-phase trials. We have the know-how to design and implement adaptive complex protocols, incorporating patients, early POC measures and efficacy endpoints, biomarkers, pharmacodynamic assessments, and more. With our centralized program and project management, we help you achieve critical Phase I milestones efficiently to maximize your investment.
Standalone Proof-of-Concept Studies
With Altasciences, your POC study can be designed once initial efficacy-relevant data emerges. Our approach focuses on selecting the right biomarkers, clinical endpoints, and patient cohorts to evaluate early therapeutic activity. Operational readiness—including parallel feasibility, early site engagement, and adaptive designs—supports faster access to the data you need, but the goal remains early insight into efficacy to inform Phase Ib/IIa progression.Our tailor-made, fully integrated solutions, adaptive protocol designs, and focused clinical study management ensure we’re ready to support your next steps, in Phase Ib and IIa clinical trials.
Integrated Clinical Solutions for Phase Ib/IIa Studies
We offer a comprehensive solution to site selection and the flexibility to conduct your study entirely through our clinical site network, solely within our clinical research units, or through a hybrid model utilizing both. Through expert feasibility and site identification, we assess each site's capabilities based on therapeutic alignment and patient access, geographic and regulatory considerations, and historical enrollment performance to ensure the right fit for your protocol. Combining our three North American clinics with a global network of 300+ active partner sites, we have the infrastructure to meet your specific needs to access a variety of specialized patient populations and therapeutic areas.
Watch our podcast to discover how our unique site identification and feasibility process lays the groundwork for a quick study startup.
Podcast— Strategic Site Identification: A Proven Approach
Learn more about our Clinical Trial Site Identification & Selection services.
Accelerated Proof-Of-Concept Clinical Trial Start-up
We know that speed matters in early development, because it accelerates access to early clinical data. That’s why we have developed a comprehensive and streamlined approach to our start-up process that is proactive while reducing common risks to support both a single site execution and complex multi-center strategy. Our dedicated Project Manager coordinates all aspects of planning and activation, ensuring seamless communication and consistency across all clinical locations, cross-functional areas, and vendors. Because we operate as a unified team, our Altasciences integrated Clinical Research Support Services are built for speed, agility, accuracy, and consistency to help you begin collecting early efficacy-relevant data sooner—supporting informed continuation, modification, or termination or your program.
Our proven FIH hybrid model employs a two-pronged strategy to mobilize initiation of early healthy volunteer cohorts at one of our Altasciences clinics in parallel, while expanding patient populations at one of our external partner sites —without delay. If your trial is best suited for our clinical site network, Altasciences expertly leverages our vetted sites. With our long-standing site relationships, we have pre-established Master Services Agreements (MSAs) and budget templates, allowing us to significantly reduce start-up timelines by eliminating common bottlenecks.
Knowing the operational aspects of our partners, which are tracked in our proprietary Ask Albert proprietary database, and continued engagement and discussions on growing capabilities, Altasciences Site Solutions has a streamlined feasibility and operational planning process with our external partner sites, allowing for accelerated initiation without sacrificing quality or compliance. This level of readiness ensures sponsors can evaluate early signs of therapeutic activity as soon as possible which is foundational to POC decision-making.
Proof-Of-Concept Clinical Study Enrollment Strategies
We tailor our enrollment strategies to match your study’s needs, leveraging the strengths of both our internal clinics and our external site network to ensure the appropriate patient populations are recruited to evaluate early clinical efficacy and biomarker responses.
At Altasciences’ clinics, we routinely perform POC clinical research in healthy participants and also leverage our access to a wide range of specialty patient populations for indications that cannot be administered in healthy participants.
Study Participant Populations
- Healthy Participants
- Special Populations
- Patient Populations
Prior to study start, our recruiting department produces an accurate estimate of enrollment projections and timelines verifying that the availability and characteristics of the population support meaningful POC signal detection. For some indications, we can recruit a large number of patients (i.e., diabetes, obesity, and osteoarthritis), while in others, we conduct POC research with smaller cohorts and may involve external sites.
For studies requiring patient populations not readily available in-house, or those needing geographic diversity, we engage our clinical site network. Each partnership is supported by a structured enrollment strategy and proactive risk management framework to ensure success.
- Pre-award feasibility, which leverages existing relationships with partnered sites, provides reliable site screening, enrollment and patient data allowing accurate early modeling and protocol design feedback.
- Post-award feasibility focuses on selecting the best, fit for purpose sites evaluating population access, historical performance, and competing studies.
- Project and site-specific enrollment plans are developed that align with protocol requirements and local patient availability.
- Risk identification tools are used to flag potential enrollment barriers early (e.g., seasonal factors, eligibility constraints, or regulatory delays)
- Ongoing communication with site staff allows for effective patient enrollment and retention monitoring with contingency planning for underperforming sites, including access to backup sites through our broader network.
Turnkey Solution for Proof-Of-Concept Trials
At Altasciences, we offer a turnkey solution for conducting POC studies, handling every aspect of your trial from start to finish. Our comprehensive services include:
Study Design:
Our clinical pharmacologists perform background research, and consult with research specialists, to ensure we craft the ideal, customized design to meet your POC objectives, including collaborating with our preclinical colleagues to select the models or biomarkers, endpoints, and translational measures needed to evaluate early efficacy.
Protocol Review and Development:
Protocol deviations can pose serious threats to the overall success of a clinical trial, which is why we are committed to mitigating these risks before they occur. Through detailed protocol analysis and top-level insight, our risk management planning identifies, evaluates, controls, communicates, reviews, and reports risks.
Clinical Conduct:
Our three state-of-the-art clinics across North America are fully equipped to run your POC study, with expert teams managing dosing, sampling, and intensive monitoring. We have dedicated USP 797 pharmacies on site and, from QT assessment to pain scales, cognitive evaluations to MRI scans, we can deliver the specialized procedures your study requires.
Comprehensive Clinical Monitoring:
Our CRAs are conveniently located in close proximity to our clinics across North America and offer clinical trial expertise and experience in multiple therapeutic indications.
Real-time tracking of pharmacokinetics (PK) and pharmacodynamics (PD) data, safety assessments, and adverse event reporting, ensures compliance with regulatory requirements and maintaining data integrity throughout the POC trial.
- Remote and Decentralized Monitoring:
Using remote monitoring platforms to support virtual visits and real-time communication, we improve monitoring efficiency as well as patient experience and retention. Further increasing efficiency, decentralized monitoring leveraging mobile apps and wearable devices to collect real-time patient data remotely, can reduce the need for site visits while also expanding access to diverse populations./li> - Risk Based Monitoring (RBM:
Leveraging centralized analytics to identify and prioritize high-risk sites, evaluations, and datapoints, allows us to efficiently allocate our CRA resources to conduct targeted oversight. We ensure continuous risk assessment throughout the trial, focusing monitoring efforts where they are most needed.
Bioanalytical Services:
Our state-of-the-art, co-located bioanalytical labs are equipped with over 685 validated methods for a wide range of POC studies, ensuring precise and timely data delivery. Leveraging these established methods, we provide the reliable data you need to meet regulatory standards, informing and enabling faster decision-making. We support clinical trials worldwide with accurate, efficient, and safe preparation, collection, and shipment of clinical samples. Our customized collection kits and manuals are designed to facilitate the handling of your samples at every step of your study.
Data Services and Reporting:
We handle data management, analysis, and comprehensive reporting to deliver high-quality results on time. Our data management and reporting services are designed to deliver accuracy, speed, and regulatory confidence from study start to submission. With integrated teams, industry-leading technology, and a relentless focus on data quality and security, we help you make informed decisions faster while ensuring full compliance and transparency every step of the way. Experience real-time data access, customizable reporting, and expert guidance that put your trial - and your patients - first.
Regulatory Consulting:
Need support navigating regulatory requirements? We begin with the end in mind and can engage with you as early as possible to map out your complete development plan. From preclinical testing through FIH, POC, and clinical pharmacology testing, our experts can assist with mapping out what will be required to satisfy guidelines across regulatory agencies and when you should conduct each phase, including the critical POC trial. We begin with the end in mind and can engage with you as early as possible to map out your complete development plan.
At Altasciences, we’re fully equipped to conduct your POC study, deliver it on time, and ensure it is fully enrolled with the right participants.
Related Resources for POC Clinical Trials
Including proof of concept arms in a Phase I protocol gives you earlier access to data, enabling more informed go/no-go decisions early in your program. Learn how this strategy can accelerate development, optimize resource allocations, reduce cost, and balance timelines. Learn more
With facilities across Canada and the U.S., Altasciences offers an integrated, comprehensive solution for all your early phase development needs. Discover each of our facilities and their unique expertise.
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Choosing the right CRO/CDMO for your FIH trials enables you to run a successful study that generates high-quality data with speed and ease.
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Discover how ethnobridging can prevent the need for region-specific development programs, helping you safely achieve commercial milestones more efficiently.
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Learn More About Our Phase I-Ii Clinical Services
Click below to explore more of Altasciences’ clinical solutions.
Proof-Of-Concept Clinical Trials FAQs
What is clinical proof of concept (POC)?
POC clinical studies are an early stage of clinical drug development, when a compound has shown potential for human therapeutic use, after preclinical animal models and early safety testing. This step often links Phase I (first in human) and dose-ranging Phase-II studies. Proof of concept is a milestone in drug development, as it is a critical step towards marketing a viable human therapeutic. Read the relevant FDA guideline here.
What are some of the challenges associated with a proof-of-concept study?
It is important to design your proof of concept study in such a way that you maximize your opportunities. Working with our regulatory experts, we provide guidance in consideration of preclinical results for design of POC clinical research, biomarker selection, possible additional evaluations required in the case of CNS activity, such as driving simulation or human abuse potential, and all the other details that should be considered for an efficient and effective proof of concept study.
Does Altasciences offer clinical trial monitoring?
Yes, we have a dedicated team of monitors who ensure your trial stays on track, from site selection and qualification through source document verification, eCRF review, and all other questions related to the successful conduct of your POC study.
What services do you offer to help us beyond POC?
We offer CDMO manufacturing for small molecules for all phases of clinical trials (including blinding capabilities), through to commercial manufacturing and packaging. Our bioanalytical labs support trials to Phase IV and offer stand-alone services. In fact, all our research support services can be offered as stand-alone services, to support you up to, including, and beyond your POC clinical research.
How Long does a typical proof of concept study take from start to finish?
The duration of recruitment, enrollment, and conduct of the clinical portion of any POC study varies based on patient population, inclusion/exclusion criteria, and study design. Study start-up and close-out are the phases when we can really leverage our processes and resources to align with early-phase trial durations. Understanding the difference between POC studies and large-scale Phase III trials, we right-size our approach. Site selection begins at the time of first inquiry, and we have streamlined site contracting to ensure that the key rate limiters in study start-up do not slow us down. This allows us to maintain early-phase start-up as well as backend timelines from LSLV to topline results, database lock, and final analysis.
What are the key success factors in proof-of-concept studies?
- Patient engagement and retention
- Risk Mitigation
What is the benefit of adding proof-of-concept arm to a first-in-human study?
The key benefit of including a POC arm in an FIH study is that it provides early efficacy data earlier in the development process, aligning with the goals of de-risking, attracting investment, or exit strategy. This approach can significantly enhance the company’s ability to achieve a favorable exit or secure additional funding while saving time and reducing overall development costs.