We recruit the hardest pediatric trials there are. From ultra-rare HAE in children as young as 2 to OI, Leapcure delivers the families other recruiters cannot reach.

Leapcure has recruited across ultra-rare pediatric HAE and OI, reaching scarce, dispersed families through trusted advocacy and a dedicated Patient Success team. On one pediatric HAE study we generated 205 eligible or interested families and engaged 72 physicians across 18 states.

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205
Interested families
for an ultra-rare pediatric HAE trial
72 / 18
Physicians engaged across 18 states
in one pediatric HAE study
23
Advocacy groups engaged
~70% awareness lift in a pediatric HAE community
50+
Global sites mobilized
to widen an ultra-narrow pediatric pool (OI)

Understanding Pediatric Rare Disease as a Clinical Trial Area

Pediatric rare disease recruitment stacks two enrollment problems on top of each other. First, the population is tiny: many eligible children are undiagnosed, or are diagnosed but too young to present in the way a protocol requires. Second, the patient cannot consent, so a protective parent is the sole decision-maker on whether a child enters a trial at all. On top of that, travel demands and complex screening cause even interested families to withdraw, shrinking an already small eligible pool further.

These are rare and ultra-rare pediatric diseases where the eligible population is a fraction of an already small base. Hereditary angioedema (HAE) is commonly cited at about 1 in 50,000 people, with recent systematic reviews placing it nearer 1 to 1.5 cases per 100,000 (Annals of Allergy, Asthma and Immunology, 2023; systematic reviews, 2024). Osteogenesis imperfecta (OI) affects about 1 in 15,000 to 20,000 births, an estimated 20,000 to 50,000 people in the US (NORD; StatPearls, 2024). In any single region these are very small numbers of children, which is why generic recruitment underperforms.

Leapcure reaches scarce families through the advocacy communities they already trust, supports every parent one to one with a dedicated Patient Success team (including families who do not qualify), and has delivered in more than one pediatric rare disease, from ultra-rare HAE in children as young as 2 through OI. We treat the parent as the person we are actually recruiting, build trust before any site referral, and widen the eligible pool with physician outreach and a global site network when a single-country pool is too small.

The Challenge

Pediatric rare disease trials are hard for reasons that compound: the population is tiny and dispersed, the parent is the sole decision-maker, families are reached only through trusted communities, and travel plus complex screening shrink the eligible pool even further.

Tiny, dispersed, often-undiagnosed populations

Many eligible children are undiagnosed, or are diagnosed but too young to present the way a protocol requires. The eligible pool is a fraction of an already ultra-rare base, scattered across regions, so no single market holds enough patients.

205 eligible or interested families generated for an ultra-rare pediatric HAE study, and a roughly 50-site global network used for OI to widen the eligible pool beyond the US.

Parents are the sole, protective decision-makers

A child cannot consent, so the person you are recruiting is a protective parent weighing risk for their child. That decision is earned through honest, empathetic, one-to-one conversation, not volume outreach.

31 in-depth conversations with parents on the HAE program, building trust in the trial process, with empathy-led, one-to-one Patient Success support.

Families are reached only through trusted communities, not cold ads

Rare disease families gather in the advocacy organizations and condition-specific parent groups they already rely on. Cold advertising rarely reaches them, and when it does, it rarely earns trust on its own.

23 advocacy groups engaged on the HAE program, driving roughly a 70% increase in awareness in that community, with multi-channel parent outreach across Facebook, Instagram, and TikTok plus keyword-targeted search for OI.

Travel and complex screening cause withdrawal, and eligible pools can be smaller than expected

Site travel and complex screening cause even interested families to drop out, and the diagnosed eligible pool can turn out smaller than the protocol assumed. Recruitment has to adapt in flight to keep enrollment moving.

Mid-campaign messaging was adjusted to also reach hereditary carriers when the diagnosed HAE pool proved too small (in-flight optimization), with direct engagement of pediatric endocrinologists, orthopedists, and geneticists for OI to surface eligible patients inside existing care pathways.

How Leapcure recruits pediatric rare disease trials

We activate the trusted communities for each condition, support and pre-screen every family one to one before any site referral, and optimize in real time when the diagnosed pool is too small.

1

Activate the trusted communities for each condition

We engage the advocacy communities families already trust for each condition, alongside precision digital and physician outreach mapped to the eligible pediatric population. Research reaches parents through voices they already rely on, not cold lists.

2

Support and pre-screen every family one to one

Each family is supported and pre-screened through real one-to-one conversations before any site referral, with empathy-led Patient Success that includes families who do not qualify. Parents make an informed decision, and sites receive prepared, eligible families.

3

Optimize in real time when the pool is too small

When the diagnosed pool proves too small, we adapt in flight, for example expanding outreach to hereditary carriers or widening to a global site network, so enrollment keeps moving instead of stalling.

Delivered in the hardest pediatric populations: an ultra-rare HAE program in young children, and OI across a global site network

Challenge Context

Programs

Two real pediatric rare disease programs: an ultra-rare HAE study in children ages 2 to 11, and an OI study in children ages 12 to 17, expanding to ages 5 to 18.

Shared challenge

Tiny diagnosed populations, protective parents as the sole decision-makers, and family logistics (travel and complex screening) that shrink an already small eligible pool.

Results & Metrics

Pediatric HAE: Reach

205 eligible or interested families generated, raising awareness of pediatric HAE trials among parents in an ultra-rare population.

Pediatric HAE: Trust and Physician Engagement

31 in-depth conversations with parents, and 72 physicians engaged across 18 states contributing referrals and long-term outreach impact.

Pediatric HAE: Community and In-Flight Optimization

23 advocacy groups engaged, driving roughly a 70% increase in awareness within the pediatric HAE community, with a mid-campaign pivot to hereditary carriers when the diagnosed pool proved too small.

Pediatric OI: Multi-Channel Reach and Global Network

Multi-channel parent outreach (Facebook, Instagram, TikTok) plus keyword-targeted search ads, direct engagement of pediatric endocrinologists, orthopedists, and geneticists, and a network of roughly 50 global sites used to widen an extremely narrow eligible pool beyond the US.

Four channels. Built for pediatric rare disease

Leapcure reaches scarce, dispersed families through the advocacy communities they trust, empathy-led one-to-one parent support, a physician network paired with precision digital, and answer-engine visibility delivered to your trial.

Channel 1: Advocacy communities families trust

  • •Per-condition pediatric rare disease groups and caregiver organizations that families already rely on, engaged as active, co-developed partnerships rather than list buys.
  • •Content is built with these communities so research reaches parents through trusted voices.
  • •40+ advocacy partnerships is the program standard across pediatric rare disease.

Channel 2: Empathy-led Patient Success and one-to-one parent support

  • •A dedicated Patient Success team supports every family one to one, including families who do not qualify.
  • •Honest, empathetic conversations help protective parents make an informed decision for their child.
  • •Supporting every family, qualified or not, builds long-term trust in research within these small communities.

Channel 3: Physician network and precision digital

  • •Multi-state physician engagement surfaces eligible patients inside existing care pathways, including pediatric specialists who manage these conditions.
  • •Parent-targeted multi-channel digital reaches families across Facebook, Instagram, TikTok, and keyword-targeted search.
  • •Together this widens an ultra-narrow eligible pool beyond what advocacy alone can reach.

Channel 4: AEO and AI-search visibility for your trial

  • •Parents increasingly ask AI engines like ChatGPT, Perplexity, and Google's AI Overview which trials their child may be eligible for. We make your trial's information genuinely retrievable for those engines.
  • •We do this with crawlable, server-rendered content, first-party data backed by sourced statistics, a named reviewer, and a visible last-updated date, plus presence in trusted third-party sources such as rare disease advocacy organizations and ClinicalTrials.gov.
  • •This is not schema tricks or markup promises. AI engines cite trusted third-party sources more often than any single brand domain, so our advocacy partnerships and ClinicalTrials.gov presence are the structural advantage we bring to your trial.

Built on the communities rare disease families already trust

Leapcure's partnerships are active and co-developed, not list buys, and 40+ advocacy partnerships is the program standard across pediatric rare disease. We name only confirmed partners and confirm others before logos go live.

Pediatric HAE community organizations

Pediatric HAE community partners, to be confirmed before logos go live

Rare disease caregiver communities

Caregiver organizations serving families across pediatric rare disease

Condition-specific parent and family groups

Per-condition parent and family groups where rare disease families gather

Pediatric rare disease KOLs and community leaders

Clinicians and community leaders trusted within pediatric rare disease communities

Caring for a child with a rare disease? A clinical trial may be an option to explore.

If your child has a rare disease, a clinical trial may be an option to explore alongside their care, never instead of it. We know how much weighs on this decision, and we will never pressure you. Leapcure's team answers your questions honestly, explains what a trial involves, and supports your family step by step.

Explore trials for your child's condition
Leapcure kangaroo mascot

Leapcure has delivered in the hardest pediatric populations, from ultra-rare HAE in children as young as 2 to OI recruited across a global site network, reaching scarce, dispersed families through the advocacy communities they trust and a Patient Success team that supports every parent one to one. We treat the parent as the person we are recruiting, build trust before any site referral, and optimize in real time when the eligible pool is too small. We can mobilize quickly.

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