Hemophilia studies are built on a diary: every bleed, every infusion, every treated event, counted per year. Capture gives participants a phone diary, gives sites the factor activity, inhibitor and joint forms, and keeps one audit trail across both. Free sandbox, no credit card, pay only at go-live.
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| Assessment | Screen | Baseline | Wk 4 | Wk 12 | Wk 26 | Wk 52 |
|---|---|---|---|---|---|---|
| Bleed and infusion diary | D | D | D | D | D | |
| Factor activity (trough or peak) | ||||||
| Inhibitor titre (Bethesda) | ||||||
| Joint health assessment | ||||||
| Haemophilia quality of life | ||||||
| Adverse events and thrombosis review |
x = visit form, D = participant diary entries
What a hemophilia trial needs from its EDC
The study data
Annualised bleeding rate (ABR) looks simple and hides a lot of definition. Registered trials typically count only treated bleeds, meaning bleeds followed by a dose of the study product or another factor, and exclude surgery-related bleeds. One efanesoctocog alfa protocol derives ABR as treated bleeds divided by days in the efficacy period times 365.25 and treats a bleed as ending 72 hours after the last treating injection; an emicizumab protocol analyses ABR with negative binomial regression to allow for different follow-up times. Other trials report total ABR at 28 weeks with a 52-week measure as secondary, and separate spontaneous from traumatic bleeds. The point for an EDC: capture enough per bleed (date and time, anatomical site, joint or muscle or other, spontaneous or traumatic, treated yes or no, and treatment given) that your own analysis plan can apply any of these definitions.
The data live in two places. Participants log bleeds and infusions in a diary on their phone, and site staff review them at visits. The diary can use date, time, single-choice, numeric and long-text question types and is opened from a secure link with no app install; reminders and completion windows are set per task. Site staff then confirm and complete each bleed on a site form, adding anything the participant could not, and edit checks query a bleed with no treatment record. See digital patient diary and ePRO compliance and diary completion.
Hemophilia designs vary widely: prophylaxis studies with factor concentrates, non-factor agents given subcutaneously, gene therapy with a single infusion and years of follow-up, pharmacokinetic studies with intensive sampling, surgery studies recording haemostatic efficacy, and immune tolerance induction in inhibitor patients. Each shares the same bleed and exposure core; only the surrounding forms change.
Joint damage drives long-term disability. Studies record target joints (commonly defined as three or more bleeds into one joint in a six-month window, though protocols differ), clinician joint scores such as the Hemophilia Joint Health Score, and sometimes imaging scores. A table section holds one row per joint with the score components; keep licensing of any published score with your study documents. Quality-of-life instruments specific to hemophilia, such as Haem-A-QoL for adults, are completed by participants on their phone.
Episode
Start date and time
BLSTDTLocation
BLLOCJoint
BLJOINTCause
BLCAUSETreated with factor or study drug
BLTRTCounts toward treated ABR per the SAP
Doses to resolve
BLDOSESProtocol to build
| Protocol element | What the data looks like | Where it lives in Capture |
|---|---|---|
| Bleed episodes | Date, site, cause, treated, doses | Participant diary plus site confirmation form |
| Prophylaxis and on-demand infusions | Date, product, lot, dose, reason | Infusion diary and a repeating table on the site form |
| Factor activity | FVIII or FIX activity (one-stage or chromogenic), trough or peak | Lab form with assay method field and units |
| Inhibitors | Bethesda titre, date, neutralising result | Lab form; a query when a titre crosses a protocol threshold |
| Exposure history | Cumulative exposure days, prior products | Medical history and treatment history forms |
| Joint health | Score per joint, target joint flag | Repeating joint table |
| Quality of life | Disease-specific questionnaire | Participant questionnaire on a phone link |
| Safety | Thrombotic events, injection-site reactions, hepatic events | Adverse event form with SAE sign-off |
Bleed definitions, thresholds and exposure-day requirements are protocol-specific. Build to yours.
Build the diary and bleed confirmation form in the free sandbox and put it on a phone. No credit card.
Inhibitors and pharmacokinetics
Inhibitor development is the main immunological risk of factor replacement. Previously treated patient studies test for inhibitors at set intervals and after exposure milestones, and an immune tolerance induction protocol can set its primary endpoint as titre below 0.6 Bethesda units per mL on two consecutive measurements, with factor recovery. Capture holds the result, the assay method and the date, and an edit check can raise a query when a titre crosses a threshold written into the protocol, leaving the clinical decision to the investigator.
Pharmacokinetic studies sample at precise times after dosing (pre-dose, then multiple timepoints out to days). Calculated fields can derive minutes or hours between two date-times, such as dose to sample, so deviations from the nominal time are visible. Laboratory results enter by site staff transcription; there is no built-in laboratory import, so agree an exchange process with the central laboratory. See lab data and reference range management and, for the broader blood-disease set, EDC for hematology trials.
Some products can raise thrombotic risk or affect liver enzymes, so safety forms should combine a thrombosis review, liver chemistry and concomitant bypassing-agent or factor use in the same visit. Serious events route through the SAE workflow.
Build sequence
Upload the protocol; the AI builder proposes a schedule and forms that a person reviews.
Agree the data dictionary for a bleed with the statistician before building the diary.
Bleed and infusion tasks, reminders, and a completion window, opened from a secure phone link.
Bleed confirmation, factor activity, inhibitor, joint and safety forms.
eConsent with email one-time code and countersignature; paediatric assent for younger participants.
Practice participants log bleeds across a simulated 52 weeks before real enrolment.
Delivery
Hemophilia is diagnosed in childhood, and children and adolescents are often enrolled after adults. Caregivers complete the diary for younger children, and assent runs alongside parental permission; see EDC for paediatric trials. Treatment centres are small, specialist and spread across countries, so QR-code enrolment, site-level numbering and the site coordinator portal fit. Home infusions mean most data is generated away from the clinic; see home health visits in clinical trials.
Where the study product is supplied in kits, the Full Platform plan includes drug supply tracking. Long-term follow-up studies run in the same platform, with field-level audit trail, soft deletes only, and exports in CSV, Excel or SDTM as SAS XPT with Define-XML. Capture is suitable for Phase 1 to 3 studies. See EDC for rare disease trials for the wider playbook.
It records every bleed with date, site, cause and treatment status so ABR can be derived in your analysis. The definition (treated or all bleeds, efficacy period) is set in your statistical plan.
Yes. The diary opens from a secure link in the phone browser, with reminders and a completion window. No app is installed.
As a lab form with titre, assay method and date. Edit checks can query titres that cross a threshold you define.
Yes, in a repeating joint table. Licences for published scores stay with your study documents.
Yes. Follow-up forms stay in the same study, with the audit trail intact across years.
Drug supply tracking is part of the Full Platform plan; see the pricing page.
Free sandbox with every feature, no credit card; you pay at go-live.
Free sandbox with every feature. No credit card, and you pay only when you go live.