Northwind Genetics · RARE-007
X-linked hypophosphatemia in adults
Pivotal Ph3 placebo-controlled study. Enrolment is critically behind (41% vs 65% planned) and a Type B FDA meeting is scheduled to discuss sample-size re-estimation. EAC has breached the contracted budget.
Sit-rep
Status
Enrolling
Health
At risk
Forecast end
Aug 30, 2028
+259d vs plan
Projected EAC
31.2M USD
+4.8M USD vs contract
Live activity
No activity yetKey performance indicators
Threshold colour is supplementary to the on-target / at-risk / breached label.
- Enrolment
- 74 / 180 (41%)
- Screen failure rate
- 52%
- Query aging (>14d)
- 92 open
- Protocol deviations
- 61
- Projected DBL variance
- +142 days
- Budget burn
- 78%
- Forecast EAC
- $31.2M
Timeline & milestones
Bar shows planned span; tick marks current forecast end. Markers are colour-coded by milestone status (labels in tooltip / table below).
| Milestone | Planned | Actual / forecast | Variance | Status |
|---|---|---|---|---|
| FPI | Mar 1, 2024 | Apr 18, 2024 | +48d | Complete |
| 25% enrolment | Oct 15, 2024 | Feb 8, 2025 | +116d | Missed |
| 50% enrolment | May 30, 2025 | Dec 15, 2025 | +199d | In progress |
| 75% enrolment | Dec 15, 2025 | Aug 20, 2026 | +248d | At risk |
| LPI | Jun 1, 2026 | Feb 15, 2027 | +259d | At risk |
| LPLV | Apr 1, 2027 | Dec 15, 2027 | +258d | Planned |
| Database lock | Jul 15, 2027 | Apr 1, 2028 | +261d | Planned |
| CSR draft | Oct 1, 2027 | Jun 15, 2028 | +258d | Planned |
| CSR final | Dec 15, 2027 | Aug 30, 2028 | +259d | Planned |
| Severity | Title | Owner | Mitigation | Status |
|---|---|---|---|---|
| Critical | Sample size insufficient at current event rate | Biostatistics Lead | Type B FDA meeting requested to discuss adaptive SSR; protocol amendment v4 drafted. | Open |
| High | Patient identification network exhausted at top 5 sites | Feasibility Lead | Expanding to APAC region (3 new countries); RWD partnership signed. | Mitigating |
| High | EAC has breached contracted budget | Finance Lead | Change order in negotiation; sponsor steering committee notified. | Open |
| Medium | Central reader inter-rater variability above threshold | Medical Monitor | Retraining and re-adjudication of 12% of reads in progress. | Mitigating |
| Medium | IRT vendor outage on 2026-04-12 caused 14 mis-randomisations | Data Management Lead | Vendor RCA delivered; all subjects reconciled; CAPA closed. | Closed |
Resources
| Role | Headcount | FTE | Monthly rate | Monthly cost |
|---|---|---|---|---|
| CRA | 11 | 9.0 | 19k USD | 173k USD |
| Data Management | 5 | 4.5 | 16k USD | 71k USD |
| Pharmacovigilance | 3 | 1.8 | 18k USD | 33k USD |
| Biostatistics | 3 | 2.5 | 23k USD | 56k USD |
| Medical Writing | 1 | 0.3 | 20k USD | 6k USD |
| Regulatory | 2 | 1.0 | 21k USD | 21k USD |
| Total | 25 | 19.1 | — | 360k USD |
Financials
Contracted budget
26.4M USD
EBV spent
20.6M USD
78% of contract
EAC (estimate at completion)
31.2M USD
+4.8M USD vs contract
Burn vs time elapsed
78% / 51%
spend ahead of schedule
Recent activity
Provenance events (24h)
1087
Staging items pending
19
Signatures (7d)
41
View provenance for this study →View staged records for this study →
Study goals
Demonstrate ≥ 30% improvement in 6-minute walk test vs placebo at Week 40
Maintain blinded data integrity through interim analysis
Obtain Type B FDA alignment on adaptive sample-size re-estimation
Hold projected DBL within +60 days of original plan