Not provided
Not provided
Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| ALX-MCD-501 | Registry Identifier | ALX-MCD-501 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The primary objective is to assess safety and efficacy data of Escherichia coli-derived cPMP in patients with molybdenum cofactor deficiency (MoCD).
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Measure | Description | Time Frame |
|---|---|---|
| Safety and Efficacy | This is a noninterventional, observational, retrospective study to collect data on pediatric patients with MoCD who have received E. coli derived cPMP by intravenous only administration. The study will neither provide treatment with cPMP nor alter any ongoing treatment schedules; rather, its objective is to retrospectively collect data on MoCD history and previous treatment with intravenous E. coli derived cPMP, which is documented in the medical records of patients who have received treatment according to a named patient treatment plan. | For up to 60 months from the initial date of treatment with cPMP |
Not provided
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
All patients who received only intravenous cPMP under named-patient use will be eligible
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Vanderbilt Children's Hospital | Nashville | Tennessee | 37232 | United States | ||
| Children's Hospital of Wisconsin |
Not provided
Not provided
Not provided
Not provided
| Milwaukee |
| Wisconsin |
| 53226 |
| United States |
| Neonatologist, Department of Paediatrics, Mercy Hospital for Women | Heidelberg | Australia |
| Monash Medical Centre | Melbourne | Australia |
| Western Sydney Genetics Program & Sydney Medical School | Westmead | Australia |
| University Hospital of Cologne | Cologne | Germany |
| Frankfurt Children's Hospital | Frankfurt | Germany |
| Akademisches Lehrkrankenhaus der Johannes Gutenberg | Koblenz | Germany |
| Beatrix Children's Hospital | Groningen | Netherlands |
| TC Saglik Bakanligi Gaziantep Cocuk Hastaliklari Hastanesi | Gaziantep | Turkey (Türkiye) |
| Birmingham Children's Hospital | Birmingham | United Kingdom |
| Royal Hospital for Sick Children | Glasgow | United Kingdom |
| Manchester Academic Health Science Centre | Manchester | United Kingdom |
| ID | Term |
|---|---|
| C535811 | Molybdenum cofactor deficiency |
Not provided
Not provided
Not provided