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ALXN1101 · 1 trial · 3 indications
Physical examination, vital signs, ECGs, laboratory evaluations, and Adverse Events.
| Arm | Type | Description |
|---|---|---|
| ALXN1101 | EXPERIMENTAL | Four cohorts planned. Within each cohort, healthy volunteers are randomized to ALXN1101 IV single dose or placebo IV single dose. Each subsequent cohort is testing an increased dose of ALXN1101 IV or placebo IV. |
| Placebo | PLACEBO_COMPARATOR | Four cohorts planned. Within each cohort, healthy volunteers are randomized to ALXN1101 IV single dose or placebo IV single dose. Each subsequent cohort is testing an increased dose of ALXN1101 IV or placebo IV. |
| Name | Type | Description |
|---|---|---|
| ALXN1101 | DRUG | Randomized to receive a single dose of ALXN1101 or placebo as per assigned cohort dose level. |
| Placebo | DRUG | Randomized to receive a single dose of ALXN1101 or placebo as per assigned cohort dose level. |
Key Inclusion Criteria: 1. Male or female subjects ≥ 18 and ≤ 60 years of age and weight ≥ 55 kg and ≤ 100 kg 2. Willing and able to give written informed consent 3. Female subjects of child bearing potential must have a negative serum pregnancy test or must be practicing an approved contraceptive ...
ALXN1101 is an investigational small molecule being developed for Molybdenum Cofactor Deficiency (MoCD), a rare autosomal recessive disorder characterized by deficiency of activity of molybdenum-dependent enzymes, including sulfite oxidase, xanthine dehydrogenase, and aldehyde oxidase. It is currently in clinical development and has not been approved by the FDA.
ALXN1101 is being developed by BridgeBio Pharma, Inc. (ticker: BBIO). The company is conducting clinical trials to evaluate the drug for the treatment of Molybdenum Cofactor Deficiency (MoCD), a rare genetic disorder.
ALXN1101 is in Phase 1 clinical development. A Phase 1 single-dose study in healthy volunteers has been completed. The drug is investigational and has not been approved by regulatory authorities.
ALXN1101 has one completed clinical trial, NCT01894165, a Phase 1 single-dose study in healthy volunteers. The trial enrolled 24 participants in the United States, was randomized, double-blind, and placebo-controlled, and included biomarker selection.
ALXN1101 is a small molecule designed to address Molybdenum Cofactor Deficiency (MoCD), a condition caused by deficiency of molybdenum-dependent enzymes. The drug targets the underlying metabolic defect by providing a source of the molybdenum cofactor, though the specific molecular target is not disclosed in the available data.