Acute Intermittent Porphyria Clinical Trial
Official title:
Acute Porphyrias: Biomarkers for Disease Activity and Response to Treatment
Verified date | April 2024 |
Source | The University of Texas Medical Branch, Galveston |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
The long term objective of the research is to identify new biomarkers of disease activity in the human acute porphyrias. This pilot study is intended to provide pilot and feasibility data needed to plan larger and more definitive future studies.
Status | Active, not recruiting |
Enrollment | 50 |
Est. completion date | December 31, 2025 |
Est. primary completion date | December 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: - Documented diagnosis of acute porphyria. 1. For AIP: Elevation in urine PBG, with normal or only slight increases in plasma and fecal porphyrins. Most (~90%) will have deficient activity of erythrocyte PBGD. 2. For HCP: Elevation in urine PBG, with substantial increases in fecal porphyrins (almost entirely coproporphyrin III). In the absence of skin photosensitivity, most will have normal or only slight increases in plasma porphyrins. 3. For VP: Elevation in PBG, with substantial increases in fecal porphyrins (mostly coproporphyrin III and protoporphyrin), increased plasma total porphyrins and a fluorescence emission maximum of diluted plasma at neutral pH near 626 nm. Exclusion Criteria: - Another medical condition that might confound the results, as judged by the investigator |
Country | Name | City | State |
---|---|---|---|
United States | University of Texas Medical Branch | Galveston | Texas |
Lead Sponsor | Collaborator |
---|---|
The University of Texas Medical Branch, Galveston |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Potential biomarkers Biomarkers | Expression of heme biosynthetic and heat and stress response genes | 10 days |
Status | Clinical Trial | Phase | |
---|---|---|---|
Terminated |
NCT02922413 -
Panhematin for Prevention of Acute Attacks of Porphyria
|
Phase 2 | |
Completed |
NCT02949830 -
A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)
|
Phase 1/Phase 2 | |
Completed |
NCT02943213 -
Assessment of Intra-subject Variability in the Bioavailability of Chlorpromazine Hydrochloride
|
Phase 1 | |
Completed |
NCT02082860 -
Phase I Gene Therapy Clinical Trial Using the Vector rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria
|
Phase 1 | |
Completed |
NCT03338816 -
ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)
|
Phase 3 | |
Completed |
NCT02452372 -
A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)
|
Phase 1 | |
Completed |
NCT00418795 -
Porphozym in the Treatment of Acute Attacks in AIP
|
Phase 2/Phase 3 | |
Active, not recruiting |
NCT01617642 -
Dental Health, Diet, Inflammation and Biomarkers in Patients With Acute Intermittent Porphyria(AIP)
|
||
Completed |
NCT02076763 -
Observational Study of Acute Intermittent Porphyria Patients
|
N/A |