Clinical Trial Details
— Status: Terminated
Administrative data
NCT number |
NCT02922413 |
Other study ID # |
14-0188 |
Secondary ID |
FD-R-03720 |
Status |
Terminated |
Phase |
Phase 2
|
First received |
|
Last updated |
|
Start date |
October 30, 2015 |
Est. completion date |
December 27, 2023 |
Study information
Verified date |
April 2024 |
Source |
The University of Texas Medical Branch, Galveston |
Contact |
n/a |
Is FDA regulated |
No |
Health authority |
|
Study type |
Interventional
|
Clinical Trial Summary
The purpose of this study is to determine if Panhematin is safe and effective for prevention
of acute attacks of porphyria.
The study aims to provide high quality evidence for the use on hemin for prevention of acute
attacks of porphyria. High quality studies have not been done previously for treating or
preventing acute attacks with hemin. The lack of strong evidence for efficacy of hemin for
treatment and prevention of attacks limits its availability for patients with acute
porphyrias. Funding source: FDA Office of Orphan Product Development (FDA OOPD) FD-R-03720
Description:
This is a double-blind, randomized, placebo-controlled, parallel group trial investigating
the efficacy and safety of Panhematin™ for preventing acute attacks in at least 20 subjects
with well-documented acute porphyria (acute intermittent porphyria, hereditary coproporphyria
or variegate porphyria). Subjects will (1.) have had frequent attacks in the past, with
symptoms such as abdominal, back and/or limb pain and diagnosed after exclusion of other
causes, and (2.) be on hemin prophylaxis for prevention of frequent attacks. It is expected
that they will have had 6 or more attacks in one year before starting hemin prophylaxis. This
would be considered justification for a preventive regimen of hemin on clinical grounds.
Double blind Panhematin™ or placebo will be given. The number of doses will correspond to the
number of doses that a subject receives in approximately one week for their prophylactic
regimen. An interim analysis will be carried out after completion of 10 subjects to assess
progress and possibly adjust the sample size. The trial consists of the following visits:
- A screening visit to determine eligibility and obtain informed consent.
- A treatment visit for administration of double blind prophylactic doses of Panhematin™
or placebo corresponding to the number of doses the subject receives for their
prophylactic regimen within approximately one week.
- Follow up visit at 1, 2, 3, and 4 weeks to assess response to the infusion of
Panhematin™ or placebo. These visits will be in person or by telephone.
- Additional visits may be scheduled if needed, for example for treatment of symptoms.
- Follow-up visits 3 and 6 months after the end of treatment either in person or by
telephone Subjects will have laboratory documentation of one of the acute porphyrias.
Molecular documentation is also expected, although rarely a causative mutation cannot be
detected. Upon entry into the study they will be given in a blinded fashion one or more
preventive doses of either Panhematin™ (4 mg/kg) or placebo, the number of which will
correspond to the number of prophylactic doses they have been receiving within
approximately one week for prophylaxis. A recurrent attack within the next 1, 2, 3 and 4
weeks will represent treatment failures. Because at study entry most subjects are
expected to be on weekly prophylactic hemin treatment, and hemin is a short-acting drug,
emphasis in the analysis will be on attacks occurring within 1 week after study
treatment.
Any attacks that occur during the study will be treated according to standard of care, which
may include Panhematin™, either at the study site or at a subject's usual treatment location.
It is intended that 20 subjects will complete treatment with blinded treatment and at least 4
weeks of follow up. A completed subject is one who meets all entrance criteria, has no
exclusion criteria and completes the single dosing and at least one week of follow up, or is
withdrawn because of an adverse event.