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Controlled Trial of Panhematin in Treatment of Acute Attacks of Porphyria

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ClinicalTrials.gov Identifier: NCT02180412
Recruitment Status : Recruiting
First Posted : July 2, 2014
Last Update Posted : July 27, 2018
Sponsor:
Information provided by (Responsible Party):
The University of Texas Medical Branch, Galveston

Brief Summary:

This study aims to provide high quality evidence for the effectiveness and safety of hemin (PanhematinTM , Recordati) for treatment of acute attacks of porphyria. These types of studies have not been done before with either PanhematinTM or the hemin preparation available in Europe (NormosangTM, Orphan Europe).

There are two treatment groups in this study. One group will be treated with PanhematinTM plus glucose, and the other group will be treated with glucose plus an inactive salt solution (called a "placebo"). To avoid prejudice, the treatment given to each participant will be blinded (meaning the participants and most of the hospital staff will not know which treatment the participant will receive) and randomized (meaning participants will have an equal chance of receiving either treatment, like the flip of a coin). A placebo-controlled, randomized study is the standard method used to prove treatments are effective and safe. PanhematinTM and glucose will be given in the same manner as is usual for treating an attack of porphyria. For participants who are chosen to receive the placebo, their treatment will be switched to real PanhematinTM at any time if their symptoms do not improve. This is called "rescue" treatment, and assures that they study is safe and patients who need hemin will receive it. Treatment with hemin will be for 4 days, or longer if needed. Since the study treatment is started as soon as possible after symptoms appear, there will be very little delay in providing hemin to those who need it. Funding Source - Office of Orphan Products Development (FDA OOPD)


Condition or disease Intervention/treatment Phase
Acute Porphyrias Biological: Panhematin Other: Glucose Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Double-blind, Randomized, Placebo-controlled, Parallel Group Trial on the Efficacy and Safety of PanhematinTM in the Treatment of Acute Attacks of Porphyria
Study Start Date : April 2014
Estimated Primary Completion Date : August 31, 2020
Estimated Study Completion Date : August 31, 2020


Arm Intervention/treatment
Experimental: Panhematin
Panhematin plus glucose
Biological: Panhematin
Glucose loading
Other Name: Glucose

Other: Glucose
Glucose is administered to both groups as routine care.

Placebo Comparator: Placebo
Placebo (saline) plus glucose
Other: Glucose
Glucose is administered to both groups as routine care.




Primary Outcome Measures :
  1. Pain scale [ Time Frame: 4 days ]
    Numeric rating scale for pain (0-10; 0=no pain, 10=most severe pain)


Secondary Outcome Measures :
  1. Biochemical effects of Panhematin [ Time Frame: 4 days ]
    Porphyrin precursors and porphyrins


Other Outcome Measures:
  1. Effects of clinical features on response to Panhematin [ Time Frame: 4 days ]
    Age, sex, exacerbating factors

  2. Effects of genetic features on response to Panhematin [ Time Frame: 4 days ]
    Types of mutations

  3. Use of reconstitution of Panhematin with albumin [ Time Frame: 4 days ]
    Frequency of side effects or adverse events



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Ages Eligible for Study:   18 Years to 100 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female aged 18 years
  • Willing to provide written informed consent
  • Acute symptoms (7 days duration or less to time of enrollment) such as abdominal, back and/or limb pain, diagnosed by the investigator as caused by porphyria after initial evaluation has excluded other causes.
  • Diagnosis of acute porphyria documented by a substantial increase in urinary or serum porphobilinogen (PBG).
  • Type of acute porphyria confirmed by additional testing (in addition to increased PBG), which may be completed before or after treatment begins using pretreatment samples:
  • For acute intermittent porphyria (AIP): Normal or only slight increases in plasma and fecal porphyrins. Most (~90 percent) will have deficient activity of erythrocyte porphobilinogen deaminase (PBGD), and almost all (>95 percent) will have a demonstrable disease-causing PBGD mutation.
  • For hereditary coproporphyria (HCP): 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. Almost all (>95 percent) will have a demonstrable disease-causing coproporphyrinogen oxidase (CPO) mutation.
  • For variegate porphyria (VP): 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. Almost all (~95 percent) will have a demonstrable disease-causing protoporphyrinogen oxidase (PPO) mutation.

Exclusion Criteria:

  • Symptoms such as abdominal, back or limb pain are explained by another condition, as judged by the investigator
  • Therapy with hemin within 7 days prior to enrollment in this study
  • Known or suspected allergy to Panhematin™ or related products
  • Preexisting coagulation defect or concurrent treatment with an anticoagulant
  • Previously documented renal impairment defined as a serum creatinine above 1.7 mg/dL or 150 mmol/L.
  • A diagnosis of diabetes mellitus, which might increase the risk of glucose infusion.
  • Heart failure, significant chronic anemia or any disease or condition that the investigator judges would lead to an unacceptable risk to the patient or interfere with the successful collection of date for the trial
  • Previous randomization in this trial

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02180412


Contacts
Contact: Kristin J Parks 9198302458 klparks@utmb.edu
Contact: Karl E Anderson, MD 409-772-4661 kanderso@utmb.edu

Locations
United States, Texas
University of Texas Medical Branch Recruiting
Galveston, Texas, United States, 77555
Contact: Karl E Anderson, MD    409-772-4661    kanderso@utmb.edu   
Contact: Csilla Hallberg, MD    409-772-4661    challberg@utmb.edu   
Principal Investigator: Karl E Anderson, MD         
Sponsors and Collaborators
The University of Texas Medical Branch, Galveston
Investigators
Principal Investigator: Karl E Anderson, MD UT, Galveston

Responsible Party: The University of Texas Medical Branch, Galveston
ClinicalTrials.gov Identifier: NCT02180412     History of Changes
Other Study ID Numbers: 10-203
FD-R-03720 ( Other Grant/Funding Number: FDA OOPD )
First Posted: July 2, 2014    Key Record Dates
Last Update Posted: July 27, 2018
Last Verified: July 2018

Keywords provided by The University of Texas Medical Branch, Galveston:
Acute porphyria
Hemin

Additional relevant MeSH terms:
Porphyrias
Porphyria, Erythropoietic
Porphyria, Acute Intermittent
Metabolic Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Porphyrias, Hepatic
Liver Diseases
Digestive System Diseases