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New enzyme therapy shows promise in controlling rare blood clotting disorder

NCT ID NCT03393975

Disease control Sponsor: Baxalta now part of Shire Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a medicine called BAX 930 in 52 people born with severe TTP, a rare condition that causes dangerous blood clots. The medicine replaces a missing enzyme to prevent or treat sudden flare-ups. Participants received either BAX 930 or standard treatment for 6 months, then switched, followed by everyone getting BAX 930. The goal was to see if BAX 930 reduces flare-ups and is safe.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

52 people

The number who actually took part.

Started

Oct 2017

Finished

May 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

0 to 70 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Participant or legally authorized representative has provided signed informed consent \>= 18 years of age and/or assent form (signed by legal representative if participants is \<18 years of age). * Participant is 0 to 70 years of age, inclusive, at the time of screening. (Participants \< 18 years of age will be enrolled only after at least 5 adults (\>= 18 years of age) each have at least 10 exposures with BAX 930 and reviewed by the Data Monitoring Committee (DMC). In France, no participants younger than 18 years of age will be enrolled into the study before the first adult participant has been treated with BAX 930 for a minimum of 6 months. * Participant has a documented diagnosis of severe hereditary ADAMTS13 deficiency, defined as: * Confirmed by molecular genetic testing, documented in participant history or at screening, and * ADAMTS13 activity \< 10 % as measured by the fluorescent resonance energy transfer- von Willebrand factor73 (FRETS-VWF73) assay, documented in participant history or at screening (participants currently receiving standard of care (SoC) prophylactic therapy may exceed 10% ADAMTS13 activity at screening). Note: Participants currently receiving prophylactic therapy will be screened immediately prior to their usual prophylactic infusion * Participant does not display any severe thrombotic thrombocytopenic purpura (TTP) signs (platelet count \< 100,000/ microliter (mcL) and elevation of lactate dehydrogenase (LDH) greater than (\>2)\* ULN) at screening. (Prophylactic cohort only). * Participant is currently on a prophylactic dosing regimen or has a documented history of at least 1 TTP event and an ability to tolerate SoC prophylactic dosing (prophylactic cohort only). * Participants \>= 16 years of age must have a Karnofsky score \>= 70% and participants \< 16 years of age must have a Lansky score \>= 80%. * Participant is hepatitis C virus (HCV)-negative as confirmed by antibody or polymerase chain reaction testing OR HCV-positive if their disease is chronic but stable. * If female of childbearing potential, participant presents with a negative blood or urine pregnancy test, confirmed no more than 7 days before the first administration, and agrees to employ adequate birth control measures for the duration of the study and to undergo quarterly pregnancy testing. * Sexually active males must use an accepted and effective method of contraception during the treatment and until a minimum of 16 days after the last dose administered. * Participant is willing and able to comply with the requirements of the protocol. Exclusion Criteria: * Participant has been diagnosed with any other TTP-like disorder (microangiopathic hemolytic anemia), including acquired TTP. * Participant has known hypersensitivity to hamster proteins. * Participant has experienced an acute TTP event less than 30 days prior to screening (prophylactic cohort only). * Participant has a medical history or presence of a functional ADAMTS13 inhibitor at screening. * Participant has a medical history of genetic or acquired immune deficiency that would interfere with the assessment of product immunogenicity, including participants who are human immunodeficiency virus (HIV)-positive with an absolute cluster of differentiation 4 (CD4) count \< 200/ cubic millimeter (mm\^3) or who are receiving chronic immunosuppressive drugs. * Participant has been diagnosed with severe cardiovascular disease (New York Heart Association classes 3 to 4). * Participant with end stage renal disease requiring chronic dialysis. * Participant has been diagnosed with hepatic dysfunction, as evidenced by, but not limited to, any of the following: * Serum alanine aminotransferase (ALT) \>= 2\* ULN. * Severe hypoalbuminemia \< 24 gram per liter (g/L). * Portal vein hypertension (e.g., presence of otherwise unexplained splenomegaly, history of esophageal varices). * In the opinion of the investigator, the participant has another clinically significant concomitant disease that may pose additional risks for the participant. * Participant has been treated with an immunomodulatory drug, excluding topical treatment (e.g., ointments, nasal sprays), within 30 days prior to enrollment. Use of corticosteroids in conjunction with administration of fresh frozen plasma (FFP) to prevent allergic reactions is permitted. * Participant has an acute illness (e.g., influenza, flu-like syndrome, allergic rhinitis/conjunctivitis, bronchial asthma) at the time of screening (prophylaxis cohort only). * Participant is receiving or anticipates receiving another investigational drug and/or interventional drug within 30 days before enrollment. * Participant has a history of drug and/or alcohol abuse within the last 2 years. * Participant has a progressive fatal disease and/or life expectancy of less than 3 months. * Participant is identified by the investigator as being unable or unwilling to cooperate with study procedures. * Participant suffers from a mental condition rendering him/her unable to understand the nature, scope, and possible consequences of the study and/or evidence of an uncooperative attitude. * Participant is a family member or employee of the sponsor or investigator. * If female, participant is pregnant or lactating at the time of enrollment. * Any contraindication to SoC medicinal product(s) as per local prescribing information.

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Conditions

The condition(s) this trial relates to.

congenital thrombotic thrombocytopenic purpura thrombotic thrombocytopenic purpura

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Thrombotic thrombocytopenic purpura (TTP)

Contacts and locations

Locations

  • AKH - Medizinische Universität Wien

    Vienna, 1090, Austria

  • Alliance for Childhood Diseases, Cure 4 the Kids Foundation

    Las Vegas, Nevada, 89135, United States

  • Azienda Ospedaliera Universitaria "Policlinico - Vittorio Emanuele" (Presidio Ferrarotto Alessi)

    Catania, 90124, Italy

  • Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII (Presidio Papa Giovanni XXIII)

    Bergamo, 24127, Italy

  • CHU Saint Etienne - Hôpital Nord

    Saint-Priest-en-Jarez Cedex, Pays de la Loire Region, 42270, France

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Complejo Hospitalario Universitario A Coruña

    A Coruña, La Coruña, 15006, Spain

  • Dipartimento di Medicina Traslazionale e di Precisione - "Sapienza" Universita di Roma

    Rome, 00161, Italy

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

    Milan, 20122, Italy

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Roma, 168, Italy

  • Hopital Claude Huriez - CHU Lille

    Lille, Nord, 59037, France

  • Hospital General Universitario de Alicante

    Alicante, 3010, Spain

  • Hospital Universitari i Politecnic La Fe

    Valencia, 46026, Spain

  • Hospital Universitario Virgen del Rocio

    Seville, 41013, Spain

  • Hospital Universitario de Salamanca

    Salamanca, 37007, Spain

  • Hospital de Cruces

    Barakaldo, Vizcaya, 48903, Spain

  • Hyogo College of Medicine Hospital

    Nishinomiya-shi, Hyōgo, 663-8501, Japan

  • Hôpital Necker - Enfants Malades

    Paris, Paris, 75015, France

  • Hôpital Robert Debré - Paris

    Paris, 75019, France

  • Hôpital Saint-Antoine

    Paris, Paris, 75571, France

  • Instytut Hematologii i Transfuzjologii

    Warsaw, 02-776, Poland

  • Kyushu University Hospital

    Fukuoka, Fukuoka, 812-8582, Japan

  • Medical Hospital, Tokyo Medical and Dental University

    Bunkyō City, Tokyo-To, 113-8519, Japan

  • Ohio State Univ College Of Medicine

    Columbus, Ohio, 43210, United States

  • Royal Manchester Children's Hospital

    Manchester, M139WL, United Kingdom

  • Samodzielny Publiczny Dzieciecy Szpital Kliniczny

    Warsaw, 02-091, Poland

  • The Methodist Hospital

    Houston, Texas, 77030, United States

  • Universitaetsklinikum Hamburg-Eppendorf

    Hamburg, 20246, Germany

  • Universitaetsklinikum Jena

    Jena, Thuringia, 07747, Germany

  • University College London Hospitals

    London, Greater London, NW1 2PG, United Kingdom

  • University of Oklahoma

    Oklahoma City, Oklahoma, 73104, United States

  • Winship Cancer Institute

    Atlanta, Georgia, 30322, United States

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