Health TrialsbyHealthPanel

A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A

ConditionHemophilia A
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexMale
Age18+
SponsorHoffmann-La Roche

About this trial

This study will assess the safety and tolerability of SPK-8011QQ in adult males with moderately severe to severe hemophilia A.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Signed Informed Consent Form (ICF)

≥18 years of age at the time of signing the ICF

Male sex assigned at birth

Severe or moderately severe hemophilia A, defined as endogenous FVIII:C activity levels ≤3%, as documented (historically or during the Screening Period) by a certified laboratory and where the FVIII:C level is measured more than 96 hours after the prior dose of an extended half-life FVIII replacement product or more than 72 hours after the prior dose of a standard half-life FVIII replacement product

Disqualifiers

Are currently undergoing antiviral therapy for chronic hepatitis B or chronic hepatitis C

Have an inherited or acquired bleeding disorder other than hemophilia A

Have known inherited or acquired thrombophilia, have signs of thromboembolic disease in the Investigator's judgement, or are on current treatment for thromboembolic disease. A history of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing is not considered an exclusion criterion

Have had prior treatment with a vector or gene transfer agent. Nucleic acid-based vaccines, such as the vaccine for coronavirus disease 2019 (COVID-19), are not considered gene transfer agents

Trial design

Design model

Single group

Treatments tested in this trial

  • SPK-8011QQ

    Drug

    Participants will receive IV SPK-8011QQ

Treatment groups

5 Participants
are divided into 1 treatment group
Group A: SPK-8011QQExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence of participants with adverse events (AEs)

Time frame
Up to approximately 5 years
2

Severity of AEs as determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0 grading scale

Time frame
Up to approximately 5 years
3

Incidence of adverse events of special interest (AESIs)

Time frame
Up to approximately 5 years
4

Incidence of serious adverse events (SAEs)

Time frame
Up to approximately 5 years

Secondary outcomes

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Hoffmann-La Roche

Lead sponsor

Source ClinicalTrials.gov
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