Have you or your loved ones been diagnosed with Sickle Cell Disease?

If so, you may be eligible for a clinical trial.

Have you or your loved ones been diagnosed with Sickle Cell Disease? If so, you may be eligible for a clinical trial.

What is a clinical trial? Is participating in a clinical trial right for you? Learn more

Recruiting for this study? Get access to patient recruitment tools.

Sickle Cell Disease Clinical Trial in Duarte CA

NCT04091737 | Phase 1 | Interventional

Have you or your loved ones been diagnosed with Sickle Cell Disease?

If so, you may be eligible for a clinical trial.

Have you or your loved ones been diagnosed with Sickle Cell Disease? If so, you may be eligible for a clinical trial.

Recruiting

Male & Female

18 - 45

Years old

This study is looking to recruit 3 Participants

This is a phase 1 pilot study of CSL200 in adult subjects with severe sickle cell disease. The primary objectives of this study are to evaluate the safety of the following: collection of CD34+ hematopoietic stem / progenitor cells by apheresis after mobilization with plerixafor, reduced intensity conditioning with melphalan, and administration of CSL200.

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Where you'll go

Selected location

City of Hope Medical Center Change

1500 East Duarte Road, Duarte, CA, USA 91010

Joseph Rosenthal, MD 626-930-5414
jrosenthal@coh.org

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Details for the study

Brief Title

CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease

Official Title

A Phase 1 Pilot Study to Evaluate the Safety and Feasibility of Gene Therapy With CSL200 (Autologous Enriched CD34+ Cell Fraction That Contains CD34+ Cells Transduced With Lentiviral Vector Encoding Human γ-GlobinG16D and Short-Hairpin RNA734) in Adult Subjects With Severe Sickle Cell Disease

Brief Summary

Treatments and/or Procedures

Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ globinG16D and short hairpin RNA734

Cryopreserved formulated autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734 in a bag for infusion Plerixafor to mobilize hematopoietic stem cells prior to each apheresis Single dose melphalan before administration of CSL200

Outcome Measures

Outcome measures are the tests that investigators perform to prove whether or not a treatment being tested in a clinical trial is having any effect.

Primary

Number of adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESIs) associated with the administration of CSL200

Adverse event (AE) is defined as any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. Serious adverse event (SAE) is defined as any untoward medical occurrence that at any dose results in death, is life-threatening, requires in-patient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability or incapacity, or is medically significant. Adverse event of special interest (AESI) is defined in this study as any of the following: acute immune reactions, autoimmunity to CSL200; malignancy; predominant integration site in presence of malignancy or other abnormality.

Up to 48 weeks

Secondary

Vector copy number (VCN)

VCN will be determined by using the average number of CAL-H vector genomes per cell

Up to 48 weeks

Secondary

The number of subjects undergoing reduced intensity conditioning with melphalan and able to receive CSL200

Reduced intensity conditioning assessed by subjects receiving melphalan

2 days

Secondary

Number of subjects undergoing apheresis and number of apheresis sessions by subject

Collection of CD34+ HSPCs by apheresis after mobilization with plerixafor assessed by apheresis sessions

Up to 2 days

Secondary

Total by-subject number of CD34+ HSPCs collected in total and in each apheresis session

Collection of CD34+ HSPCs by apheresis after mobilization with plerixafor assessed by CD34+ HSPCs collected

Up to 2 days

Secondary

Number of subjects receiving plerixafor and number of plerixafor doses administered by subject

Collection of CD34+ HSPCs by apheresis after mobilization with plerixafor assessed by plerixafor administrations

Up to 2 days

Study Criteria

Inclusion Criteria:

  -  Diagnosis of sickle cell disease with the homozygous HbSS or an HbSβ thalassemia
     variant (ie, HbSβ0 thalassemia or HbSβ+ thalassemia) genotype, confirmed by hemoglobin
     studies.

  -  Fetal hemoglobin (HbF) ≤ 15%.

  -  Severe sickle cell disease symptomatology, defined as any one or more of the
     following:

       1. ≥ 2 episodes of acute chest syndrome in the last 2 years.

       2. ≥ 3 episodes of severe pain events requiring a visit to a medical facility and
          treatment with opioids in the last 2 years.

       3. > 2 episodes of recurrent priapism in the last 2 years.

       4. Red-cell alloimmunization (> 2 antibodies) during long-term transfusion therapy
          (lifetime history).

       5. Chronic transfusions for primary or secondary prophylaxis (lifetime history).

       6. Trans-thoracic echocardiograph evidence of tricuspid valve regurgitant jet
          velocity ≥ 2.7 m/sec (lifetime history).

       7. Clinically significant neurologic event (eg, ischemic stroke) or any neurological
          deficit lasting > 24 hours.

Exclusion Criteria:

  -  Hypoxanthine-guanine phosphoribosyl transferase (HPRT) deficiency.

  -  Thiopurine S-methyltransferase (TPMT) deficiency.

  -  Alpha thalassemia.

  -  Serum ferritin ≥ 2500 ng/mL.

  -  Inadequate bone marrow function, defined as at least 1 of the following:

       1. Absolute neutrophil count < 1000/µL.

       2. Platelet count < 120,000/µL.

Interested in learning more?

Selected location

City of Hope Medical Center

1500 East Duarte Road, Duarte, CA, USA 91010

Joseph Rosenthal, MD 626-930-5414
jrosenthal@coh.org

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